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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

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Frequently Asked Questions

How much do clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest clinical trials?

Most recently, we added Gene Therapy for Charcot-Marie-Tooth Disease, Tolinapant + Eribulin for Breast Cancer and CAR T Cell Therapy for Brain Cancer to the Power online platform.

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Gene Therapy for Huntington's DiseaseColumbus, Ohio

The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.53 Participants Needed

Epcoritamab + Ibrutinib for Non-Hodgkin's LymphomaColumbus, Ohio

This phase Ib/II trial evaluates the safety, optimal dose, and efficacy of the combination of epcoritamab and ibrutinib in treating patients with aggressive B-cell non-Hodgkin lymphoma that has come back (relapsed) or responded to previous treatment (refractory). Epcoritamab, a bispecific antibody, binds to two different types of receptors (proteins present on the cell surface) at the same time. The two receptors that epcoritamab binds to are called CD3 and CD20. CD3 is found on T cells, which are important cells of the immune system that help fight cancer and infections. CD20 is found on the surface of most types of aggressive B-cell non-Hodgkin lymphoma cells. By binding to both CD3 and CD20, epcoritamab brings the two cells close together so the T cells can fight and kill the lymphoma B cells. Ibrutinib, a Bruton's tyrosine kinase (BTK) inhibitor, binds to a protein on B cells, a type of white blood cell from which the lymphoma developed. By doing this it decreases the ability of the lymphoma B cells to survive and grow. Ibrutinib may also improve the health (or fitness) of T cells thus making epcoritamab safer and/or more effective.38 Participants Needed

Gene Therapy for Acute Lymphoblastic LeukemiaColumbus, Ohio

This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19-positive hematologic malignancies.32 Participants Needed

SMP-3124LP for CancerColumbus, Ohio

An Open-label, Phase I Dose Escalation and Phase 2 Dose Expansion Study to Assess Safety, Tolerability, Preliminary Antitumor Activity of SMP 3124LP in Adults with Advanced Solid Tumors120 Participants Needed

Iadademstat + Atezolizumab/Durvalumab for Small Cell Lung CancerColumbus, Ohio

This phase I/II trial tests the safety, side effects, and best dose of iadademstat when given together with atezolizumab or durvalumab, and studies the effect of the combination in treating patients with small cell lung cancer that has spread outside of the lung in which it began or to other parts of the body (extensive stage) who initially received standard of care chemotherapy and immunotherapy. Iadademstat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as atezolizumab or durvalumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Adding iadademstat to either atezolizumab or durvalumab may be able to stabilize cancer for longer than atezolizumab or durvalumab alone in treating patients with extensive stage small cell lung cancer.45 Participants Needed

Iberdomide + Belantamab Mafodotin + Dexamethasone for Multiple MyelomaColumbus, Ohio

This phase I/II trial tests the safety, side effects, best dose, and effectiveness of iberdomide in combination with belantamab mafodotin and dexamethasone in treating patients with multiple myeloma (MM) that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Multiple myeloma is a cancer that affects white blood cells called plasma cells, which are made in the bone marrow and are part of the immune system. Multiple myeloma cells have a protein on their surface called B-cell maturation antigen (BCMA) that allows the cancer cells to survive and grow. Immunotherapy with iberdomide, may induce changes in body's immune system and may interfere with the ability of cancer cells to grow and spread. Belantamab mafodotin has been designed to attach to the BCMA protein, which may cause the myeloma cell to become damaged and die. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Iberdomide plus belantamab mafodotin may help slow or stop the growth of cancer in patients with multiple myeloma.88 Participants Needed

CMTX-101 for Cystic FibrosisColumbus, Ohio

CMTX-101 is a bacterial biofilm disrupting monoclonal antibody being developed as an adjunctive therapy to standard of care antibiotics. The goal of this clinical trial is to assess the safety and tolerability of CMTX-101 in people with cystic fibrosis (pwCF). The main questions the study aims to answer are: * Are single doses of CMTX-101 IV infusion safe and tolerated * What is the pharmacokinetic (PK) profile of single doses of CMTX-101 * Do single doses of CMTX-101 induce development of anti-drug antibodies (ADA) and neutralizing antibodies (Nabs)41 Participants Needed

Gene Therapy for Duchenne Muscular DystrophyColumbus, Ohio

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.40 Participants Needed

Triple Immune Regimen for HIVColumbus, Ohio

The purpose of this study is to evaluate the safety, tolerability, and efficacy of therapeutic vaccination with chimpanzee adenovirus ChAdOx1- and poxvirus modified vaccinia Ankara (MVA)-vectored conserved mosaic T-cell vaccines in a sequential regimen with the toll-like receptor 7 (TLR7) agonist vesatolimod (VES) and two broadly neutralizing antibodies (bNAbs) compared to placebo, to induce HIV-1 control during analytic treatment interruption (ATI).45 Participants Needed

Gene Therapy for Frontotemporal DementiaColumbus, Ohio

The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.9 Participants Needed

Gene Therapy for EpilepsyColumbus, Ohio

This trial is testing a new treatment called AMT-260 for adults with a type of epilepsy that doesn't respond to usual treatments. The treatment is delivered directly to the affected part of the brain using MRI guidance. The goal is to see if this method can better control seizures and improve safety and tolerability.12 Participants Needed

OBX-115 for Skin CancerColumbus, Ohio

This trial tests a new treatment called OBX-115 for adults with advanced solid tumors. It aims to see if the treatment is safe and effective by measuring its ability to shrink tumors, control disease progression, and improve survival.52 Participants Needed

RSP-1502 Inhalation for Cystic FibrosisColumbus, Ohio

A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.72 Participants Needed

Brain Radiotherapy for Lung CancerColumbus, Ohio

This study will consist of a Phase 1b and Phase 2 portion. The Phase 1b portion will enroll first followed by the Phase 2 portion. Each cycle of treatment = 28 days. Subjects will receive alectinib twice daily. Those in the Phase 1b portion will receive alectinib alone. Those in Phase 2 Arm A will receive alectinib alone. Those in Phase 2, Arm B will receive SRS + alectinib. A maximum of 25 cycles (2 years) of alectinib may be administered on study.56 Participants Needed

Citicoline for Coronavirus-related Lung InjuryColumbus, Ohio

This trial is testing if citicoline, given through a vein, is safe and helpful for adults with severe COVID-19 who have trouble breathing. The study aims to see if citicoline can improve oxygen levels and reduce illness severity. Patients will receive either citicoline or another treatment, and their oxygen levels will be monitored regularly.80 Participants Needed

STM-416 for Bladder CancerColumbus, Ohio

This trial tests STM-416, a new treatment, in bladder cancer patients whose disease has returned after standard treatments. The treatment is given to see if it is safe and effective.75 Participants Needed

XTX301 for Solid TumorsColumbus, Ohio

This is a first-in-human, multicenter, Phase 1/2, open-label study designed to evaluate the safety and tolerability of XTX301 as monotherapy in patients with advanced solid tumors.358 Participants Needed

Gene-Modified T Cell Therapy for GlioblastomaColumbus, Ohio

This multicenter, Phase 1b/2 study is being conducted to determine if the experimental cell therapy is safe, tolerable and can delay the return of cancer in patients with a newly diagnosed or recurrent glioblastoma multiforme (GBM) in combination with standard chemotherapy treatment temozolomide (TMZ). If there is a 25% or greater improvement in survival in this study then the therapy should be studied further.4 Participants Needed

Targeted Alpha-Particle Therapy for Neuroendocrine TumorsColumbus, Ohio

This study is Phase I/IIa First-in-Human Study of \[212Pb\]VMT-α-NET Targeted Alpha-Particle Therapy for Advanced SSTR2 Positive Neuroendocrine Tumors280 Participants Needed

NK Cells + Chemotherapy for SarcomaColumbus, Ohio

The purpose of this study is to determine if the addition of infusions of a type of immune cell called a "natural killer", or NK cell to the sarcoma chemotherapy regimen GEM/DOX (gemcitabine and docetaxel) can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies. The goals of this study are: * To determine the safety and efficacy of the addition of adoptive transfer of universal donor, TGFβ imprinted (TGFβi), expanded NK cells to the pediatric sarcoma salvage chemotherapeutic regimen gemcitabine/docetaxel (GEM/DOX) for treatment of relapsed and refractory pediatric sarcomas To determine the 6-month progression free survival achieved with this treatment in patients within cohorts of relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and non-rhabdomyosarcoma soft tissue sarcoma. * To identify toxicities related to treatment with GEM/DOX + TGFβi expanded NK cells Participants will receive study drugs that include chemotherapy and NK cells in cycles; each cycle is 21 days long and you can receive up to 8 cycles. * Gemcitabine (GEM): via IV on Days 1 and 8 * Docetaxel (DOX): via IV on Day 8 * Prophylactic dexamethasone: Day 7-9 to prevent fluid retention and hypersensitivity reaction * Peg-filgrastim (PEG-GCSF) or biosimilar: Day 9 to help your white blood cell recover and allow more chemotherapy to be given * TGFβi NK cells: via IV on Day 1250 Participants Needed

NK Cell Therapy + Temozolomide for Melanoma with Brain MetastasisColumbus, Ohio

This trial is testing a combination of special immune cells and a cancer drug to treat patients with advanced melanoma that has spread to the brain. The immune cells aim to kill the cancer, and the drug helps stop its growth. A new treatment has shown effectiveness against advanced melanoma and brain involvement.30 Participants Needed

Tazemetostat + Rituximab + Bendamustine for Follicular LymphomaColumbus, Ohio

This study is planned as a single arm clinical trial of tazemetostat in combination with bendamustine and rituximab with both a phase I and phase II component. All patients will receive tazemetostat twice daily on days 1-28 in combination with bendamustine 90 mg/m2 IV on days 1 and 2 and rituximab 375 mg/m2 IV on day 1 of a 28-day cycle for up to three cycles. Following this, patients will receive tazemetostat twice daily on days 1-28 and rituximab 375 mg/m2 IV on day 1 of a 28-day cycle for up to three cycles.42 Participants Needed

Ipatasertib + Megestrol Acetate for Endometrial CancerColumbus, Ohio

This trial is testing if combining two drugs, ipatasertib and megestrol acetate, is more effective than using megestrol acetate alone for treating women with endometrial cancer that has returned or spread. Ipatasertib blocks enzymes needed for cancer cell growth, while megestrol acetate reduces and blocks estrogen, which some cancer cells need to grow.96 Participants Needed

DYNE-251 for Duchenne Muscular DystrophyColumbus, Ohio

This trial tests DYNE-251, an IV drug, in people with Duchenne muscular dystrophy who can benefit from exon 51 skipping. The drug aims to help their muscles by increasing the dystrophin protein. It works by skipping a faulty part of their gene to produce more of this important protein. Eteplirsen (Exondys 51) was the first drug approved for exon 51 skipping in Duchenne muscular dystrophy.86 Participants Needed

Natural Killer Cell Therapy for Acute Myeloid LeukemiaColumbus, Ohio

This is a phase I/II dose escalation study designed to determine the safety and estimate the efficacy of UD-NK cells combined with FLA chemotherapy in patients age 1-24.99 with relapsed or refractory acute myeloid leukemia. PRIMARY OBJECTIVE: I. To determine the safety and recommended phase II dose of adoptive NK cell therapy using UD-NK cells in pediatric and young adult patients with relapsed/refractory AML. SECONDARY OBJECTIVES: I. To estimate the efficacy of UD- NK cells with FLA chemotherapy in pediatric and young adult patients with relapsed/refractory AML. EXPLORATORY OBJECTIVES: I. To determine the immunophenotype and function of UD-NK cells II. To characterize in vivo expansion of UD-NK cells III. To determine the persistence of UD-NK cells Six doses of universal donor mbIL-21 expanded NK cells (UD-NK) given thrice weekly for two weeks. Days may vary and NK cells can be given from days 0 to 21. Patients may receive up to 2 cycles of fludarabine/cytarabine (FLA) + NK cells (up to 12 NK cell infusions) if they do not achieve CR after cycle 1 or if necessary to bridge to transplant.20 Participants Needed

Peanut SLIT-Tablet for Peanut AllergyColumbus, Ohio

This trial tests a tablet containing small amounts of peanut protein taken under the tongue. It aims to help people with peanut allergies become less sensitive to peanuts. Participants will take gradually increasing doses to see if their tolerance improves.192 Participants Needed

Iberdomide Combo for Multiple MyelomaColumbus, Ohio

Similar to the paradigm established in other hematologic malignancies that are considered curable, the achievement of MRD(-) status is necessary for long term disease control in MM. The fact that the majority of patients remain MRD (+) after induction therapy and AHCT points to the opportunity to deploy novel agents with complementary mechanism of action and favorable toxicity profile to reach and maintain MRD (-) status. Given its favorable toxicity profile, the convenience of oral administration, and compelling single agent activity even in heavily pretreated MM, iberdomide is likely amenable to long term therapy in patients with high-risk of relapse/progression identified by the persistence of MRD(+). The investigators intend to develop combination(s) of iberdomide with other agents with complementary mechanism of action in the consolidation setting post AHCT in order to achieve and sustain MRD (-).80 Participants Needed

Selinexor + Temozolomide for Recurrent GlioblastomaColumbus, Ohio

This trial tests selinexor with temozolomide for patients whose brain cancer has returned. Selinexor blocks a protein that helps cancer cells grow, and temozolomide damages their DNA to kill them or stop their growth. Temozolomide is a DNA methylating drug currently used as a first-line treatment in glioblastoma therapy.97 Participants Needed

Tiragolumab + Atezolizumab for Solid TumorsColumbus, Ohio

This trial studies how well tiragolumab and atezolizumab work in children and adults with difficult-to-treat tumors missing specific genes. These treatments help the immune system attack cancer and stop it from growing. Atezolizumab is an antibody that has been used with other treatments for various cancers.86 Participants Needed

BDTX-1535 + Temozolomide for Glioblastoma and Lung CancerColumbus, Ohio

BDTX-1535-101 is an open-label, Phase 1 dose escalation and Phase 2 multiple cohort study designed to evaluate the safety, pharmacokinetics (PK), optimal dosage, central nervous system (CNS) activity, and antitumor activity of BDTX-1535. The study population comprises adults with either advanced/metastatic non-small cell lung cancer (NSCLC) with non-classical or acquired epidermal growth factor receptor (EGFR) resistance (EGFR C797S) mutations with or without CNS disease (in Phase 1 and Phase 2), or glioblastoma (GBM) expressing EGFR alterations (Phase 1 only). All patients will self-administer BDTX-1535 monotherapy by mouth in 21-day cycles. Phase 1 enrollment is now complete. Phase 2 is currently enrolling.200 Participants Needed

Gene Therapy (4D-710) for Cystic FibrosisColumbus, Ohio

This trial is testing a new gene therapy called 4D-710 in adults with cystic fibrosis who can't use standard treatments. The goal is to see if it is safe and effective. 4D-710 is part of a new generation of AAV vectors being developed for cystic fibrosis gene therapy.40 Participants Needed

Oral Azacitidine for LeukemiaColumbus, Ohio

This trial studies the best dose and effects of oral azacitidine in patients with T-cell large granular lymphocytic leukemia that has come back or did not respond to previous treatments. Azacitidine is a chemotherapy drug that stops cancer cells from growing, dividing, or spreading.27 Participants Needed

Avutometinib + Sotorasib for Lung CancerColumbus, Ohio

This study will assess the safety and efficacy of avutometinib (VS-6766) in combination with sotorasib with or without defactinib in patients with KRAS G12C Non-Small Cell Lung Cancer (NSCLC) in patients who have been exposed to prior G12C inhibitor and those who have not been exposed to prior G12C inhibitor.153 Participants Needed

Elimusertib for Solid CancersColumbus, Ohio

This trial tests elimusertib, a pill that blocks enzymes needed for cancer cell growth, in children and young adults with difficult-to-treat cancers. The drug aims to stop cancer cells from repairing and growing.31 Participants Needed

XTX202 for Solid TumorsColumbus, Ohio

This trial is testing XTX202, a new drug designed to treat advanced cancers. It targets patients with late-stage solid tumors that may not respond to other treatments. The drug remains inactive until it reaches the tumor, where it then activates to help the immune system fight the cancer.189 Participants Needed

DSP-5336 for LeukemiaColumbus, Ohio

This trial is testing a new drug called DSP 5336 to help treat adults with certain types of blood cancer that have come back or didn't respond to previous treatments. The study will first find the best amount to give and then check how safe and helpful it is.362 Participants Needed

CFI-400945 + Azacitidine for LeukemiaColumbus, Ohio

The purpose of this study is to test the safety of an investigational drug called CFI-400945 alone and in combination with azacitidine.72 Participants Needed

Diagnostic Testing for Pediatric LeukemiaColumbus, Ohio

This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults.960 Participants Needed

T-lymphocyte Therapy for Adenovirus InfectionsColumbus, Ohio

This open-label, single-arm, phase I/II clinical trial will assess the safety and efficacy of related donor adenovirus-specific T lymphocytes isolated from whole blood or leukapheresis products. The adenovirus-specific T lymphocytes will be generated automatically by the CliniMACS Prodigy using the CliniMACS Cytokine Capture System (IFN-γ) after incubation with MACS GMP PepTivator Peptide Pools of Hexon 5 for enrichment.12 Participants Needed

AB-101 + Immunotherapy for Non-Hodgkin's LymphomaColumbus, Ohio

AB-101 is an off-the shelf, allogeneic cell product made of "natural killer" cells, also called NK cells. White blood cells are part of the immune system and NK cells are a type of white blood cell that are known to kill cancer cells. This clinical trial will enroll patients with relapsed/refractory non-Hodgkin lymphoma of B-cell origin and is conducted in two phases. The primary objectives of Phase 1 are as follows: 1) to evaluate the safety of AB-101 given alone or in combination with rituximab (including the DLBCL specific cohort) or in combination with bendamustine and rituximab; 2) to evaluate the potential clinical activity of AB-101 when given in combination with rituximab or in combination with bendamustine and rituximab (combination cohorts only); and 3) to identify the recommended Phase 2 dose (RP2D). The primary objective of Phase 2 is to determine whether AB-101 in combination with rituximab or in combination with bendamustine and rituximab has anti-cancer activity in patients. Patients will be assigned to receive either AB-101 alone as monotherapy, in combination with rituximab (including DLBCL specific cohort) or in combination with bendamustine and rituximab. All patients will receive at least 1 treatment cycle of AB-101, followed by scheduled assessments of overall health and tumor response. Patients receiving AB-101 in combination with rituximab may receive up to 3 additional cycles of treatment. Patients receiving AB-101 in combination with bendamustine and rituximab may receive up to 5 additional cycles of treatment. Patients enrolled into the DLBCL specific cohort receiving AB-101 in combination with rituximab may receive up to 3 cycles of treatment.45 Participants Needed

Bosutinib for Chronic Myeloid LeukemiaColumbus, Ohio

This trial tests bosutinib, a daily oral medication, in children with a type of leukemia. It aims to find a safe dose and see how well it works in those newly diagnosed or who haven't responded to other treatments. Bosutinib helps by blocking proteins that cancer cells need to grow. Bosutinib is a type of medication used for treating leukemia, especially in cases not responding to other treatments.60 Participants Needed

NK Cell Infusion + Chemotherapy for NeuroblastomaColumbus, Ohio

This trial is testing a new cancer treatment that combines special immune cells with three cancer-fighting drugs. It aims to see if this combination is safe and effective for patients whose cancers are hard to treat with standard therapies.31 Participants Needed

Dabrafenib + Trametinib + Hydroxychloroquine for Brain TumorColumbus, Ohio

This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive) or tumors that came back while receiving a similar agent (recurrent). Patients must also have specific genetic mutations including BRAF V600 mutations or BRAF fusion/duplication, with or without neurofibromatosis type 1. Neurofibromatosis type 1 is an inherited genetic condition that causes tumors to grow on nerve tissue. Hydroxychloroquine, works in different ways to stop the growth of tumor cells by killing the cells or stopping them from dividing. Trametinib and dabrafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving hydroxychloroquine with trametinib and/or dabrafenib may lower the chance of brain tumors growing or spreading compared to usual treatments.57 Participants Needed

BK CTL for Viral InfectionsColumbus, Ohio

BK cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be safe and effective in decreasing specific viral load in children, adolescents and young adults (CAYA) with refractory BK infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) or with primary immunodeficiencies (PID).40 Participants Needed

Oral TP-3654 for MyelofibrosisColumbus, Ohio

This study is a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of nuvisertib (TP-3654) in patients with intermediate or high-risk primary or secondary MF.240 Participants Needed

Gene Therapy for Huntington's DiseaseColumbus, Ohio

This trial tests AMT-130, a one-time gene therapy, in patients with early-stage Huntington's Disease. The treatment aims to lower a harmful brain protein to slow down the disease's progression. AMT-130 has shown promise in early research.37 Participants Needed

PMD-026 for Breast CancerColumbus, Ohio

The purpose of this study is to test the safety and tolerability of PMD-026 in patients with metastatic breast cancer. PMD-026 is a targeted oral agent designed to kill tumor cells in metastatic breast cancer.61 Participants Needed

SNDX-5613 for Acute LeukemiaColumbus, Ohio

Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia. In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of revumenib.413 Participants Needed

Vyxeos for Acute Myeloid LeukemiaColumbus, Ohio

This phase II trial studies the side effects and how well Vyxeos works in treating patients with intermediate and high-risk acute myeloid leukemia who have failed an initial cycle of standard cytarabine and daunorubicin chemotherapy. Vyxeos is a combination of both chemotherapy drugs cytarabine and daunorubicin contained in a liposome. Drugs used in chemotherapy, such as cytarabine and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Cytarabine and daunorubicin given together in liposomes may have fewer side effects and work better than cytarabine and daunorubicin given alone in patients with acute myeloid leukemia.28 Participants Needed

BXCL701 + Pembrolizumab for Prostate CancerColumbus, Ohio

An open-label, multicenter, Phase 1b/2 study to identify the recommended Phase 2 dose and assess the efficacy and safety of BXCL701 administered orally, as monotherapy and in combination with PEMBRO, in patients with mCRPC. Patients enrolled in the Phase 2a portion of the study will have either Small Cell Neuroendocrine Prostate Cancer(SCNC)(Cohort A) or adenocarcinoma phenotype (Cohort B), while the Phase 2b randomized portion of the study will enroll only the histologic subtype(s) showing preliminary evidence in Phase 2a. The study will also assess other efficacy parameters, such as rPFS, PSA PFS, OS, and DOR, as well as the safety of the combined treatment. The study will consist of three components.98 Participants Needed

CX-4945 for MedulloblastomaColumbus, Ohio

This trial tests an oral drug called CX-4945 for patients with a recurring type of brain tumor. The drug aims to block a protein that helps cancer cells grow. The study will determine the best dose and check for side effects. CX-4945 has shown promising results in other types of cancer and may be developed as a new therapy.66 Participants Needed

Ruxolitinib + CPX-351 for Acute Myeloid LeukemiaColumbus, Ohio

This phase I/II trial studies the best dose of ruxolitinib when given together with CPX-351 and to see how well they work in treating patients with accelerated phase or blast phase myeloproliferative neoplasm. Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. CPX-351 is a mixture of 2 chemotherapy drugs (daunorubicin and cytarabine) given for leukemia in small fat-based particles (liposomes) to improve the drug getting into cancer cells. Giving ruxolitinib and CPX-351 may work better in treating patients with secondary acute myeloid leukemia compared to CPX-351 alone.12 Participants Needed

Tuspetinib for Acute Myeloid LeukemiaColumbus, Ohio

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.240 Participants Needed

2'-FL Supplement for Inflammatory Bowel DiseaseColumbus, Ohio

This trial tests 2'-FL, a sugar from breast milk, as a dietary supplement for young IBD patients in stable remission. It aims to boost good gut bacteria and improve gut health by increasing butyrate levels. 2'-FL, a human milk oligosaccharide, has been shown to promote the growth of beneficial gut bacteria and improve gut health in various studies.116 Participants Needed

Telaglenastat + Osimertinib for Non-Small Cell Lung CancerColumbus, Ohio

This phase Ib trial studies the side effects and best dose of telaglenastat hydrochloride when given together with osimertinib in treating patients with stage IV non-small cell lung cancer and a mutation in the EGFR gene. Telaglenastat hydrochloride and osimertinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.23 Participants Needed

Gene Therapy for Charcot-Marie-Tooth DiseaseColumbus, Ohio

This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.3 Participants Needed

Cell-Depleted Donor Grafts for Blood CancersColumbus, Ohio

This is a Phase I/II study designed to evaluate the kinetics of hematopoietic reconstitution and the incidence of acute chronic GVHD after partially matched related donor hematopoietic cell transplantation using an αβTCR/CD19+ cell depleted graft.20 Participants Needed

CAB-AXL-ADC + PD-1 Inhibitor for SarcomaColumbus, Ohio

This trial is testing mecbotamab vedotin, a medicine that targets and kills cancer cells, in patients with advanced solid tumors, including specific types of sarcomas. It works by using an antibody to find the cancer cells and a drug to destroy them.300 Participants Needed

Olaparib + Radium-223 for Prostate CancerColumbus, Ohio

This phase I/II trial studies the best dose and side effects of olaparib and how well it works with radium Ra 223 dichloride in treating patients with castration-resistant prostate cancer that has spread to the bone and other places in the body (metastatic). PARPs are proteins that help repair DNA mutations. PARP inhibitors, such as olaparib, can keep PARP from working, so tumor cells can't repair themselves, and they may stop growing. Radioactive drugs, such as radium Ra 223 dichloride, may carry radiation directly to tumor cells and not harm normal cells. Giving olaparib and radium Ra 223 dichloride may help treat patients with castration-resistant prostate cancer.133 Participants Needed

Inotuzumab Ozogamicin Post-Transplant for Leukemia and LymphomaColumbus, Ohio

This study has two phases, Phase I and Phase II. The main goal of the Phase I portion of this research study is to see what doses post-transplant inotuzumab ozogamicin can safely be given to subjects without having too many side effects. The Phase II portion of this study is to see what side effects are seen with medication after transplant. Inotuzumab ozogamicin is a combination of an antibody and chemotherapy which has been shown to have significant activity against relapsed/refractory acute lymphocytic leukemia (ALL). Inotuzumab ozogamicin is considered experimental in this study.44 Participants Needed

Nivolumab + Lenalidomide for LymphomaColumbus, Ohio

This trial studies the combination of lenalidomide and nivolumab for patients with non-Hodgkin or Hodgkin lymphoma that has come back and does not respond to treatment. Lenalidomide works by killing or stopping the growth of cancer cells, while nivolumab helps the immune system attack the cancer. The goal is to find the best dose and see how well this combination works. Lenalidomide has shown significant activity in some subtypes of lymphoma, and combining it with other treatments like rituximab has been promising.36 Participants Needed

ASTX660 for CancerColumbus, Ohio

This trial is testing ASTX660, a new drug for patients with advanced cancers who have no other treatment options. It works by blocking proteins that help cancer cells survive, making it easier to kill them.230 Participants Needed

Acalabrutinib + Combination Therapy for B-Cell LymphomaColumbus, Ohio

Part 1: To characterize the safety profile of acalabrutinib alone or in combination with rituximab in subjects with R/R FL. Part 2: To characterize the activity of acalabrutinib alone or in combination with rituximab in subjects with R/R MZL, as measured by ORR. Part 3: To characterize the safety of acalabrutinib in combination with rituximab and lenalidomide in subjects with R/R FL113 Participants Needed

Navitoclax + Dabrafenib + Trametinib for MelanomaColumbus, Ohio

This phase I/II trial studies the side effects and best dose of dabrafenib, trametinib, and navitoclax and to see how well they work in treating patients with BRAF mutant melanoma or solid tumors that has spread from where it first started (primary site) to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Dabrafenib and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Navitoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of tumor cells by blocking Bcl-2, a protein needed for tumor cell survival. Giving navitoclax, dabrafenib, and trametinib may help shrink tumors in patients with melanoma.75 Participants Needed

Brentuximab Vedotin + Nivolumab ± Ipilimumab for Hodgkin's LymphomaColumbus, Ohio

This phase I/II trial studies the side effects and best dose of ipilimumab and nivolumab when given together with brentuximab vedotin, and how well they work in treating patients with Hodgkin lymphoma that has returned after a period of improvement (recurrent) or has not responded to previous treatment (refractory). Immunotherapy with monoclonal antibodies, such as ipilimumab and nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of cancer cells to grow and spread. Brentuximab vedotin is a monoclonal antibody, brentuximab, linked to a toxic agent called vedotin. Brentuximab attaches to CD30 positive cancer cells in a targeted way and delivers vedotin to kill them. It is not known whether giving brentuximab vedotin and nivolumab with or without ipilimumab may kill more cancer cells.146 Participants Needed

RO7446603 + Aflibercept/Faricimab for Diabetic Macular EdemaColumbus, Ohio

This study aims to evaluate the ocular and systemic safety, tolerability and efficacy of RO7446603 in participants with diabetic macular edema (DME). The study consists of 2 segments: Phase I (Parts 1-4) and Phase II (Part 5). Phase I investigated the safety of RO7446603 following a single and multiple intravitreal (IVT) doses as monotherapy or co-administered with IVT aflibercept or IVT faricimab (in separate injections). Phase II will investigate the safety, tolerability, pharmacokinetics (PK) and efficacy of two dose levels of RO7446603 in combination with faricimab, with the two drugs co-mixed and administered as a single IVT injection, compared to faricimab alone. The first participant was enrolled in the Phase I segment on June 22, 2022. Phase I has been completed.546 Participants Needed

TNG456 + Abemaciclib for Solid TumorsColumbus, Ohio

This is a first in human study of TNG456 alone and in combination with abemaciclib in patients with advanced or metastatic solid tumors known to have an MTAP loss. The first part of the study is an open-label, dose escalation and the second part is an open label dose expansion in specific solid tumor types with a confirmed MTAP loss. The study drug, TNG456, is a selective PRMT5 inhibitor administered orally. The study is planned to treat up to 191 participants.191 Participants Needed

Telisotuzumab Adizutecan + Budigalimab for Non-Small Cell Lung CancerColumbus, Ohio

Non small cell lung carcinoma (NSCLC) is the most frequently occurring histologic subtype of lung cancer and is the leading cause of cancer-related deaths worldwide. The purpose of this study is to assess adverse events and change in disease activity when Telisotuzumab Adizutecan (ABBV-400) is given in combination with a programmed cell death receptor 1 (PD1) inhibitor (budigalimab) to adult participants to treat NSCLC. ABBV-400 and budigalimab are investigational drugs being developed for the treatment of NSCLC. This study will be divided into two stages, with the first stage treating participants with several doses of ABBV-400 in combination with budigalimab within the dose escalation regimen until the dose reached is tolerable and expected to be efficacious. In Stage 2 there will be 4 treatment groups. Two groups will receive budigalimab with different optimized doses of telisotuzumab adizutecan (to allow for the best dose to be studied in the future). One group will receive budigalimab, pemetrexed, and investigator's choice of carboplatin or cisplatin, followed by budigalimab and pemetrexed. One group will receive the standard of care (SOC) pembrolizumab, pemetrexed, and investigator's choice of carboplatin or cisplatin, followed by pembrolizumab and pemetrexed. Approximately 172 adult participants with NSCLC will be enrolled in the study in 132 sites worldwide. In the dose escalation stage participants will be treated with increasing intravenous (IV) doses of Telisotuzumab Adizutecan in combination with budigalimab until the dose of Telisotuzumab Adizutecan reached is tolerable and expected to be efficacious. In the dose optimization stage participants will be receive IV optimized doses of Telisotuzumab Adizutecan in combination with budigalimab or receive IV budigalimab, pemetrexed, and investigator's choice of carboplatin or cisplatin, followed by budigalimab and pemetrexed, or IV SOC pembrolizumab, pemetrexed, and investigator's choice of carboplatin or cisplatin, followed by pembrolizumab and pemetrexed. The study will run for a duration of approximately 33 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at an approved institution (hospital or clinic). The effect of the treatment will be frequently checked by medical assessments, blood tests, questionnaires and side effects.172 Participants Needed

BSB-1001 for Blood CancersColumbus, Ohio

The goal of this clinical trial is to test BSB-1001 which is a new type of cellular therapy to treat blood cancers (AML, ALL and MDS). It will evaluate the safety of BSB-1001 and also determine whether it works to prevent relapse of your cancer.38 Participants Needed

AI-081 Antibody for Advanced CancerColumbus, Ohio

BIPAVE-001 is a Phase 1-2 study for evaluating the safety, pharmacokinetics (PK), and efficacy of AI-081 in solid tumors.204 Participants Needed

SAR443579 + Other Agents for Blood CancersColumbus, Ohio

This is a parallel, Phase 1/Phase 2, randomized, open label, multi-cohort, multi-center study assessing the safety, tolerability and preliminary efficacy of SAR443579 with different agents for treatment in adolescent and/or adult participants with CD123 expressing hematological malignancies. This protocol is structured as a master protocol (containing common protocol elements). Individual sub-studies will explore SAR443579 with combination partners, which may include approved or investigational agents. Experimental sub-studies will be tested through 3 parts: Part 1: dose finding (such as dose escalation/ safety run-in). Part 2: dose optimization (when applicable). Part 3: dose expansion. In each sub-study, a dose escalation will identify preliminary recommended dose for expansion (pRDE) of SAR443579 and its respective combination partner. Following the determination of the preliminary RDE, additional participants will be enrolled in the dose expansion part, or if dose optimization needs to be further evaluated, additional participants will be enrolled in the "dose optimization/expansion" part. Dose optimization and dose expansion part could involve randomization depending on specific sub-study design. Study will consist of a screening period, treatment period, and follow-up period. Participants will receive study treatment until documented disease progression, unacceptable adverse events, participant's decision to stop study treatment, or completion of the maximum cycles allowed in the sub-studies, or the participant meets other criteria for discontinuation per study protocol (whichever occurs first).18 Participants Needed

Linvoseltamab for AmyloidosisColumbus, Ohio

This study is researching an experimental drug called linvoseltamab ("study drug"). This study is focused on patients who have AL amyloidosis that has returned or have failed other therapies and need to be treated again. The study consists of 2 phases (Phase 1 and Phase 2): * In Phase 1, linvoseltamab will be given to a small number of participants to study the side effects of the study drug and to determine the recommended doses of the study drug to be given to participants in Phase 2. * In Phase 2, linvoseltamab will be given to more participants to continue to assess the side effects of the study drug and to evaluate the ability of linvoseltamab to treat AL amyloidosis. The study is looking at several other research questions, including: * How many participants treated with linvoseltamab have improvement in the abnormal proteins that cause organ problems and for how long * How many participants treated with linvoseltamab have improvement in the heart or kidney and for how long * What the right dosing regimen is for linvoseltamab * What side effects may happen from taking linvoseltamab * How much linvoseltamab is in the blood at different times * Whether the body makes antibodies against linvoseltamab (which could make the drug less effective or could lead to side effects)220 Participants Needed

AZD9829 for Blood CancersColumbus, Ohio

This is a modular, multicentre, open-label, Phase I/II, dose-setting study. AZD9829 will be administered intravenously as monotherapy or in combination in participants with CD123 positive hematological malignancies.104 Participants Needed

Immunotherapy Combinations for Lung CancerColumbus, Ohio

This is a Phase 1b/2 study evaluating the anti-PD1 antibody, cemiplimab, in combination with either S095018 (anti-TIM3 antibody), S095024 (anti-CD73 antibody), or S095029 (anti-NKG2A antibody) in adult participants with previously untreated advanced/metastatic non-small cell lung cancer (NSCLC) with high PD-L1 expression. The study includes two parts: part A, the combination-therapy safety lead-in phase to determine the recommended dose for expansion (RDE) for S095018, S095024, and S095029 in combination with cemiplimab and part B, the randomized dose expansion phase to assess the efficacy of S095018, S095024, or S095029 in combination with cemiplimab. Study treatment will be administered for a maximum of 108 weeks, or until confirmed disease progression per iRECIST and/ or until meeting other treatment discontinuation criteria.176 Participants Needed

AZD5851 for Liver CancerColumbus, Ohio

This trial is testing AZD5851, a new drug, in adults with advanced liver cancer that has a specific marker (GPC3) and hasn't responded to other treatments. The drug aims to target and kill these specific cancer cells.94 Participants Needed

Felzartamab for Lupus NephritisColumbus, Ohio

In this study, researchers will learn more about the use of felzartamab in people with active lupus nephritis, also known as LN. In people with LN, antibodies build up in the glomeruli of the kidneys. Antibodies are proteins in the blood used by the immune system to fight infection. Glomeruli are small filters that remove waste and extra fluid from the blood. This buildup leads to inflammation and damage to the kidneys. Kidney damage can lead to too much protein and blood leaking into the urine. High levels of protein in the urine, called proteinuria, are common in people with LN. Symptoms of LN can include fever, swelling in the legs and body, and high blood pressure. If left untreated, LN can eventually lead to kidney failure. In this study, researchers will learn more about how a study drug called felzartamab affects people with LN. Felzartamab is a monoclonal antibody, which means it is an antibody made in a laboratory. Felzartamab can target immune cells that produce antibodies, helping to lower their buildup in the kidneys. The main goal of this study is to learn more about the safety of felzartamab and how it works in the body of people with LN who are taking standard of care. This will help researchers decide if they should do more studies with felzartamab in people with LN. Standard of care is the usual treatment or care given to patients for a disease, as prescribed by their doctor. The main question researchers want to answer in this study are: • How many participants had adverse events during the study? An adverse event is a health problem that may or may not be caused by the study drug. It can happen during a clinical study or within a certain amount of time after the study has ended. Researchers will also learn more about: * How much felzartamab affects proteinuria and the level of creatinine in the urine. Creatinine is a protein that is released into the blood from normal muscle wear and tear. Its levels can help doctors understand how well your kidneys are working. * How many participants have a complete response. A complete response means that their urine protein levels decrease to a low level, and their kidney function stays stable. * How many participants have a 50% decrease in the level of protein and creatinine in their urine. * How much felzartamab affects the participants' lupus-related blood tests. * How the body processes felzartamab. * How many participants develop antibodies against felzartamab in the blood. This study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 42 days. * Throughout the study, all participants will continue taking their standard of care, as prescribed by their doctor. * There are 2 parts in this study. In both parts, participants will receive felzartamab through an intravenous infusion, also known as an IV. This means it is being given into a vein. * In Part 1, participants will have up to 14 visits to their study research center. In Part 2, participants may have up to 15 visits. * Each participant will be in the study for about 2 years.20 Participants Needed

Gemcitabine + NK Cells +/- Naxitamab for Breast CancerColumbus, Ohio

This phase Ib/II trial tests the safety, best dose and how well gemcitabine and ex vivo expanded allogenic universal donor TGFBi NK cells with or without naxitamab work for the treatment of patients with GD2 expressing, HER2 negative breast cancer that has spread from where it first started (primary site) to other places in the body (metastatic). Gemcitabine is a chemotherapy drug that blocks the cells from making deoxyribonucleic acid (DNA) and may kill cancer cells. TGFBi NK cells are manufactured cells that are a part of your natural immunity. NK cells can recognize missing or incorrect proteins on tumor cells and then eliminate these tumor cells and TGFBi NK cells are created to be able to better kill the tumor cells. Naxitamab is a monoclonal antibody that targets GD2, which is a protein or sugar present on tumor cells but not very commonly found on normal cells. This antibody helps draw the attention of the immune system to the tumor cells that have GD2 to help attack the tumor cells. Giving gemcitabine and TGFBi NK cells with or without naxitamab may kill more tumor cells in patients with metastatic GD2 expressing, HER2 negative breast cancer.42 Participants Needed

CAR T-Cell Therapy for Lupus NephritisColumbus, Ohio

This trial is testing a new treatment that uses modified immune cells to target and destroy harmful cells in adults with severe kidney inflammation from lupus that doesn't respond to usual treatments.32 Participants Needed

AZD5335 for CancerColumbus, Ohio

This trial is testing a new treatment called AZD5335, which combines an antibody with a cancer-killing drug. It aims to see if this treatment is safe and effective for patients with advanced solid tumors. The antibody helps deliver the drug directly to the cancer cells, potentially reducing harm to healthy cells.396 Participants Needed

STX-478 + Fulvestrant for Advanced Breast CancerColumbus, Ohio

This trial is testing a new drug called STX-478 to see if it can help treat advanced solid tumors and breast cancer. The study will look at how safe the drug is, how it behaves in the body, and if it can stop or slow down cancer growth. Some patients will receive just STX-478, while others will get it combined with another drug called fulvestrant, which is often used to treat advanced breast cancer.720 Participants Needed

AOC 1020 for Facioscapulohumeral Muscular DystrophyColumbus, Ohio

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)90 Participants Needed

Lurbinectedin for Ewing SarcomaColumbus, Ohio

This trial is testing a medication called lurbinectedin in children and young adults who have already been treated for solid tumors or have a type of cancer called Ewing sarcoma that has come back or didn't respond to other treatments. The medication aims to stop cancer cells from growing and spreading. The study will check if the treatment is safe and effective.60 Participants Needed

ONC-392 + Lutetium for Prostate CancerColumbus, Ohio

In this Phase 2 study, mCRPC patients with PSMA positive scans who progressed on prior ARTA and up to 2 lines of taxanes, and are naïve to lutetium Lu 177 vipivotide tetraxetan, will be enrolled. The study is open-label, randomized with active control, multi-center study.141 Participants Needed

STAR0602 for Advanced CancersColumbus, Ohio

This trial is testing a new drug called STAR0602 to see if it is safe and effective for treating advanced cancers with specific markers. The drug is given through an IV and aims to find and destroy cancer cells by recognizing these markers. The study will first determine the best dose and then see how well the drug works in patients.365 Participants Needed

Triple-Drug Therapy for Acute Myeloid LeukemiaColumbus, Ohio

People with acute myeloid leukemia (AML) are usually treated with chemotherapy. Some people with AML have a changed FLT3 gene which causes leukemia cells to grow faster. Therefore, chemotherapy is less suitable to treat AML in people with the changed FLT3 gene. Gilteritinib, given with venetoclax and azacitidine, is a potential new treatment for people with AML with the changed FLT3 gene. They cannot have chemotherapy due to old age or other conditions. Before these combined 3 medicines are available as a treatment, the researchers need to understand how they are processed by and act upon the body when given together. In this study, they do this to find a suitable dose for venetoclax and to check for potential medical problems from the treatment. In this study, people newly diagnosed with AML who have the changed FLT3 gene and cannot have chemotherapy can take part. The main aims of this study are: to find suitable doses of gilteritinib, venetoclax and azacitidine as a combined treatment; to learn how they are processed by and act upon the body; to learn the remission rate; to check for medical problems during this treatment. In the study, people will visit the study clinic many times. The first visit is to check if they can take part. People will be asked about their medical history, have a medical examination, and have their vital signs checked. Also, they will have an ECG to check their heart rhythm and have some blood and urine samples taken for laboratory tests. They will have a chest X-ray and a bone marrow sample will be taken. The changed FLT3 gene will be confirmed, either by the bone marrow or a blood sample. This study will be in 2 phases. In Phase 1, different small groups of people will take venetoclax tablets containing lower to higher doses in the combined treatment. The doses of gilteritinib and azacytidine will be unchanged. This is done to find a suitable dose of venetoclax to use in phase 2 of the study. People will take tablets of gilteritinib and venetoclax once a day on a 28-day cycle. They will be given azacytidine as an infusion or an injection just under the skin. This will be for 7 days at the beginning of each 28-day cycle. They will continue cycles of treatment throughout this phase of the study. In Phase 2, more people newly diagnosed with AML with the changed FLT3 gene will take part. They will be treated with the suitable doses of the combined treatment worked out from Phase 1. Treatment will be on a 28-day cycle. People will continue on cycles of treatment throughout this phase of the study. Researchers will work out the remission rate from this phase of the study. In each phase of the study, people can continue with up to 12 cycles of treatment if they can manage any medical problems. People will visit the study clinic many times during their first treatment cycle, and less often during the next cycles. During these visits, medical problems will be recorded and some blood samples will be taken for laboratory tests. On some visits, people will also have their vital signs checked. Bone marrow samples will be taken during cycle 1, and at the beginning of cycle 3. More samples will be taken during the study from people who are not in remission. When people have finished treatment, those who have responded well to treatment and are in remission will be invited to continue with up to 24 more cycles of gilteritinib plus azacitidine. All people taking part in the study will visit the study clinic for an end-of-treatment visit. During this visit, medical problems will be recorded and some blood samples will be taken for laboratory tests. People will have a medical examination, an ECG, and will have their vital signs checked. Also, a bone marrow sample will be taken. There will be a follow-up visit 30 days later to check for medical problems. Then people will visit the clinic or get a phone call every 3 months for up to 3 years. This is to give an update on their current treatment for AML. Some people can have a stem cell transplant during the study if they meet certain study rules. They will pause their study treatment during the stem cell transplant process and continue study treatment afterwards.70 Participants Needed

DR-01 for Leukemia and LymphomaColumbus, Ohio

This trial is testing a new drug called DR-01 to see if it is safe and effective for adults with specific blood cancers. The study will check if the drug can safely reach and affect cancer cells to stop their growth.69 Participants Needed

NVL-655 for NSCLCColumbus, Ohio

This trial is testing a new drug called NVL-655 to see if it is safe and effective for patients with advanced ALK-positive lung cancer and other solid tumors. The drug works by blocking a protein that helps cancer cells grow. The study focuses on patients who have specific genetic changes and may not respond well to standard treatments. Newer treatments have been shown to improve outcomes in ALK-translocated lung cancer compared to older treatments.470 Participants Needed

CFI-402257 + Fulvestrant for Breast CancerColumbus, Ohio

This trial tests a new drug, CFI-402257, alone and with Fulvestrant in patients with advanced solid tumors and advanced breast cancer. It aims to determine if the drug is safe and effective by studying its effects on the body and cancer cells. Fulvestrant is an established treatment for hormone receptor-positive advanced breast cancer, often used in combination with other therapies to improve outcomes.44 Participants Needed

Intravitreal Cerliponase Alfa for Batten DiseaseColumbus, Ohio

This trial involves injecting a medication called cerliponase alfa directly into the eyes of 5 subjects. The goal is to see if this treatment is safe and effective over a period of time. The subjects will then be monitored periodically for an extended duration to collect more data.5 Participants Needed

Gene Therapy for Spinal Muscular AtrophyColumbus, Ohio

This trial uses a harmless virus to deliver a healthy gene directly into the spinal fluid of patients with diseases caused by IGHMBP2 gene defects. The virus helps correct the genetic issues by bringing the healthy gene to the affected cells. This approach has been shown to improve conditions in similar diseases in animals.10 Participants Needed

REGN4336 + Cemiplimab for Prostate CancerColumbus, Ohio

This trial is testing a new cancer drug called REGN4336, alone or with other drugs, to see if it is safe and effective. It aims to help the immune system attack cancer cells. The study focuses on finding the right dose and understanding side effects.370 Participants Needed

NVL-520 for ROS1-Positive Solid TumorsColumbus, Ohio

This trial tests NVL-520, a new drug, for safety and effectiveness in patients with advanced ROS1-positive cancers. It targets those who haven't responded well to other treatments by blocking a protein that helps cancer cells grow. NVL-520 is being tested for its effectiveness in treating advanced ROS1-positive cancers, a targetable mutation in non-small cell lung cancer (NSCLC) that has seen various treatments like crizotinib and entrectinib.359 Participants Needed

SAR443579 for LeukemiaColumbus, Ohio

This trial is testing a new drug called SAR443579 to see if it is safe and effective for treating blood cancers. The study will look at how the drug moves through and affects the body, and whether it can help fight cancer. Patients with various types of blood cancers are participating in this study.169 Participants Needed

Droxidopa for Dysautonomia in Menkes DiseaseColumbus, Ohio

The purpose of this study is to evaluate whether Northera (Droxidopa) is safe and effective in young adults with Menkes disease who survived the most severe complications of their illness or adults with occipital horn syndrome (OHS), who have trouble with intermittent low blood pressure and other symptoms of dysautonomia. The outcomes and information from this study may help adult survivors of Menkes disease and individuals with OHS lead more normal day-to-day lives.6 Participants Needed

BGB-11417 + Dexamethasone + Carfilzomib for Multiple MyelomaColumbus, Ohio

The purpose of this study is to assess the safety, tolerability, and efficacy of sonrotoclax as monotherapy and in various combinations in patients with relapsed/refractory (R/R) multiple myeloma (MM) and chromosomal translocation t(11;14). The study investigates sonrotoclax alone and in combination with dexamethasone and other agents, including carfilzomib, daratumumab, and pomalidomide.246 Participants Needed

EDIT-301 for Sickle Cell DiseaseColumbus, Ohio

This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.45 Participants Needed

Avapritinib for Pediatric Solid CancersColumbus, Ohio

This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to \< 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).37 Participants Needed

Pembrolizumab Combinations for MelanomaColumbus, Ohio

Substudy 02D is part of a larger research study that is testing experimental treatments for melanoma, a type of skin cancer. The larger study is the umbrella study. The goal of substudy 02D is to evaluate the safety and efficacy of investigational treatment arms in programmed cell-death 1 (PD-1) naïve or PD-1 exposed participants with melanoma brain metastasis (MBM) and to identify the investigational agent(s) that, when used in combination, are superior to the current treatment options/historical control available. As of amendment 2 (effective 01DEC2022) enrollment into the treatment arm of pembrolizumab and lenvatinib has been discontinued.300 Participants Needed

ASTX727 + Venetoclax for Acute Myeloid LeukemiaColumbus, Ohio

The Phase 1 portion of this study is a single-arm, open-label, multicenter, non-randomized interventional study to evaluate the pharmacokinetic (PK) interaction, safety, and efficacy of ASTX727 when given in combination with venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. The primary purpose of the study is to rule out drug-drug interactions between ASTX727 and venetoclax combination therapy by evaluating area under the curve (AUC) and maximum plasma concentration (Cmax) exposure. The Phase 2 portion of the study is to assess the efficacy of ASTX727 and venetoclax when given in combination and to evaluate potential PK interactions. Phase 2 will follow the same overall study design as Phase 1 and has two parts, Part A and Part B.101 Participants Needed

REGN7075 + Cemiplimab for Advanced CancerColumbus, Ohio

This trial is testing a new drug called REGN7075, alone or with other treatments, on patients with advanced solid tumors. The goal is to see if it can stop tumor growth and shrink the tumors.933 Participants Needed

Epcoritamab for Chronic Lymphocytic LeukemiaColumbus, Ohio

The study is a global, multi-center safety and efficacy trial of epcoritamab, an antibody also known as EPKINLY™ and GEN3013 (DuoBody®-CD3xCD20). Epcoritamab will be tested either in Relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) as: * Monotherapy, or * Combination therapy: * epcoritamab + venetoclax * epcoritamab + pirtobrutinib Treatment-naïve (TN) high risk (HR) (CLL): • epcoritamab + pirtobrutinib Combination therapy for Richter's Syndrome (RS): * epcoritamab + lenalidomide * epcoritamab + R-CHOP (i.e., rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine \[Oncovin®\] and prednisone). The study includes participants with R/R or TN HR CLL/small lymphocytic lymphoma (SLL) and participants with RS. The trial consists of two parts, a dose-escalation phase (phase Ib) and an expansion phase (phase II). Participants with RS are only included in the expansion phase. Epcoritamab will be injected subcutaneously (under the skin). Standard-of-care and combination treatments (venetoclax, pirtobrutinib, lenalidomide, and R-CHOP) will be given either orally (by mouth) or intravenously (in a vein). Study details include: * Study duration will be up to 5 years after the last participant's first treatment in the trial. * The treatment duration for each participant will be between 12 months (1 year) and 24 months (2 years), depending upon the treatment arm assigned. * The visit frequency will be either weekly, every other week, or monthly, depending upon the part of the study. All participants will receive active drug; no one will be given placebo.424 Participants Needed

REGN5668 + Cemiplimab/REGN4018 for Ovarian CancerColumbus, Ohio

This study is researching an investigational drug called REGN5668. Participants will receive additional investigational drugs in combination with REGN5668. These additional drugs include cemiplimab or REGN4018 (with or without sarilumab). The main purposes of this study are to: * Learn about the safety and profile of any side effects from the study drugs and to determine the highest, safe dose that can be given to participants with ovarian cancer or cancer of the uterus * Look for signs that the study drugs can treat ovarian cancer or cancer of the uterus This study has 2 parts. The purpose of Part 1 (Escalation) to find the highest, safe dose of the study drug(s). The purpose of Part 2 (Expansion) is to use the doses chosen in Part 1. Participants with cancer of the uterus will only participate in Part 2. The study is looking at several other research questions, including: * Side effects that may be experienced by participants taking REGN5668 alone and/or in combination with cemiplimab or REGN4018 * How REGN5668 works in the body either alone and/or in combination with cemiplimab or REGN4018 * How much of the study drugs (REGN5668, cemiplimab, REGN4018) are in the blood * To see if REGN5668 in combination with cemiplimab or REGN4018 works to treat cancer * To find out how safe, tolerable, and effective in mitigating Cytokine Release Syndrome (CRS) sarilumab pretreatment is when given before REGN4018612 Participants Needed

Zifibancimig for Age-Related Macular DegenerationColumbus, Ohio

This trial is testing a new treatment called zifibancimig for people with a serious eye condition known as neovascular age-related macular degeneration (nAMD). The treatment is given either as an injection into the eye or through a small implant. It works by stopping harmful blood vessels from growing in the eye, which can help protect or improve vision.251 Participants Needed

T-DXd Combinations for Breast CancerColumbus, Ohio

DESTINY-Breast07 will investigate the safety, tolerability, and anti-tumour activity of trastuzumab deruxtecan (T-DXd) in combination with other anti-cancer agents in patients with HER2-positive Metastatic Breast Cancer245 Participants Needed

AGN-193408 SR for GlaucomaColumbus, Ohio

This trial is testing a new medication called AGN-193408 SR to see if it can safely lower eye pressure in people with open-angle glaucoma or ocular hypertension.96 Participants Needed

CT041 CAR-T Therapy for Stomach and Pancreatic CancerColumbus, Ohio

A Phase 1b/2, open label, multi-center, clinical study of Chimeric Antigen Receptor T Cells (CAR-T) targeting claudin18.2 in patients with advanced gastric, pancreatic or other specified digestive system cancers110 Participants Needed

RP1 for Advanced Skin Cancer in Transplant PatientsColumbus, Ohio

This trial tests RP1, a modified virus, in organ transplant recipients with advanced skin cancer. RP1 works by killing cancer cells and boosting the immune system to fight the cancer. The HIV-1 accessory protein Vpr has been shown to induce cell cycle arrest and kill tumor cells by apoptosis.65 Participants Needed

Pembrolizumab Combinations for MelanomaColumbus, Ohio

Substudy 02B is part of a larger research study where researchers are looking for new ways to treat advanced melanoma that has not been treated before. The larger study is the umbrella study. Researchers want to know if adding other treatments to pembrolizumab can treat advanced melanoma. The goals of this study are to learn: * About the safety and how well people tolerate pembrolizumab given with other treatments * How many people have melanoma that responds (gets smaller or goes away) to treatment Arm 1: Pembrolizumab + Vibostolimab was added in the base protocol on 13-Nov-2019, and enrollment into this arm has been completed. Arm 2: Pembrolizumab was added in the base protocol on 13-Nov-2019, and enrollment stopped prematurely on 15-Aug-2022. Arm 3: Coformulation Pembrolizumab/Quavonlimab was added in Amendment 01 on 20-Oct-2020, and enrollment stopped prematurely on 15-Aug-2022. Arm 4: Coformulation Pembrolizumab/Quavonlimab + Lenvatinib was added in Amendment 01 on 20-Oct-2020, and enrollment is ongoing. Arm 5: Coformulation Favezelimab/Pembrolizumab was added in Amendment 03 on 01-DEC-2022 and has paused enrollment, Arm 6: Coformulation Favezelimab/Pembrolizumab + All-trans Retinoic Acid (ATRA) was added in Amendment 03 on 01-DEC-2022 and has paused enrollment, and Arm 7: Coformulation Favezelimab/Pembrolizumab + Vibostolimab was added in Amendment 03 on 01-DEC-2022 and enrollment was stopped prematurely on 22-SEP-2023.315 Participants Needed

Pembrolizumab Combo for MelanomaColumbus, Ohio

Substudy 02A is part of a larger research study that is testing experimental treatments for melanoma, a type of skin cancer. The larger study is the umbrella study. The goal of substudy 02A is to evaluate the safety and efficacy of investigational treatment arms in participants with PD-1 refractory melanoma to identify the investigational agent(s) that, when used in combination, are superior to the current treatment options/historical control available. As of Amendment 4 (effective date: 05JAN2022), a third arm has been opened to participant enrollment, treatment with pembrolizumab and all-trans retinoic acid (ATRA). Enrollment into the first two arms, treatment with pembrolizumab + quavonlimab+ vibostolimab and treatment with pembrolizumab + quavonlimab + lenvatinib has been completed per protocol as of September 2021.200 Participants Needed

Pembrolizumab + Other Agents for MelanomaColumbus, Ohio

Substudy 02C is part of a larger research study that is testing experimental treatments for melanoma, a type of skin cancer. The larger study is the umbrella study. The goal of substudy 02C is to evaluate the safety and efficacy of investigational treatment arms in participants with Stage III melanoma who are candidates for neoadjuvant therapy to identify the investigational agent(s) that, when used in combination, are superior to the current treatment options/historical control available. Arm 1: Pembrolizumab + Vibostolimab, Arm 2: Pembrolizumab + Gebasaxturev, and Arm 3: Pembrolizumab were added in the base protocol on 13-Nov-2019, and enrollment into those arms has been completed. Arm 4: Pembrolizumab + MK-4830 was added in Amendment 04 on 20-Dec-2021, and enrollment into that arm has been completed. Arm 5: Favezelimab + Pembrolizumab and Arm 6: Pembrolizumab + all-trans retinoic acid (ATRA) were added in Amendment 06 on 25-Jun-2022, and enrollment is ongoing.90 Participants Needed

Emavusertib (+ Venetoclax) for Acute Myeloid LeukemiaColumbus, Ohio

This is a multicenter, open-label, Phase 1/2a dose escalation and expansion study of orally administered emavusertib (CA-4948) monotherapy in adult patients with AML or higher- risk Myelodysplastic Syndrome (hrMDS). Patients enrolling in the Phase 1 dose escalation of the study must meet one of the following criteria prior to consenting to the study: * Relapse/refractory (R/R) AML with FMS-like tyrosine kinase-3 (FLT3) mutations who have been previously treated with a FLT3 inhibitor * R/R AML with spliceosome mutations of splicing factor 3B subunit 1 (SF3B1) or U2AF1 * R/R hrMDS with spliceosome mutations of SF3B1 or U2 small nuclear RNA auxiliary factor 1 (U2AF1) * Number of pretreatments: 1 or 2 The Phase 2a Dose Expansion will be in 3 Cohorts of patients: 1. R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor; 2. R/R AML with spliceosome mutations of SF3B1 or U2AF1; and 3. R/R hrMDS (Revised International Prognostic Scoring System \[IPSS-R\] score \> 3.5) with spliceosome mutations of SF3B1 or U2AF1. All patients above have had ≤ 2 lines of prior systemic anticancer treatment. In previous versions of this protocol there was a Phase 1b portion of the study, in which patients with AML or hrMDS received CA-4948 in combination with venetoclax. This part of the study is no longer open for enrollment.366 Participants Needed

Gene Therapy for Duchenne Muscular DystrophyColumbus, Ohio

This trial uses a special virus to deliver genetic material to boys with Duchenne muscular dystrophy who have a specific gene issue. The treatment helps their bodies make a better version of an important muscle protein.3 Participants Needed

Pembrolizumab + Chemotherapy for Non-Small Cell Lung CancerColumbus, Ohio

The purpose of this study is to assess the efficacy and safety of pembrolizumab (MK-3475) with or without chemotherapy in combination with vibostolimab (MK-7684), boserolimab (MK-5890), MK-4830, MK-0482, I-DXd, or HER3-DXd in treatment-naïve participants with advanced squamous or non-squamous NSCLC. This study is one of the pembrolizumab substudies being conducted under one pembrolizumab umbrella master protocol (MK-3475-U01/KEYMAKER-U01).450 Participants Needed

Ramucirumab for Desmoplastic Small Round Cell TumorColumbus, Ohio

This study is being conducted to test the safety and efficacy of ramucirumab in combination with other chemotherapy in the treatment of relapsed, recurrent, or refractory desmoplastic small round cell tumor (DSRCT) in children and young adults. This trial is part of the CAMPFIRE master protocol (NCT05999994) which is a platform to accelerate the development of new treatments for pediatric and young adult participants with cancer. Your participation in this trial could last 12 months or longer, depending on how you and your tumor respond.34 Participants Needed

DF1001 for Solid CancersColumbus, Ohio

This trial is testing a new treatment called DF1001-001 that helps the immune system target and destroy cancer cells. It focuses on patients with certain types of cancers that have a protein called HER2. The treatment works by activating immune cells to recognize and attack these cancer cells.378 Participants Needed

ONC-392 + Pembrolizumab for Non-Small Cell Lung CancerColumbus, Ohio

This trial tests ONC-392, an antibody that helps the immune system fight cancer, in patients with advanced or spreading tumors who haven't responded to other treatments. It works by blocking a protein that usually keeps immune responses in check, making it easier for the body to attack cancer cells.733 Participants Needed

CC-92480 + Standard Treatments for Multiple MyelomaColumbus, Ohio

This trial is testing a new drug called CC-92480 to see if it can help patients who are already getting standard treatments. The goal is to find out if adding this new drug can make their treatment work better.424 Participants Needed

Ixazomib + Chemotherapy for LeukemiaColumbus, Ohio

This is a phase 1/2 study of a drug called Ixazomib in combination with cytotoxic chemotherapy consisting of Vincristine, Dexamethasone, Asparaginase, and Doxorubicin (VXLD).24 Participants Needed

MRTX849 for CancerColumbus, Ohio

This trial is testing a new pill called MRTX849 (adagrasib) for patients with advanced cancers that have a specific genetic change called KRAS G12C. The pill aims to block this change and stop the cancer from growing. Adagrasib is similar to another approved treatment and is intended for patients with this genetic change who have already received other treatments.822 Participants Needed

REGN5458 for Multiple MyelomaColumbus, Ohio

The main purpose of this study is to learn about the safety of REGN5458 and to find out what is the best dose of REGN5458 to give to patients with multiple myeloma. An additional purpose is to look for any signs that REGN5458 can treat cancer. The study is looking at several other research questions, including: * Side effects that may be experienced by people receiving REGN5458 * How REGN5458 works in the body * How much REGN5458 is present in the blood * How REGN5458 may work to treat cancer387 Participants Needed

Oral LOXO-305 for Chronic Lymphocytic LeukemiaColumbus, Ohio

This trial is testing pirtobrutinib, a tablet taken by mouth, in patients with certain types of blood cancers who haven't responded to or can't tolerate usual treatments. The medication works by blocking a protein that cancer cells need to grow. The study aims to find the best dose and see how well it works.860 Participants Needed

Gene Therapy for Limb-Girdle Muscular DystrophyColumbus, Ohio

This trial tests SRP-9003, a gene therapy, in patients with LGMD2E, a type of muscular dystrophy. The therapy uses a harmless virus to deliver a healthy gene into muscle cells to fix the genetic issue. SRP-9003 is a gene therapy that uses a harmless virus to deliver a healthy gene into muscle cells, a method previously demonstrated to be effective in treating muscular dystrophies.6 Participants Needed

Rucaparib + Pembrolizumab for Lung CancerColumbus, Ohio

This study is a multi-center, Phase I/II, single arm trial to assess the safety and efficacy of the combination of oral rucaparib plus intravenous pembrolizumab as maintenance therapy in patients with stage IV non-squamous non-small cell lung cancer (NSCLC) without progressive disease (PD), as confirmed on CT scans, after induction therapy with carboplatin/pemetrexed/pembrolizumab (CPP) triplet therapy.25 Participants Needed

Cetuximab + Nivolumab for Head and Neck CancerColumbus, Ohio

The purpose of this study is to find out if the combination of two established anti-cancer therapies are beneficial in participants with Head and Neck Squamous Cell Carcinoma (HNSCC). Specifically, investigators want to determine if the combination of Cetuximab and nivolumab can help people with advanced cases of HNSCC. Both cetuximab and nivolumab have been used separately to treat HNSCC and are Food and Drug Administration (FDA) approved in this type of cancer.95 Participants Needed

JCAR017 for Chronic Leukemia or LymphomaColumbus, Ohio

This trial tests JCAR017, a therapy using modified immune cells, in adults with hard-to-treat CLL or SLL. It aims to see if these enhanced immune cells can better fight the cancer.320 Participants Needed

Nemtabrutinib for Blood CancersColumbus, Ohio

This trial is testing a new pill called nemtabrutinib for patients with blood cancers that have returned or didn't respond to other treatments. The goal is to find a safe and effective dose of the drug. Nemtabrutinib is a new drug being tested for blood cancers, designed to reduce adverse effects.190 Participants Needed

Selpercatinib for Solid TumorsColumbus, Ohio

This trial is testing a new oral drug called selpercatinib in patients with advanced cancers that have specific gene changes. The drug aims to block a gene that helps cancer grow, potentially slowing or stopping the disease.857 Participants Needed

Repotrectinib for Solid TumorsColumbus, Ohio

Phase 1 dose escalation will determine the first cycle dose-limiting toxicities (DLTs), the maximum tolerated dose (MTD), the biologically effective dose and recommended Phase 2 dose (RP2D) of repotrectinib given to adult subjects with advanced solid malignancies harboring an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. Midazolam DDI substudy will examine effect of of repotrectinib on CYP3A induction. Phase 2 will determine the confirmed Overall Response Rate (ORR) as assessed by Blinded Independent Central Review (BICR) of repotrectinib in each subject population expansion cohort of advanced solid tumors that harbor a ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. The secondary objective will include the duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS) and clinical benefit rate (CBR) of repotrectinib in each expansion cohort of advanced solid tumors that harbor a ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement.500 Participants Needed

Targeted Therapies for Acute Myeloid LeukemiaColumbus, Ohio

This screening and multi-sub-study Phase 1b/2 trial will establish a method for genomic screening followed by assigning and accruing simultaneously to a multi-study "Master Protocol (BAML-16-001-M1)." The specific subtype of acute myeloid leukemia will determine which sub-study, within this protocol, a participant will be assigned to evaluate investigational therapies or combinations with the ultimate goal of advancing new targeted therapies for approval. The study also includes a marker negative sub-study which will include all screened patients not eligible for any of the biomarker-driven sub-studies.2000 Participants Needed

Atezolizumab + Cisplatin + Gemcitabine for Bladder CancerColumbus, Ohio

The purpose of this study is to test the safety of the study drug, atezolizumab, when combined with the standard chemotherapy drugs, gemcitabine and cisplatin (or GC). This study will help researchers begin to understand whether combining GC with atezolizumab is better, the same, or worse than the usual approach of using GC alone.54 Participants Needed

TL-895 + Navtemadlin for Chronic Lymphocytic LeukemiaColumbus, Ohio

The purpose of this research study is to determine the safety and tolerability of TL-895. There are 2 parts of this study. Part 1 tested increasing doses of TL-895 to identify the recommended safe dose for participants with relapsed/refractory (R/R) B cell malignancies who failed at least 1 but no more than 3 prior therapies. Part 1 of this study is no longer enrolling participants. Arms 1 \& 2 of Part 2 of this study will test different doses of TL-895 in participants with R/R CLL or SLL who have failed at least 1 prior therapy. Arms 1 \& 2 of Part 2 of this study is randomized (like the flip of a coin) to receive a specific treatment dose. If someone participates in arms 1 or 2 of Part 2, the dose they receive will be either 100mg twice a day or 150mg twice a day. Arms 3 and 4 of Part 2 of this study will test the 150mg and 100mg BID dose of TL-895, respectively in treatment naïve participants with CLL/SLL. Arms 5 and 6 of Part 2 will test 150mg TL-895 BID in combination with 240 mg navtemadlin QD in participants with relapsed/refractory and treatment naïve without 17p(del). Arm 7 will test 150mg TL-895 in combination with 240 mg navtemadlin QD in participants with relapsed/refractory CLL/SLL with 17p(del). Every participant in this study will receive TL-895.130 Participants Needed

CC-220 Combination Therapy for Multiple MyelomaColumbus, Ohio

This trial tests a new drug, CC-220, alone and with other drugs for patients with multiple myeloma who haven't responded to other treatments or are newly diagnosed. The drugs work by controlling cancer cell growth and killing cancer cells through different mechanisms.466 Participants Needed

Ruxolitinib + Chemotherapy for Ovarian CancerColumbus, Ohio

This phase I/II trial studies the side effects and the best dose of ruxolitinib phosphate when given together with paclitaxel and carboplatin and to see how well they work in treating patients with stage III-IV epithelial ovarian, fallopian tube, or primary peritoneal cancer. Ruxolitinib phosphate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as paclitaxel and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ruxolitinib phosphate together with paclitaxel and carboplatin may be a better treatment for epithelial ovarian, fallopian tube, or primary peritoneal cancer compared to paclitaxel and carboplatin alone.147 Participants Needed

Entrectinib for Solid TumorsColumbus, Ohio

This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain tumors harboring NTRK1/2/3 or ROS1 gene fusions, and extracranial solid tumors harboring NTRK1/2/3 or ROS1 gene fusions.69 Participants Needed

PARP Inhibitor + CTLA-4 Blockade for Ovarian CancerColumbus, Ohio

Of the approximately 21,000 cases of ovarian cancer diagnosed annually in the U.S, ten percent are attributed to hereditary syndromes, most commonly the result of mutations in the breast cancer susceptibility genes 1 or 2 (BRCA1 or BRCA2). Mutation in these genes results in the inability to repair double-stranded breaks in DNA. Treating these tumors with poly(adenosine diphosphate \[ADP\]-ribose) polymerase (PARP) inhibitors results in the specific killing of BRCA negative cells by blocking a second DNA-repair mechanism. Treatment of ovarian cancer patients with PARP inhibitors has resulted in improved progression free survival (PFS), but not overall survival (OS). It's not completely understood why this is the case, but some preclinical studies using ovarian cancer models in mice have suggested that combining PARP inhibitors with immune system modulators like T cell checkpoint inhibitors improves long-term survival. Therefore, the purpose of this study is to evaluate the safety and efficacy of a combination of a PARP inhibitor (Olaparib) with a T cell checkpoint inhibitor (the anti-CTLA-4 antibody Tremelimumab) in women with recurrent BRCA mutation-associated ovarian cancer.50 Participants Needed

Methoxyamine + Chemotherapy for MesotheliomaColumbus, Ohio

This phase I/II trial studies the side effects and the best dose of methoxyamine when given together with cisplatin and pemetrexed disodium and to see how well it works in treating patients with solid tumors or mesothelioma that have spread to other places in the body and usually cannot be cured or controlled with standard treatment (advanced), or mesothelioma that does not respond to pemetrexed disodium and cisplatin or carboplatin (refractory). Methoxyamine may shrink the tumor and may also help cisplatin and pemetrexed disodium work better by making tumor cells more sensitive to the drugs. Drugs used in chemotherapy, such as cisplatin and pemetrexed disodium, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving methoxyamine together with cisplatin and pemetrexed disodium may be a better treatment for solid tumors or mesothelioma than methoxyamine and pemetrexed disodium.30 Participants Needed

Acalabrutinib + Pembrolizumab for Blood CancersColumbus, Ohio

This study is evaluating the safety, pharmacodynamics (PD), and efficacy of acalabrutinib and pembrolizumab in hematologic malignancies.161 Participants Needed

Pembrolizumab for Pediatric CancerColumbus, Ohio

Researchers are looking for new ways to treat children with different types of melanoma (skin cancer), solid tumors, and lymphomas (blood cancers) that are any of these: * Advanced, which means cancer spread in the body or cannot be removed with surgery * Relapsed, which means cancer has come back after it had responded to previous treatment (responded means it stopped growing, gets smaller, or disappeared) * Refractory, which means cancer did not respond to previous treatment Pembrolizumab is an immunotherapy, which is a treatment that helps the immune system fight cancer. Researchers want to learn if different doses of pembrolizumab can cause at least 1 of the types of cancer to get smaller or go away. With Amendment 8, enrolment of participants with solid tumours and participants 6 months to under 12 years old with melanoma were closed. Enrolment of participants 12-18 years old with melanoma continues. Enrolment of participants who have tumours with specific traits (microsatellite-instability-high (MSI-H), and tumour-mutational burden-high ≥10 mutation/Mb (TMB-H)) also continues.370 Participants Needed

Acalabrutinib for Chronic Lymphocytic LeukemiaColumbus, Ohio

This study is evaluating the safety and efficacy of a new BTK inhibitor, acalabrutinib, for the treatment of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).306 Participants Needed

Pomalidomide + Dexamethasone +/- Ixazomib for Multiple MyelomaColumbus, Ohio

This randomized phase I/II trial studies the side effects and best dose of pomalidomide and ixazomib when given together with dexamethasone and to see how well pomalidomide and dexamethasone with or without ixazomib works in treating patients with multiple myeloma that has come back. Biological therapies, such as pomalidomide and dexamethasone, may stimulate the immune system in different ways and stop cancer cells from growing. Ixazomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It is not yet known whether pomalidomide and dexamethasone are more effective with or without ixazomib in treating multiple myeloma.118 Participants Needed

Elotuzumab + Triple Therapy for Multiple MyelomaColumbus, Ohio

This partially randomized phase I/II trial studies the side effects and best dose of elotuzumab and to see how well it works when given together with lenalidomide, bortezomib, and dexamethasone in treating patients with newly diagnosed multiple myeloma that is likely to recur (come back), or spread (high-risk). Lenalidomide and bortezomib may stop the growth of multiple myeloma by blocking blood flow to the tumor. Also, bortezomib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide and dexamethasone, also work in different ways to kill cancer cells, by stopping them from dividing, or by stopping them from spreading. Giving elotuzumab together with lenalidomide, bortezomib, and dexamethasone may be a better way to block cancer growth.142 Participants Needed

Cetuximab + Interleukin-12 for Head and Neck CancerColumbus, Ohio

This phase I/II trial studies the side effects and best dose of recombinant interleukin-12 when given together with cetuximab and to see how well they work in treating patients with squamous cell carcinoma of the head and neck that has come back, spread to another place in the body, or cannot be removed by surgery. Recombinant interleukin-12 may stimulate the white blood cells to kill tumor cells. Immunotherapy with monoclonal antibodies, such as cetuximab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread Giving recombinant interleukin-12 together with cetuximab may kill more tumor cells.23 Participants Needed

Pemetrexed + Cisplatin +/- Cediranib for MesotheliomaColumbus, Ohio

This randomized phase I/II trial is studying the side effects and best dose of cediranib maleate when given together with pemetrexed disodium and cisplatin and to see how well it works in treating patients with malignant pleural mesothelioma. Drugs used in chemotherapy, pemetrexed disodium and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Cediranib maleate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. Giving pemetrexed disodium and cisplatin together with cediranib maleate may kill more tumor cells.117 Participants Needed

Entinostat + Aldesleukin for Kidney CancerColumbus, Ohio

This phase I/II trial studies the side effects and best dose of entinostat when given together with aldesleukin and to see how well this works in treating patients with kidney cancer that has spread to other places in the body. Entinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Aldesleukin may stimulate the white blood cells to kill kidney cancer cells. Giving entinostat together with aldesleukin may be a better treatment for metastatic kidney cancer.47 Participants Needed

Vorinostat + Rituximab + Chemotherapy for Diffuse Large B-Cell LymphomaColumbus, Ohio

This phase I/II trial is studying the side effects and best dose of vorinostat when given together with rituximab and combination chemotherapy and to see how well it works in treating patients with newly diagnosed stage II, stage III, or stage IV diffuse large B-cell lymphoma. Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the cancer. Monoclonal antibodies, such as rituximab, can block cancer cell growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cell-killing substances to them. Drugs used in chemotherapy, such as cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving vorinostat together with rituximab and combination chemotherapy may kill more cancer cells.83 Participants Needed

CNP-106 for Myasthenia GravisColombus, Ohio

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.54 Participants Needed

SARS-CoV-2 CTLS for COVID-19Columbus, Ohio

The 2019 Severe Acute Respiratory Syndrome (SARS) is a global pandemic secondary to a novel coronavirus - SARS-CoV-2. The reported case-fatality ratio for SARS-CoV-2 in the United States is 1.8% with a current death toll of \>300,000 and climbing.4 There is no accepted standard of care or FDA approved therapies for treatment of COVID-19. Virus specific cytotoxic T lymphocytes (CTLs) have become an important part of the treatment landscape for viral reactivation post hematopoietic and solid organ transplantation. Donor derived CTLs have been shown to be safe and effective against a variety of viruses including CMV, EBV, BK and adenovirus. We hypothesize that SARS-CoV-2 specific CTLs generated from a previously infected family donor will be safe and effective for treatment of COVID-19 in family members with mild to moderate disease.50 Participants Needed

Gene Therapy for Sickle Cell DiseaseColumbus, Ohio

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.15 Participants Needed

Radium-223 + M3814 + Avelumab for Prostate CancerColumbus, Ohio

This phase I/II trial studies the best dose of M3814 when given together with radium-223 dichloride or with radium-223 dichloride and avelumab and to see how well they work in treating patients with castrate-resistant prostate cancer that had spread to other places in the body (metastatic). M3814 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Radioactive drugs, such as radium-223 dichloride, may carry radiation directly to tumor cells and not harm normal cells. Immunotherapy with monoclonal antibodies, such as avelumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. This study is being done to find out the better treatment between radium-223 dichloride alone, radium-223 dichloride in combination with M3814, or radium-223 dichloride in combination with both M3814 and avelumab, to lower the chance of prostate cancer growing or spreading in the bone, and if this approach is better or worse than the usual approach for advanced prostate cancer not responsive to hormonal therapy.90 Participants Needed

Venetoclax + Lenalidomide + Rituximab for Mantle Cell LymphomaColumbus, Ohio

The purpose of this study is to determine if giving an experimental drug called venetoclax in combination with lenalidomide and rituximab is safe and effective for treating people with Mantle Cell Lymphoma (MCL).28 Participants Needed

Flu and COVID-19 Vaccines for Coronavirus and InfluenzaColumbus, Ohio

The purpose of this study is to learn about how the flu and COVID vaccines act when given alone or when mixed together. This study is seeking healthy participants aged 18 or older. All participants in this study will receive only 1 shot to their arm, either a flu or COVID vaccine, alone or mixed. Participants will take part in this study for about 6 months, and participants will need to visit the clinical study site at least 4 times.2050 Participants Needed

CAR T Cell Therapy for LeukemiaColumbus, Ohio

This is a Phase Ib/II study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting CD19 in adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).153 Participants Needed

Papaverine + Chemoradiation for Lung CancerColumbus, Ohio

This phase I trial finds out the best dose, possible benefits and/or side effects of papaverine when given together with chemoradiation intreating patients with stage II-III non-small cell lung cancer. Papaverine targets mitochondrial metabolism to decrease the cancer growth process. Giving papaverine with chemoradiation may work best to treat patients with non-small cell lung cancer.28 Participants Needed

Mammaglobin-A DNA Vaccine for Breast CancerColumbus, Ohio

The purpose of this research study is to find out about the safety of injecting the gene (DNA) for mammaglobin-A into people with breast cancer. The DNA used in this study was purified from bacteria and contains the gene for mammaglobin-A. Mammaglobin-A is a protein that is highly expressed by breast cancer cells. Injection of mammaglobin-A DNA may be a way to generate an immune response to breast cancer cells. There is evidence that an immune response may be a way to fight cancer. In addition to evaluating the safety of the mammaglobin-A injection, this study is also looking at the immune response that the participant's body has after each injection.27 Participants Needed

VRC07-523LS + PGT121.414.LS for HIVColumbus, Ohio

The purpose of this study is to evaluate the safety, tolerability, and efficacy of combination broadly neutralizing antibodies (bNAbs), to induce HIV-1 control during analytic treatment interruption (ATI).40 Participants Needed

CAR T Cell Therapy for Brain CancerColumbus, Ohio

This phase I trial tests the safety, side effects, and best dose of anti-glycoprotein-A repetitions predominant (GARP) chimeric antigen receptor (CAR) T cell therapy and how well it works in treating patients with grade III or IV gliomas that have come back after a period of improvement (recurrent). CAR T-cell therapy is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack tumor cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein, such as GARP, on the patient's tumor cells is added to the T cells in the laboratory. The special receptor is called a CAR. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain tumors. Giving anti-GARP CAR T cell therapy may be safe, tolerable, and/or effective in treating patients with recurrent grade III or IV gliomas.30 Participants Needed

NN3201 for CancerColumbus, Ohio

This open-label clinical trial will evaluate the safety and tolerability of NN3201 in subjects with advanced and/or metastatic solid tumors known to express c-Kit.67 Participants Needed

Peptide Vaccine for Cancer PreventionColumbus, Ohio

This phase 1B trial tests the safety, side effects and best dose of rh-Hsc70- BZLF1 peptide complex (OSU-2131) with Stimulon (Trademark) QS-21 and evaluates how well it works in preventing Epstein-Barr virus (EBV) infection and related cancers in healthy volunteers and patients awaiting a solid organ transplant. Currently, patients who receive an organ transplant receive immune suppression therapy which can make it harder for the body to fight infections. This treatment also increases the risk for cancers that are triggered by the EBV. Vaccines made from synthetic peptide (RAKFKQLL) derived from the BZLF1 protein, may help the body build an effective immune response against EBV infections. QS-21, a saponin adjuvant, is a substance from plants that, when given with vaccine therapy, may improve the way the immune system responds to disease. Giving OSU-2131 with QS-21 may help the immune system fight EBV and protect against EBV infection and the cancers that it can cause in patients awaiting solid organ transplants.55 Participants Needed

Epcoritamab for PTLDColumbus, Ohio

This phase Ib trial tests the safety and effectiveness of epcoritamab in treating patients with post-transplant lymphoproliferative disorder (PTLD) that has come back after a period of improvement (relapsed) or has not responded to previous treatment (refractory). Epcoritamab, a bispecific antibody, binds to a protein called CD3, which is found on T cells (a type of white blood cell). It also binds to a protein called CD20, which is found on B cells (another type of white blood cell) and some lymphoma cells. This may help the immune system kill cancer cells. Giving epcoritamab may be safe and effective in treating patients with relapsed or refractory B-cell PTLD.26 Participants Needed

Vismodegib + Atezolizumab for Lung CancerColumbus, Ohio

This phase Ib trial tests the safety, side effects, and best dose of the combination of vismodegib and atezolizumab in treating patients with non-small cell lung cancer (NSCLC) that has come back after a period of improvement (recurrent) or has spread from where it first started (primary site) to other places in the body (metastatic). Vismodegib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving a combination of vismodegib and atezolizumab may be safe, tolerable and/or effective than either drug alone in treating patients with recurrent or metastatic NSCLC.24 Participants Needed

Tolinapant + Eribulin for Breast CancerColumbus, Ohio

This phase I/Ib trial tests the safety, side effects, best dose, and effectiveness of ASTX660 (tolinapant) in combination with eribulin mesylate (eribulin) in treating patients with triple negative breast cancer that cannot be removed by surgery (unresectable) or that has spread to nearby tissues or lymph nodes (locally advanced) or to other places in the body (metastatic). Tolinapant may stop the growth of tumor cells by blocking proteins, such as XIAP and cIAP1, needed for tumor cell survival. Chemotherapy drugs, such as eribulin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving tolinapant in combination with eribulin may be safe, tolerable, and/or effective in treating patients with unresectable, locally advanced, or metastatic triple negative breast cancer.42 Participants Needed

LH-001 for Healthy VolunteersColumbus, Ohio

The purpose of this clinical trial is to evaluate the safety and tolerability of LH-001 when administered as an oral, single or multiple dose(s) at ascending dose levels in healthy participants.56 Participants Needed

SYNCAR-001 + STK-009 for LupusColumbus, Ohio

This is a phase 1 study of SYNCAR-001 + STK-009 in patients with severe, refractory systemic autoimmune rheumatic disease.48 Participants Needed

Eganelisib + Cytarabine for AMLColumbus, Ohio

This is a Phase 1b open-label, multicenter, dose-escalation and dose-optimization study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and anti-tumor efficacy of eganelisib as monotherapy and in combination with cytarabine in patients with relapsed/refractory (r/r) acute myeloid leukemia (AML) or r/r higher-risk myelodysplastic syndromes (HR-MDS). The study consists of 2 parts: * Part 1: Dose Escalation (DE) in both monotherapy and in combination. * Part 2: Dose Optimization125 Participants Needed

Teclistamab + Iberdomide for Multiple MyelomaColumbus, Ohio

This phase Ib trial tests the safety, side effects, and best dose of iberdomide in combination with teclistamab in treating multiple myeloma that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Iberdomide is a medication that belongs to a group of drugs known as cereblon E3 ligase modulators. Iberdomide works by targeting and destroying proteins that help myeloma cancer cells to survive. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Immunotherapy with monoclonal antibodies, such as teclistamab, may help the body's immune system attack the cancer, and may interfere with the ability of cancer cells to grow and spread. Giving iberdomide in combination with teclistamab may be safe and tolerable in treating patients with relapsed or refractory multiple myeloma.26 Participants Needed

AJ1-11095 for MyelofibrosisColumbus, Ohio

AJX-101 is a first-in-human (FIH), phase 1, non-randomized, multi-center, open-label clinical trial designed to investigate the safety, tolerability, pharmacokinetics (PK), clinical activity and changes in biomarkers of an orally administered type II JAK2 inhibitor, AJ1-11095, in subjects with primary or secondary myelofibrosis previously treated with at least one type I JAK2 inhibitor.76 Participants Needed

SRD-001 for Duchenne Muscular DystrophyColumbus, Ohio

This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.12 Participants Needed

SNDX-5613 + Gilteritinib for Acute Myeloid LeukemiaColumbus, Ohio

This phase I trial tests the safety, side effects, and best dose of SNDX-5613 and gilteritinib for treating patients with acute myeloid leukemia that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory) and has a mutation in the FLT3 gene along with either a mutation in the NMP1 gene or a type of mutation called a rearrangement in the MLL gene. SNDX-5613 is in a class of medications called menin inhibitors. It works by blocking the action of mutated MLL and NMP1 proteins that signal cancer cells to multiply. Gilteritinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of mutated FLT3 proteins that signal cancer cells to multiply. Giving SNDX-5613 with gilteritinib may be safe, tolerable and/or effective in treating patients with relapsed/refractory FLT3 mutated acute myeloid leukemia.30 Participants Needed

CC-99282 + Rituximab for Non-Hodgkin's LymphomaColumbus, Ohio

This phase I trial tests the safety, side effects and best dose of CC-99282 with rituximab for the treatment of patients who have received chimeric antigen receptor (CAR) T cell therapy for non-Hodgkins lymphoma and in whom have had a sub-optimal response early on to CAR T-cell therapy. Immunotherapy with CC-99282 may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Rituximab is a monoclonal antibody. It binds to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Giving CC-99282 with rituximab may be a safe and effective treatment option for patients who have received CAR-T cell therapy for relapsed or refractory non-Hodgkin's lymphoma.18 Participants Needed

Donor Immune Cells + Isatuximab for Multiple MyelomaColumbus, Ohio

This phase I trial tests the side effects and best dose of TGFbi natural killer (NK) cells (TiNK) when given together with isatuximab for the treatment of patients with multiple myeloma that has come back after a period of improvement (relapsed) or that has not responded to treatment (refractory). NK cells are a type of white blood cell that are known to spontaneously attack cancer cells. TiNK are NK cells made in a laboratory to have a higher response to tumor cells. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Immunotherapy with monoclonal antibodies, such as isatuximab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Patients also receive standard treatment (cyclophosphamide and dexamethasone) on this trial. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill cancer cells. It may also lower the body's immune response. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Giving TiNK and isatuximab with standard treatment may be a safe and effective treatment for relapsed or refractory multiple myeloma.30 Participants Needed

ZEN003694 + Chemotherapy for Colorectal CancerColumbus, Ohio

This phase I trial tests the safety, best dose, and effectiveness of ZEN003694 in combination with cetuximab and encorafenib in treating patients with colorectal cancer that has not responded to previous treatment (refractory), that has come back after a period of improvement (relapsed), and that has spread from where it first started (primary site) to other places in the body (metastatic). ZEN003694 is a protein inhibitor that binds to BET proteins. When ZEN003694 binds to BET proteins, it disrupts gene expression. Preventing the expression of certain growth-promoting genes may inhibit proliferation of tumor cells that over-express BET proteins. Immunotherapy with monoclonal antibodies, such as cetuximab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Encorafenib is an enzyme inhibitor. It inhibits pathways that are responsible for controlling cell proliferation and survival, which may lead to a decrease in tumor cell proliferation. Both cetuximab and encorafenib have been approved to treat cancer. Adding ZEN003694 to cetuximab and encorafenib may be more effective at treating patients with refractory metastatic colorectal cancer than giving the usual treatment (cetuximab and encorafenib) alone.30 Participants Needed

ONM-501 + Cemiplimab for CancerColumbus, Ohio

A phase 1, multicenter, open label, non-randomized dose escalation and dose expansion study to examine the maximum tolerated dose, (MTD), minimum effective dose (MED) and/or recommended dose for expansion (RDE) of intratumoral ONM-501 as monotherapy and in combination with a PD-1 checkpoint inhibitor in patients with advanced solid tumors and lymphomas.168 Participants Needed

Elotuzumab + Mezigdomide + Dexamethasone for Multiple MyelomaColumbus, Ohio

This phase Ib trial tests the safety, side effects, and best dose of CC-92480 in combination with elotuzumab and dexamethasone in treating patients with multiple myeloma that has come back after a period of improvement (relapsed) or that does not respond to treatment or has not responded to previous treatment (refractory). Multiple myeloma (MM) remains the second most common hematologic malignancy in the United States. A number of therapies have been approved for patients with MM, including CD38- and B-cell maturating antigen (BCMA)-targeted therapies (antibody and plasma cell treatments that help the body's immune system to kill cancer cells); however, patients will often relapse and become refractory to these therapies. Because of this, it is important to identify effective treatment options for patients progressing on anti-CD38 therapy and BCMA-directed therapies. Elotuzumab is a humanized IgG1 monoclonal antibody, which is a type of protein that can bind to other target cells to prevent them from working the way they should or cause them to act differently. Elotuzumab works by targeting a protein called SLAMF7, which is present on myeloma cells, and makes it easier for the immune system to target the cancer. CC-92480 works by binding to a protein called CRBN that triggers the breakdown of proteins: Ikaros and Aiolos, leading to cell death in multiple myeloma cells. Dexamethasone is a synthetic adrenocortical steroid, or steroid normally naturally made by the adrenal gland in the brain which has been produced in a laboratory, that helps to regulate the amount of different chemicals and water that are being processed by the kidneys. It is also used in patients with myeloma to help treat their disease. The combination of CC-92480 with elotuzumab and dexamethasone may be a safe and effective treatment when given to patients with relapsed or recurrent MM.27 Participants Needed

M1774 + ZEN-3694 for Ovarian and Endometrial CancerColumbus, Ohio

This phase Ib trial tests the safety, side effects, and best dose of M1774 when given with ZEN-3694 in treating patients with ovarian and endometrial cancer that has come back (recurrent). M1774 and ZEN-3694 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. M1774 and ZEN-3694 combined together has demonstrated to be better than either drug alone in killing ovarian tumor cells.60 Participants Needed

SNDX-5613 + Chemotherapy for Acute Myeloid LeukemiaColumbus, Ohio

This phase Ib trial tests the safety, side effects, and best dose of SNDX-5613 when given in combination with the standard chemotherapy treatment (daunorubicin and cytarabine) in treating patients with newly diagnosed acute myeloid leukemia that has changes in the NPM1 gene or MLL/KMT2A gene. SNDX-5613 blocks signals passed from one molecule to another inside cancer cells that are needed for cancer cell survival. Drugs used in chemotherapy, such as daunorubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding SNDX-5613 to the standard chemotherapy treatment may be able to shrink or stabilize the cancer for longer than the standard chemotherapy treatment alone.28 Participants Needed

Tegavivint for Large B-Cell LymphomaColumbus, Ohio

This phase I trial tests the safety, side effects, and best dose of tegavivint in treating patients with large b-cell lymphomas that has come back (relapsed) or does not respond to treatment (refractory). Tegavivint may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving tegavivint may help control the disease.20 Participants Needed

Ziftomenib Combinations for Acute Myeloid LeukemiaColumbus, Ohio

This Phase 1 study will assess the safety, tolerability, and preliminary antileukemic activity of ziftomenib in combination with venetoclax and azacitidine (ven/aza), ven, and 7+3 for two different molecularly-defined arms, NPM1-m and KMT2A-r.212 Participants Needed

INZ-701 for Metabolic Bone DiseaseColumbus, Ohio

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.16 Participants Needed

Novobiocin for CancerColumbus, Ohio

This trial tests novobiocin, an antibiotic, in cancer patients with specific DNA repair gene mutations who haven't responded to other treatments. Novobiocin blocks a protein that helps cancer cells repair themselves, leading to their death. Studies show that novobiocin can make other cancer treatments more effective.31 Participants Needed

CA-4948 + Chemotherapy for Pancreatic CancerColumbus, Ohio

This phase I trial tests the safety, side effects, and best dose of emavusertib (CA-4948) in combination with gemcitabine and nab-paclitaxel in treating patients with pancreatic ductal adenocarcinoma that has spread from where it first started (primary site) to other places in the body (metastatic) or cannot be removed by surgery (unresectable). CA-4948 is in a class of medications called kinase inhibitors. It works by blocking the action of abnormal proteins called interleukin-1 receptor-associated kinase 4 (IRAK4) and FMS-like tyrosine kinase 3 (FLT3) that signal cells to multiply. This may help keep cancer cells from growing. The usual approach for patients with pancreatic ductal adenocarcinoma is treatment with chemotherapy drugs gemcitabine and nab-paclitaxel. Gemcitabine is a chemotherapy drug that blocks the cells from making DNA and may kill cancer cells. Paclitaxel is in a class of medications called anti-microtubule agents. It stops cancer cells from growing and dividing and may kill them. Nab-paclitaxel is an albumin-stabilized nanoparticle formulation of paclitaxel which may have fewer side effects and work better than other forms of paclitaxel. Giving CA-4948 in combination with gemcitabine and nab-paclitaxel may shrink or stabilize metastatic or unresectable pancreatic ductal adenocarcinoma.36 Participants Needed

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