Clinical trials
Bone Marrow Failure Syndrome clinical trials
Below are recruiting bone marrow failure syndrome clinical trials, each written for real people, not researchers. We’re tracking 107 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT00027274Recruiting
Cancer risk in inherited bone marrow failure syndromes
This study looks at why people with inherited bone marrow failure syndromes (rare genetic conditions affecting blood cell production) have higher cancer risk. Researchers want to understand the genetic and medical factors that increase cancer likelihood, which could help doctors monitor and protect affected patients and their families.
Bethesda, MarylandAges 1 day–100 years - NCT06287944RecruitingPhase 1
Targeted radiation and chemo before donor stem cell transplant
This trial tests a new approach: giving a targeted radioactive antibody (attached to a drug that finds CD38 on cancer cells) along with low-dose chemotherapy and targeted radiation to the bone marrow before a donor stem cell transplant. The goal is to prepare the body for the transplant while killing remaining cancer cells more precisely.
Duarte, CaliforniaAges 18–70 - NCT02958462Recruiting
Clinic study for early blood or bone marrow warning signs
This study follows people who may have early, unclear blood count problems or certain genetic/immune patterns that can later lead to bone marrow failure or blood cancers. It may help doctors better understand risks over time and guide earlier care.
Scottsdale, ArizonaAges 18 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 - NCT04232085RecruitingPhase 2
Stem cell transplant to rebuild immunity in genetic immune disorders
This Phase 2 trial tests a stem cell (bone marrow) transplant approach to restore bone marrow function and immune function in people with certain inherited immune problems. If you have one of these conditions and a suitable donor, the study may offer a path to rebuild blood and immune systems.
Baltimore, MarylandAges 4 months–50 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 - NCT04781790Recruiting
Registry for bone marrow failure patients
This study is a national registry that collects health information and some blood or tissue samples from people diagnosed with bone marrow failure. It helps researchers understand causes and long-term outcomes, and may improve care by building a large patient database.
ParisAges Any age - NCT05012111Recruiting
Study of bone marrow failure causes over time
This study looks at the natural history (how a condition progresses) in people with acquired or inherited bone marrow failure syndromes, including related lung and liver findings in some cases. It helps researchers understand underlying causes—sometimes linked to gene or telomere changes—and how symptoms evolve, which may guide future care.
Bethesda, MarylandAges 2–99 - NCT05196789Recruiting
Genetic testing to diagnose inherited bone marrow failure
This study looks at patients with suspected inherited (families-linked) bone marrow failure to better understand the cause using genetic testing. It may help confirm a diagnosis and refine how different inherited conditions are classified.
Melbourne, VictoriaAges 3 months+ - NCT05388006RecruitingPhase 2
Testing three medicines for Richter transformation in CLL/SLL
This Phase 2 trial studies acalabrutinib, venetoclax, and durvalumab together for Richter transformation (a more aggressive lymphoma) that developed from CLL or SLL. The goal is to improve how well treatment works and how long people can respond.
Palo Alto, CaliforniaAges 18 years+ - NCT05436587Recruiting
Genetic testing study for hard-to-classify bone marrow failure
This study looks for gene changes in families affected by rare inherited bone marrow failure that can cause fragile bones and fractures. It may help doctors understand the condition better by linking genetic mutations with family health patterns.
SohagAges Any age - 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 - NCT05711849RecruitingPhase 2
Stem cell treatment for long-lasting chest pain not helped by meds
This Phase 2 trial tests whether stem cells placed into the heart artery can safely reduce symptoms in people with “refractory” angina (chest pain) despite maximum heart-healthy medication. You may qualify if your chest pain is limiting your activity and other heart procedures (stents/bypass) aren’t possible or are too risky.
London, EnglandAges 18 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 - NCT06247787RecruitingPhase 1
Imetelstat plus chemo for relapsed leukemia in kids
This trial tests a new drug called imetelstat combined with two chemotherapy drugs (fludarabine and cytarabine) for children and teens with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) that has come back or not responded to treatment. The goal is to find the safest dose of imetelstat and see if it helps control the cancer.
Birmingham, AlabamaAges 1–18 - NCT06498973RecruitingPhase 1
Maintenance therapy after stem cell transplant for CD123-positive AML/MDS
This trial tests a combination of two drugs, tagraxofusp and azacitidine, given as maintenance therapy after a stem cell transplant. The goal is to prevent the leukemia or MDS from coming back in patients whose cancer cells carry a specific marker called CD123.
Duarte, CaliforniaAges 18–75 - NCT06785415RecruitingPhase 1/Phase 2
Study of a drug combo for relapsed multiple myeloma
This trial tests a combination of two antibody drugs (elotuzumab and daratumumab) plus an oral pill (iberdomide) and a steroid (dexamethasone) for people whose multiple myeloma has come back after treatment. The goal is to see if this new mix works better than what's already available.
Rochester, MinnesotaAges 18 years+ - 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 - 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 - NCT06928662RecruitingPhase 1/Phase 2
Chemo, radiation, and donor stem cell transplant for high-risk blood cancers
This trial tests a strong chemotherapy and radiation combo followed by a donor stem cell transplant for people with aggressive blood cancers (like AML or MDS) that haven't responded to earlier treatments or came back. It aims to help prevent the cancer from returning.
Seattle, WashingtonAges 18 years+ - 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 - NCT07046078RecruitingPhase 2
Chemo, radiation, and donor transplant for older adults with high-risk AML
This trial tests a combination of chemotherapy (FLAG-Ida) followed by low-dose total body radiation and a stem cell transplant from a donor. It is for people age 60 and older with newly diagnosed high-risk blood cancers, aiming to improve outcomes with a less intense transplant preparative regimen.
Seattle, WashingtonAges 18 years+ - NCT07228273RecruitingPhase 2
A new drug combo for AML and high-risk MDS
This trial combines three standard chemotherapy drugs (fludarabine, cytarabine, idarubicin) with a targeted therapy (venetoclax) to treat acute myeloid leukemia (AML) and a related condition called MDS with high blast counts. The goal is to see if adding venetoclax improves results.
Portland, OregonAges 18–65
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Common questions
- Are there clinical trials for bone marrow failure syndrome?
- Yes. Clin2 currently lists 107 recruiting bone marrow failure syndrome 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 syndrome 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 syndrome 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.