Clinical trials
Bone Marrow Failure clinical trials
Below are recruiting bone marrow failure clinical trials, each written for real people, not researchers. We’re tracking 148 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06787560RecruitingEarly Phase 1
CAR-T cells and stem cell transplant for blood diseases
This trial tests a new approach for people with non-cancerous blood and immune system diseases. It uses your own engineered immune cells (CAR-T cells) to prepare your body for a stem cell transplant from a relative.
Hangzhou, ZhejiangAges Any age - NCT07227155Recruiting
HOPE Biobank for bone marrow failure patients
This study collects blood samples from patients with aplastic anemia, sickle cell disease, thalassemia, or other bone marrow failure conditions who are receiving a stem cell transplant or gene therapy. The samples will be stored and used for future research to better understand and treat these diseases.
Duarte, CaliforniaAges Any age - NCT06999954Recruiting
Global survey for Shwachman-Diamond syndrome and related conditions
This trial is a global survey and registry for people with Shwachman-Diamond Syndrome and related inherited blood disorders. It aims to connect patients, families, and researchers to better understand these conditions and improve care.
Woburn, MassachusettsAges Any age - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - NCT06817590RecruitingPhase 1
Nucleoside therapy for telomere disorders
This trial tests a nucleoside therapy (dC/dT) for people with telomere biology disorders, which cause premature aging and problems like low blood counts, lung or liver issues. The goal is to see if the treatment can help manage these symptoms.
Boston, MassachusettsAges 1–70 - NCT06412497RecruitingPhase 2
Stem cell transplant for aplastic anemia and bone marrow failure
This trial tests a stem cell transplant using a milder conditioning regimen and a drug called post-transplant cyclophosphamide to treat severe aplastic anemia and other acquired bone marrow failure conditions. The goal is to help patients achieve a functioning donor bone marrow with fewer side effects.
Minneapolis, MinnesotaAges birth–75 years - NCT06090669RecruitingPhase 1
Trial of imatinib for people with RUNX1 deficiency
This trial tests if imatinib can help people with a genetic condition called RUNX1 deficiency, which causes bleeding problems. It aims to increase the activity of the RUNX1 gene to improve blood clotting.
Bethesda, MarylandAges 18–120 - NCT05868499Enrolling by invitationPhase 1
Study follow-up for people who finished the first trial
This Phase 1 study is a follow-up “rollover” for people who already completed the 12-month visit of an earlier study. It checks what happens next after that prior study and may help doctors understand the ongoing safety and effects of the treatment.
Cincinnati, OhioAges 18 years+ - NCT05687149Recruiting
Study of mouth and throat cancer patterns in Fanconi anemia
This study looks at how squamous cell carcinoma (a type of cancer) naturally develops over time in people with Fanconi anemia. It may help doctors better recognize warning signs and plan care in the future.
Bethesda, MarylandAges 8–90 - NCT01962415RecruitingPhase 2
Reduced-intensity stem cell transplant for non-cancer disorders
This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.
Pittsburgh, PennsylvaniaAges 2 months–55 years - NCT04644016RecruitingPhase 2
Cord blood transplant for children and young adults with blood cancers
This Phase 2 trial studies using a cord blood transplant after finding it is risky enough that standard treatment may not be sufficient. It may help certain children and young adults with specific leukemia, lymphoma, and some non-cancer blood/immune or inherited disorders who do not have a well-matched donor available in time.
New York, New YorkAges Up to 21 years - NCT04528355Recruiting
Study of hospital data after stem cell transplant for non-cancer illness
This study collects medical information from people with certain non-cancer conditions who receive a stem cell transplant. It may help doctors better understand transplant outcomes and care, since the study focuses on data collection rather than adding new treatment.
Pittsburgh, PennsylvaniaAges 2 months–60 years - NCT04558736RecruitingPhase 2
Haploidentical transplant for severe aplastic anemia in children
This trial tests a “half-matched” stem cell transplant for children and teens with severe aplastic anemia when a fully matched donor isn’t available. It may help rebuild blood-making cells after prior treatments did not work well enough.
Memphis, TennesseeAges Up to 21 years - NCT04356469RecruitingPhase 2
Donor stem cell transplant for serious blood disorders in children
This trial tests a special type of donor stem cell transplant (with donor T-cells removed) to treat serious non-cancer blood problems in children. It may help by resetting the bone marrow so the body can make healthy blood cells again.
St. Petersburg, FloridaAges birth–21 years - NCT03579875RecruitingPhase 2
Testing an inherited bone marrow transplant approach for rare blood disorders
This Phase 2 study tests a special “T-cell depleted” transplant plan for people with inherited bone marrow failure disorders (Fanconi anemia or T-Beta-thalassemia—or “TBD”). It may help restore healthy blood production while lowering the chance of certain transplant complications.
Minneapolis, MinnesotaAges Up to 65 years - NCT03314974RecruitingPhase 2
Stem cell transplant study for blood cancers
This Phase 2 study tests a strong (myeloablative) stem cell transplant using donor stem cells (allogeneic transplant) for certain blood and bone marrow cancers. The goal is to see how well this approach works and whether it can improve outcomes for people who need transplant-level treatment.
Minneapolis, MinnesotaAges Up to 60 years - NCT03128996RecruitingPhase 1/Phase 2
Bone marrow transplant with lighter chemo for non-cancer illnesses
This early-phase study tests a safer “lighter” conditioning chemo plan before a bone marrow transplant for people with non-cancer blood and immune disorders. It aims to reduce transplant risk while using donor marrow that does not match perfectly in the HLA type.
New Haven, ConnecticutAges 1 day–21 years - NCT02720679Recruiting
Study genetics behind blood disorders in children and families
This study looks at genetic (DNA) factors that may contribute to certain blood disorders. You may be invited if you or a close family member is receiving care for a blood condition like MDS or MPN, and the study also includes related relatives.
Memphis, TennesseeAges Any age - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years - NCT01174108RecruitingPhase 2
Stem cell transplant with donor T-cells for severe bone marrow failure
This Phase 2 study tests a specific type of donor stem cell transplant for severe bone marrow failure conditions, aiming to improve cure rates while reducing transplant risks. It includes people with diseases like severe aplastic anemia and certain bone marrow disorders, especially when standard transplant plans feel too risky.
Baltimore, MarylandAges 4–80 - NCT06398457RecruitingEarly Phase 1
Darzalex Before Stem Cell Transplant for High-Risk Graft Failure
This trial tests giving a drug called Darzalex Faspro before the usual desensitization and a stem cell transplant for people at high risk of graft failure due to donor-specific antibodies. The goal is to prevent the transplant from failing.
Baltimore, MarylandAges 18 years+ - NCT03896269RecruitingPhase 1
CPX-351 for high-risk MDS or CMML that came back
This Phase 1 trial studies CPX-351, a cancer medicine, in people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) whose disease relapsed or did not respond to prior treatment. It may help shrink or control the leukemia and improve blood counts in patients who cannot or do not want a stem cell transplant.
Houston, TexasAges 18 years+ - NCT03384108Recruiting
Study of how certain immune cells use a nutrient
This study looks at how bone marrow plasma cells (a type of immune cell) use the nutrient glutamine. It may help researchers better understand what fuels these cells, which could guide future treatments.
Rochester, MinnesotaAges 18–60 - NCT03670966RecruitingPhase 1/Phase 2
Targeted iodine therapy, then donor transplant for hard leukemias
This trial tests an antibody-linked radioactive iodine treatment, followed by a donor stem cell transplant, for people with relapsed or refractory high-risk acute leukemia or certain MDS. It may help patients by targeting leukemia cells and then using a new immune system to control the disease.
Seattle, WashingtonAges 18–75
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Common questions
- Are there clinical trials for bone marrow failure?
- Yes. Clin2 currently lists 148 recruiting bone marrow failure studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a bone marrow failure trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a bone marrow failure trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.