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Why We Started Power

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
Bask GillCEO at Power
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      Why We Started Power

      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
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      46 Bone Marrow Failure Trials Near You

      Power is an online platform that helps thousands of Bone Marrow Failure 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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      Treosulfan-Based Conditioning for Bone Marrow Failure

      Columbus, Ohio
      This trial tests if a combination of three drugs can reduce complications for patients with bone marrow failure diseases. The drugs work by killing harmful cells, stopping their growth, and reducing immune reactions.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:1 - 49

      Key Eligibility Criteria

      Disqualifiers:Aplastic Anemia, Fanconi Anemia, MDS, Others

      40 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

      Bone Marrow Transplant for Dyskeratosis Congenita

      Cincinnati, Ohio
      This trial tests a new bone marrow transplant method for patients with Dyskeratosis congenita. It uses fludarabine and antibodies to avoid harmful side effects, aiming to improve survival and reduce complications. Fludarabine-based regimens have been shown to be effective and feasible in reducing transplant-related morbidity in patients with Dyskeratosis congenita.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:30 - 65

      Key Eligibility Criteria

      Disqualifiers:MDS, AML, HIV, Pregnancy, Others
      Must Not Be Taking:Fludarabine, Alemtuzumab, Mycophenolate, Others

      40 Participants Needed

      EXG34217 for Bone Marrow Failure

      Cincinnati, Ohio
      This trial tests the safety and tolerability of EXG34217 in patients with bone marrow failure due to telomere biology disorders. The treatment involves collecting, processing, and reinfusing the patient's own blood cells to help improve their bone marrow function.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:Cancer, Severe Bone Marrow Failure, Others
      Must Not Be Taking:Danazol, Androgens

      12 Participants Needed

      Emapalumab for Aplastic Anemia

      Cincinnati, Ohio
      The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:0 - 25

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Previous SAA Treatment, Inherited Bone Marrow Failure, Others

      35 Participants Needed

      EXG34217 for Bone Marrow Failure Syndrome

      Cincinnati, Ohio
      This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1

      12 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

      Eltrombopag for Aplastic Anemia

      Cleveland, Ohio
      This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:1 - 18

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      51 Participants Needed

      Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders

      Pittsburgh, Pennsylvania
      The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:2 - 55

      Key Eligibility Criteria

      Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others

      100 Participants Needed

      RIC + BMT for Non-Malignant Disorders

      Grand Rapids, Michigan
      This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:1 - 21

      Key Eligibility Criteria

      Disqualifiers:HLA-identical Sibling Donor, Cirrhosis, Uncontrolled Infection, HIV, Others

      29 Participants Needed

      Eltrombopag for Aplastic Anemia

      Bethesda, Maryland
      Background: * Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment. * Thrombopoietin (TPO) is a protein made by the body. The bone marrow needs TPO to produce platelets. TPO may also be able to stimulate bone marrow stem cells to produce red cells and white cells. However, TPO cannot be given by mouth. This has led researchers to develop the drug eltrombopag, which acts in the same way and can be given by mouth. Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts. Objectives: - To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia or patients with bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts. Eligibility: - People at least 2 years of age who have moderate aplastic anemia or bone marrow failure and unilineage cytopenia,and significantly low blood cell counts. Design: * Patients will be screened with a physical examination, medical history, blood tests, a bone marrow biopsy, and an eye exam. * Patients will receive eltrombopag by mouth once a day. * Patients will have weekly blood tests to monitor the effectiveness of the treatment and adjust the dose in response to possible side effects. * Patients may continue to take eltrombopag if their platelet count or hemoglobin increases, their requirement for platelet or blood transfusion decreases after 16 to 20 weeks of treatment, and there have been no serious side effects. Access to the drug will continue until the study is closed. Patients will be asked to return for a follow-up visit 6 months after the last dose of medication.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:2 - 100

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, HIV, Cancer, Others
      Must Not Be Taking:ATG, Campath, Cytokines, Others

      34 Participants Needed

      Imatinib for Bone Marrow Failure Syndrome

      Bethesda, Maryland
      Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes. Objective: The purpose of the study includes determining which dose of imatinib is best for people with pathogenic or likely pathogenic RUNX1 mutations without blood cancers, and to determine whether there are any changes in platelet function and inflammatory markers. Eligibility: Adults aged 18 and older with RUNX1 mutations. Healthy people without this mutation, including family members of affected participants, are also needed. Design: Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood tests. They will have a test of their heart function. They may need a new bone marrow biopsy if they haven't had one in the past year. Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 28 days or up to 84 days. They will fill out questionnaires about how they are feeling. For the first part of the study, participants will have blood tests every 2 weeks, either at home or at the NIH, while they are taking the imatinib. They will have a follow up visit, at home or at the NIH, when they are done taking imatinib on Day 28. Participants on the second part of the study will come to NIH on days 1 and days 84. They will have blood tests every 2 weeks (at home or the NIH) while they are taking imatinib. They may opt to have a bone marrow biopsy repeated after they finish their course of imatinib. Participants will have a follow-up visit (at home or the NIH) 30 days after they stop taking imatinib. Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Hematologic Therapy, Cardiac Conditions, Others
      Must Not Be Taking:Aspirin, Anti-platelet, CYP3A4 Inhibitors, Others

      75 Participants Needed

      Bitopertin for Diamond-Blackfan Anemia

      Bethesda, Maryland
      This trial tests bitopertin, a daily pill, in adults with Diamond-Blackfan anemia. The drug aims to reduce harmful levels of heme in the blood, which can help manage the disease. Participants will take the drug for several months, with periodic check-ups to monitor their response. Bitopertin has shown promise in improving anemia in a mouse model.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Cardiac Disease, Active Infections, Pregnancy, Others
      Must Not Be Taking:Androgens, Corticosteroids, Antivirals, Others

      30 Participants Needed

      Pacritinib for Myelodysplastic Syndrome

      Bethesda, Maryland
      Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed. Objective: To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN. Eligibility: Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN. Design: Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone. Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants. Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years. Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 1, 2
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:Bleeding Disorders, Cardiac Conditions, Infections, Others
      Must Not Be Taking:Anticoagulants, Antiplatelets, CYP3A4 Inhibitors

      160 Participants Needed

      Reduced Intensity BMT + Cyclophosphamide for Primary Immunodeficiency & Bone Marrow Failure

      Baltimore, Maryland
      Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:4 - 50

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis B/C, Pregnancy, Others

      27 Participants Needed

      Darzalex Faspro for Blood Cancers

      Baltimore, Maryland
      This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1

      Key Eligibility Criteria

      Disqualifiers:COPD, Asthma, HIV, Hepatitis, Others
      Must Not Be Taking:Anti-CD38 Therapies

      8 Participants Needed

      Cord Blood Transplant for Blood Diseases

      Rochester, New York
      This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Age:2 - 75

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, HIV, Uncontrolled Infection, Others

      30 Participants Needed

      Let's Get REAL Tool for Pediatric Blood Cancers

      St Louis, Missouri
      The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:8+

      Key Eligibility Criteria

      Disqualifiers:Severe Medical Problems, Cognitive Incapacity, Others

      60 Participants Needed

      Fludarabine RIC for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1
      Age:< 22

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infections, Myelodysplastic Syndrome, Others

      25 Participants Needed

      NR Supplementation + Exercise for Cancer Survivors

      Philadelphia, Pennsylvania
      This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors. The primary outcome is the change in muscle strength (isometric knee extension) from baseline to 16 weeks. Key secondary outcomes are the change in muscle strength (ankle plantarflexion) from baseline to 16 weeks, the change in grip strength from baseline to 16 weeks, the change in lower extremity muscle mass from baseline to 16 weeks, the change in muscle OXPHOS capacity from baseline to 16 weeks, and the change in aerobic capacity (VO2 max) from baseline to 16 weeks.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:10 - 30

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Kidney Disease, Liver Disease, Others
      Must Not Be Taking:Statins, Glucocorticoids, NAD+ Precursors

      80 Participants Needed

      Why Other Patients Applied

      "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

      "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

      "I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

      ID
      Pancreatic Cancer PatientAge: 40

      "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

      "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
      Match to a Bone Marrow Failure Trial

      Stem Cell Transplant for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:< 25

      Key Eligibility Criteria

      Disqualifiers:Myelodysplastic Syndrome, No Suitable Donor, Pregnancy, Others

      50 Participants Needed

      Stem Cell Transplant for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1
      Age:< 30

      Key Eligibility Criteria

      Disqualifiers:Infections, HLA Matched Sibling, Pregnancy

      100 Participants Needed

      Reduced-Intensity Conditioning for Blood Cancers

      Philadelphia, Pennsylvania
      This phase II clinical trial evaluates whether a modified modality of conditioning reduces treatment-related mortality (TRM) in patients who undergo a hematopoietic stem cell transplant (HSCT) for a hematological malignancy. HSCT is a curative therapy for many hematopoietic malignancies, however this regimen results in higher rates of TRM than other forms of treatment. In recent years, less intense conditioning regimens with radiation and chemotherapy prior to HSCT have been developed. Radiation therapy uses high energy sources to kill cancer cells and shrink tumors while chemotherapy drugs like fludarabine and cyclophosphamide 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. This study evaluates whether a two-step approach with lower-intensity regimens of these treatments prior to HSCT reduces the rate of TRM.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:HIV, CNS Malignancy, Pregnancy, Others

      63 Participants Needed

      CD45RA Depleted Stem Cell Addback for Leukemia

      Philadelphia, Pennsylvania
      The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab/CD19 depleted PSCT.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:1 - 25

      Key Eligibility Criteria

      Disqualifiers:Hodgkin Lymphoma, Non-Burkitt Lymphoma, Others
      Must Not Be Taking:Alemtuzumab

      100 Participants Needed

      Ruxolitinib for Sickle Cell Disease

      Philadelphia, Pennsylvania
      This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants. Recruitment is expected to last for about 2 years and participants will be followed for an additional 2 years post-HCT.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 1, 2
      Age:12 - 45

      Key Eligibility Criteria

      Disqualifiers:HLA-matched Sibling, Uncontrolled Infection, HIV, Others
      Must Not Be Taking:Ruxolitinib Metabolism Interferers

      24 Participants Needed

      Romiplostim for Aplastic Anemia

      Iowa City, Iowa
      This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage hematopoiesis in children and young adults.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 2
      Age:2 - 21

      Key Eligibility Criteria

      Disqualifiers:PNH, Thrombosis, Cancer Predisposition, MDS, Others
      Must Not Be Taking:Eltrombopag, Androgens, Others

      15 Participants Needed

      CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes

      New York, New York
      This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:< 40

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Pregnancy, HIV, Others

      37 Participants Needed

      Stem Cell Transplant for Leukemia

      New York, New York
      The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of the CliniMACS® Reagent System (Miltenyi Biotec), a CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD. This study involves subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's).
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:< 22

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Pregnancy, Breastfeeding

      14 Participants Needed

      Cord Blood Transplant for Blood Cancers

      New York, New York
      This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 21

      Key Eligibility Criteria

      Disqualifiers:Advanced Metabolic Disease, CNS Leukemic, Others

      31 Participants Needed

      Haploidentical Transplant for Severe Aplastic Anemia

      Memphis, Tennessee
      This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT). Primary Objectives * To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide. * To estimate the overall survival and event free survival at 1-year post transplantation. Secondary Objectives * To calculate the incidence of acute and chronic GVHD after HCT. * To calculate the rate of secondary graft rejection at 1-year post transplantation * To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus). * To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year. Exploratory Objectives * To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA. * To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT. * To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation. * To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI. * Quantify donor derived Treg cells at different time points in patients received HCT. * Determine Treg activation status at different stages after HCT. * Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant? * How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient? * Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 21

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, Dyskeratosis Congenita, MDS, Others
      Must Not Be Taking:Alemtuzumab, ATG

      21 Participants Needed

      12

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      Why We Started Power

      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
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
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      Frequently Asked Questions

      How much do Bone Marrow Failure 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 Bone Marrow Failure 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 Bone Marrow Failure 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 Bone Marrow Failure 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 Bone Marrow Failure 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 Bone Marrow Failure clinical trials?

      Most recently, we added Ruxolitinib for Sickle Cell Disease, Pacritinib for Myelodysplastic Syndrome and Ruxolitinib for Aplastic Anemia to the Power online platform.