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61 Muscular Dystrophy Trials Near You
Power is an online platform that helps thousands of Muscular Dystrophy patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerEmavusertib (+ Venetoclax) for Acute Myeloid Leukemia
Columbus, 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:APL, CNS Leukemia, CML, Others
Must Not Be Taking:Chemotherapy, Immunomodulatory Drugs
366 Participants Needed
Pomalidomide + Chemotherapy for Acute Myeloid Leukemia
Columbus, Ohio
This trial is testing if adding pomalidomide to standard chemotherapy can improve treatment for patients with a specific type of newly diagnosed leukemia. Pomalidomide works by cutting off the blood supply to cancer, boosting the immune system, and killing cancer cells. The chemotherapy drugs attack cancer cells in multiple ways. Pomalidomide is related to thalidomide and has shown remarkable activity in patients who did not respond to other treatments.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Wilson's Disease, CNS Leukemia, Others
Must Not Be Taking:Strong CYP1A2 Inhibitors
50 Participants Needed
This trial studies a chemotherapy treatment that adjusts based on how well patients respond initially. It targets younger patients with Down syndrome who have certain types of blood cancer. The treatment aims to effectively kill cancer cells while reducing side effects.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:91 - 3
Key Eligibility Criteria
Disqualifiers:Promyelocytic Leukemia, Prior Therapy, Others
280 Participants Needed
Venetoclax + ASTX727 for Chronic Myelomonocytic Leukemia
Columbus, Ohio
This trial is testing if a combination of two drugs, ASTX727 and venetoclax, is more effective than ASTX727 alone in treating certain bone marrow cancers. It focuses on patients with CMML and MDS/MPN who have too many immature blood cells. ASTX727 helps produce normal blood cells and kills abnormal ones, while venetoclax blocks a protein that cancer cells need to survive. Venetoclax is an anticancer drug used to treat lymphomas and leukemias, but it has severe side effects.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, CNS Disease, Active Infection, Others
Must Not Be Taking:Lenalidomide, Decitabine, Azacitidine, Others
132 Participants Needed
INCB057643 +/- Ruxolitinib for Myelofibrosis
Columbus, Ohio
This trial is testing a new drug called INCB057643, alone or with ruxolitinib, for patients with certain blood cancers like myelofibrosis. The goal is to see if it can stop cancer growth or help other treatments work better. Ruxolitinib has been approved for the treatment of myelofibrosis and has shown clinical benefits in reducing symptoms and improving survival.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Prior BET Inhibitor, Uncontrolled Cardiac, Others
Must Be Taking:Ruxolitinib
231 Participants Needed
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, stopping them from dividing, or by stopping them from spreading. Lenalidomide may stop the growth of cancer cells by stopping blood flow to the cancer. Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It is not yet known whether azacitidine is more effective with or without lenalidomide or vorinostat in treating myelodysplastic syndromes or chronic myelomonocytic leukemia.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:AML, Radiation, Transplant, Neurotoxicity, Others
Must Not Be Taking:Lenalidomide, Azacitidine, Vorinostat, Decitabine
282 Participants Needed
Geriatric Care Optimization for Blood Cancers
Columbus, Ohio
This clinical trial tests whether a geriatric optimization plan (GO!) works to improve survival in patients over 60 with a hematologic malignancy or bone marrow failure syndrome eligible for allogeneic hematopoietic cell transplant. GO! focuses on creating a tailored and specific plan for each patient to make changes in their daily lives. These may include changes to their diet, sleep, activity, medicines, or even referrals to other providers depending on the patient's needs. Studying survival and quality of life in patients over 60 receiving an allogeneic hematopoietic cell transplant may help identify the effects of treatment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:60+
Key Eligibility Criteria
Disqualifiers:Dementia, No Transplant, Others
30 Participants Needed
Diagnostic Testing for Pediatric Leukemia
Columbus, 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:< 22
Key Eligibility Criteria
Disqualifiers:Over 22 Years, Others
960 Participants Needed
Vaccine Therapy for Blood Cancers
Columbus, Ohio
This randomized phase II trial studies how well vaccine therapy works in reducing the frequency of cytomegalovirus severe infections (events) in patients with hematologic malignancies undergoing donor stem cell transplant. Vaccines made from a peptide may help the body build an effective immune response and may reduce cytomegalovirus events after donor stem cell transplant.
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
61 Participants Needed
This randomized phase II/III trial studies how well azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone work in treating older patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Drugs used in chemotherapy, such as azacitidine, decitabine, 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. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Midostaurin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone may kill more cancer cells.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Age:60+
Key Eligibility Criteria
Disqualifiers:Not Listed
76 Participants Needed
This randomized phase III trial studies lenalidomide to see how well it works with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia. Lenalidomide may stop the growth of myelodysplastic syndrome by blocking blood flow to the cells. Colony stimulating factors, such as epoetin alfa, may increase the number of immune cells found in bone marrow or peripheral blood. It is not yet known whether lenalidomide is more effective with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Hypertension, HIV, Others
Must Be Taking:Lenalidomide
247 Participants Needed
Venetoclax + Azacitidine for Myelodysplastic Syndrome
Cincinnati, Ohio
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute myeloid leukemia (AML), and a risk of death from the disease itself. Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to anemia (low red blood cell count), easy or unusual bruising, and red spots just beneath the skin caused by bleeding. The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS.
Venetoclax is an investigational drug being developed for the treatment of MDS. The study consists of two treatment arms - In one arm, participants will receive venetoclax and AZA. In another arm, participants will receive AZA and placebo. Adult participants with newly diagnosed higher-risk MDS will be enrolled. Around 500 participants will be enrolled in approximately 220 sites worldwide.
Participants in one arm will receive oral doses of venetoclax tablet and intravenous (infusion in the vein) or subcutaneous (given under the skin) AZA solution. Participants in another arm will receive oral doses of placebo tablet and intravenous or subcutaneous AZA solution.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.
Prior Safety Data
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Therapy-related MDS, MDS/MPN, Others
Must Not Be Taking:Hypomethylating Agents, Chemotherapy
531 Participants Needed
NS-089/NCNP-02 for Duchenne Muscular Dystrophy
Cincinnati, Ohio
This trial tests an IV medication called NS-089/NCNP-02 in boys aged 4 to 14 with a specific type of Duchenne Muscular Dystrophy. The treatment aims to help their bodies make better muscle proteins by skipping over a broken part of their gene. NS-089/NCNP-02 is a new drug utilizing exon-skipping therapy, similar to NS-065/NCNP-01, which targets specific deletions in the dystrophin gene.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 14
Sex:Male
Key Eligibility Criteria
Disqualifiers:Symptomatic Cardiomyopathy, Gene Therapy, Others
Must Be Taking:Glucocorticoids
20 Participants Needed
CAP-1002 for Duchenne Muscular Dystrophy
Cincinnati, Ohio
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:10+
Key Eligibility Criteria
Disqualifiers:Major Surgery, Respiratory Disease, Hypersensitivity, Others
13 Participants Needed
Cell Therapy for Duchenne Muscular Dystrophy
Cincinnati, Ohio
This trial tests a cell therapy called CAP-1002 in boys and young men with Duchenne muscular dystrophy. The therapy involves giving special cells through an IV to help improve muscle function. The goal is to see if this treatment can repair or regenerate damaged muscles.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:10+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Low LVEF, High BMI, Others
Must Be Taking:Glucocorticoids
104 Participants Needed
Oral Ifetroban for Duchenne Muscular Dystrophy
Cincinnati, Ohio
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD.
Funding Source - FDA OOPD
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:7+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Significant Illness, Major Surgery, Others
Must Be Taking:Corticosteroids
48 Participants Needed
CLN-049 for Leukemia and Myelodysplastic Syndrome
Cincinnati, Ohio
CLN-049-001 is a Phase 1, open-label, multicenter, first-in-human trial of CLN-049 in patients with Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:APL, CNS Leukemia, Organ Allograft, Others
Must Not Be Taking:Immunotherapy, CAR-T, Glucocorticoids, Others
60 Participants Needed
Chemotherapy + Stem Cell Transplant for Fanconi Anemia
Cincinnati, Ohio
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:3+
Key Eligibility Criteria
Disqualifiers:Active CNS Leukemia, HIV, Others
70 Participants Needed
Stem Cell Transplantation with NiCord® for Blood Cancers
Cleveland, Ohio
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Age:12 - 65
Key Eligibility Criteria
Disqualifiers:Not Listed
125 Participants Needed
Olutasidenib for Leukemia
Cleveland, Ohio
This phase I trial tests the safety, side effects, and effectiveness of olutasidenib in preventing the return of disease (relapse) in patients who have undergone donor (allogeneic) hematopoietic cell transplant for acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CMML) carrying an IDH1 mutation. Olutasidenib is in a class of medications called IDH1 inhibitors. It works by slowing or stopping the growth of cancer cells. Giving olutasidenib may be safe, tolerable and/or effective in preventing relapse in patients with IDH1 mutated AML, MDS or CMML after an allogeneic hematopoietic cell transplant.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Active GVHD, Uncontrolled Infection, Active Malignancy, Others
Must Be Taking:IDH1 Inhibitors
15 Participants Needed
Why Other Patients Applied
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
IZ
Healthy Volunteer PatientAge: 38
"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."
ZS
Depression PatientAge: 51
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
HZ
Arthritis PatientAge: 78
Stem Cell Transplantation for Leukemia
Cleveland, Ohio
This phase III trial compares hematopoietic (stem) cell transplantation (HCT) using mismatched related donors (haploidentical \[haplo\]) versus matched unrelated donors (MUD) in treating children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). HCT is considered standard of care treatment for patients with high-risk acute leukemia and MDS. In HCT, patients are given very high doses of chemotherapy and/or radiation therapy, which is intended to kill cancer cells that may be resistant to more standard doses of chemotherapy; unfortunately, this also destroys the normal cells in the bone marrow, including stem cells. After the treatment, patients must have a healthy supply of stem cells reintroduced or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. The healthy stem cells may come from the blood or bone marrow of a related or unrelated donor. If patients do not have a matched related donor, doctors do not know what the next best donor choice is. This trial may help researchers understand whether a haplo related donor or a MUD HCT for children with acute leukemia or MDS is better or if there is no difference at all.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:6 - 21
Key Eligibility Criteria
Disqualifiers:Genetic Disorders, Pregnancy, Uncontrolled Infections, Others
435 Participants Needed
Ivosidenib + Venetoclax +/- Azacitidine for Leukemia
Cleveland, Ohio
This phase Ib/II trial studies the side effects and best dose of venetoclax and how well it works when given together with ivosidenib with or without azacitidine, in treating patients with IDH1-mutated hematologic malignancies. Venetoclax and ivosidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, 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. Giving ivosidenib and venetoclax with azacitidine may work better in treating patients with hematologic malignancies compared to ivosidenib and venetoclax alone.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Active Infection, Active Malignancy, HIV, Others
Must Not Be Taking:CYP3A Inducers
96 Participants Needed
Orca-T for Blood Cancers
Cleveland, Ohio
This study will evaluate the safety, tolerability, and efficacy of Orca-T, an allogeneic stem cell and T-cell immunotherapy biologic manufactured for each patient (transplant recipient) from the mobilized peripheral blood of a specific, unique donor. It is composed of purified hematopoietic stem and progenitor cells (HSPCs), purified regulatory T cells (Tregs), and conventional T cells (Tcons) in participants undergoing myeloablative allogeneic hematopoietic cell transplant transplantation for hematologic malignancies.
This posting represents the Phase III component of Precision-T. The Precision-T Ph1b component is described under NCT04013685.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 65
Key Eligibility Criteria
Disqualifiers:Prior Allogeneic HCT, HIV, Others
Must Not Be Taking:Corticosteroids, Immunosuppressive Therapy
174 Participants Needed
Enasidenib + Cedazuridine-Decitabine for Myelodysplastic Syndrome
Ypsilanti, Michigan
This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:AML Therapy, DNA Methyltransferase Inhibitors, Others
Must Not Be Taking:Cytarabine Therapy
54 Participants Needed
Entinostat + Pembrolizumab for Myelodysplastic Syndrome
Lexington, Kentucky
This phase Ib trial studies the side effects and best dose of entinostat when given together with pembrolizumab in treating patients with myelodysplastic syndrome after deoxyribonucleic acid (DNA) methyltransferase inhibitor (DNMTi) therapy failure. Entinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving entinostat together with pembrolizumab may work better in treating patients with myelodysplastic syndrome after DNMTi therapy failure.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
28 Participants Needed
Gene Therapy for Duchenne Muscular Dystrophy
Pittsburgh, Pennsylvania
This trial tests a gene therapy treatment for boys aged 4 to 7 with muscle problems. The treatment aims to fix the genetic issues causing their muscle problems. Participants will receive the treatment and be monitored for safety and effectiveness. The treatment involves using a specific gene therapy, which has been researched for its potential to correct muscle issues in children with muscle problems.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:4 - 7
Sex:Male
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Antibodies To AAV9, Others
Must Be Taking:Prednisone, Prednisolone, Deflazacort
114 Participants Needed
Vamorolone for Becker Muscular Dystrophy
Pittsburgh, Pennsylvania
This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight \<50 kg) daily administered orally compared to placebo over a treatment period of 24 weeks in males with BMD.
Funding Source - FDA OOPD
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18 - 64
Sex:Male
Key Eligibility Criteria
Disqualifiers:Renal Impairment, Hepatic Impairment, Diabetes, Others
Must Not Be Taking:Mineralocorticoids, Herbal Supplements
39 Participants Needed
NS-050/NCNP-03 for Duchenne Muscular Dystrophy
Pittsburgh, Pennsylvania
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:4 - 14
Sex:Male
Key Eligibility Criteria
Disqualifiers:Symptomatic Cardiomyopathy, Surgery, Others
Must Be Taking:Glucocorticoids
20 Participants Needed
Vesleteplirsen for Duchenne Muscular Dystrophy
Pittsburgh, Pennsylvania
This trial tests vesleteplirsen, a drug, for safety and tolerance. It involves participants from previous studies and new ones. The goal is to find the highest safe dose and monitor side effects.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:7 - 21
Sex:Male
Key Eligibility Criteria
Disqualifiers:Not Listed
62 Participants Needed
Sleep Intervention for Duchenne Muscular Dystrophy
Pittsburgh, Pennsylvania
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary effects of the TranS-CY. Weekly remote (video web conferencing) parent training sessions will allow investigators to explore adoption through parent adherence and examine whether the essential elements of the TranS-CY intervention (e.g., motivational interviewing, goal setting, problem solving, sleep routine scheduling, monitoring) can be consistently taught by clinicians and implemented by parents into the home setting.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:6 - 18
Key Eligibility Criteria
Disqualifiers:Not Listed
100 Participants Needed
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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.
Bask GillCEO at Power
Frequently Asked Questions
How much do Muscular Dystrophy 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 Muscular Dystrophy 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 Muscular Dystrophy 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 for Muscular Dystrophy 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 Muscular Dystrophy 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 Muscular Dystrophy clinical trials?
Most recently, we added Enasidenib + Cedazuridine-Decitabine for Myelodysplastic Syndrome, Olutasidenib for Leukemia and SRP-9004 for Limb-Girdle Muscular Dystrophy to the Power online platform.
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