Clinical trials
Inherited Bone Marrow Failure Syndrome clinical trials
Below are recruiting inherited bone marrow failure syndrome clinical trials, each written for real people, not researchers. We’re tracking 21 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 - 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+ - 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 - 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - NCT07012044RecruitingPhase 1
Cedazuridine and decitabine after stem cell transplant for childhood leukemia
This trial tests if adding two chemotherapy drugs (cedazuridine and decitabine) along with a growth factor (filgrastim) can help prevent leukemia from coming back after a stem cell transplant. It is for children and young adults with high-risk acute myeloid leukemia who are in remission after transplant.
Birmingham, AlabamaAges Up to 21 years - NCT03980769RecruitingPhase 2
Stem cell transplant trial for non-cancer blood disorders
This Phase 2 study tests a specific chemotherapy combination (treosulfan, fludarabine, and thiotepa) before an unrelated or matched donor stem cell transplant. It may help people with certain non-cancer blood and immune disorders by trying to reset the immune system using donor stem cells.
Seattle, WashingtonAges Up to 50 years - NCT05757310RecruitingPhase 1
Stem cell transplant with lower-intensity chemo for severe aplastic anemia
This Phase 1 trial tests a “lower-intensity” chemotherapy plan followed by a stem cell transplant from a related half-match donor for severe aplastic anemia that has returned or did not respond to prior medicines. It aims to see if this approach is safe and works well enough to help your blood counts recover.
Duarte, CaliforniaAges Up to 75 years - NCT06466122RecruitingPhase 2
Pirtobrutinib plus Venetoclax for resistant CLL or SLL
This trial tests two oral targeted drugs—pirtobrutinib and venetoclax—for people with CLL or SLL whose disease has continued to grow despite taking a BTK inhibitor (such as ibrutinib, acalabrutinib, or zanubrutinib). The goal is to see if this combination can control the cancer when a BTK inhibitor alone is no longer working.
Columbus, OhioAges 18 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 - NCT06517641RecruitingPhase 2
Early bone marrow transplant for severe aplastic anemia
This trial tests whether an early bone marrow transplant from a half-matched family member or an unrelated donor can help people with severe aplastic anemia. It aims to restore the body's ability to make healthy blood cells.
Birmingham, AlabamaAges 3–75 - 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
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Common questions
- Are there clinical trials for inherited bone marrow failure syndrome?
- Yes. Clin2 currently lists 21 recruiting inherited 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 inherited 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 inherited 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.