Clinical trials
Congenital Bone Marrow Failure Syndromes clinical trials
Below are recruiting congenital bone marrow failure syndromes clinical trials, each written for real people, not researchers. We’re tracking 18 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 - 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 - 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 - 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 - 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 - 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 - NCT07605416RecruitingPhase 2
Testing New Drug Combination for Plasma Cell Leukemia
This study tests a new combination of medicines to treat plasma cell leukemia, a rare blood cancer. The trial aims to help patients newly diagnosed with this condition by using drugs that work against cancer cells in different ways.
Chicago, IllinoisAges 18–80 - NCT05886049RecruitingPhase 1
Testing a menin drug with standard chemo for newly diagnosed AML
This early-phase study tests a menin inhibitor drug (SNDX-5613) combined with standard induction chemotherapy (daunorubicin plus cytarabine) in newly diagnosed AML with specific gene changes (NPM1 or FLT3 status, or MLL/KMT2A or NUP98). It may help patients whose AML has these genetic features respond better to intensive treatment.
Orange, CaliforniaAges 18–75 - NCT07040982RecruitingPhase 1
Asciminib after cell therapy for Ph+ ALL
This trial tests the drug asciminib after a stem cell transplant or CAR T-cell therapy for adults with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) to help prevent the cancer from coming back.
Duarte, CaliforniaAges 18 years+ - NCT06572605RecruitingPhase 1/Phase 2
Radiation plus talquetamab for myeloma outside the bone
This trial tests combining radiation therapy with a targeted drug called talquetamab for multiple myeloma that has formed tumors outside the bone (extramedullary disease). It aims to see if this combination can shrink or control these tumors and improve outcomes.
Duarte, CaliforniaAges 18 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 - NCT04047641RecruitingPhase 1/Phase 2
Quizartinib with chemotherapy for new or returning leukemia
This trial tests a combination of anti-leukemia drugs (including quizartinib) together with chemotherapy for people with acute myeloid leukemia (AML), a rare mixed-type leukemia, or high-risk myelodysplastic syndrome (MDS). It may help by trying to better control cancer in people whose disease is newly diagnosed or has returned/not responded to prior treatment.
Houston, TexasAges 18 years+ - NCT05343572RecruitingEarly Phase 1
Stem cell treatment to improve abnormal uterine lining
This early-phase study tests whether mobilized stem cells from bone marrow can help women with certain abnormal uterine lining problems become healthier and possibly improve fertility. It’s aimed at people who are not pregnant and meet specific diagnoses and test results.
Orange, ConnecticutAges 18–40 - NCT06013423RecruitingPhase 2
Cord blood transplant with chemo and radiation for high-risk blood cancers
This trial tests a cord blood transplant after a reduced-intensity chemotherapy and radiation regimen for patients with high-risk blood cancers like leukemia, lymphoma, or MDS. The goal is to see if this approach can help destroy cancer cells and restore healthy blood cells.
Seattle, WashingtonAges 6 months–65 years - NCT06622005RecruitingPhase 1
Combination treatment for multiple myeloma that has returned
This trial tests a new drug (SX-682) added to a standard three-drug combination (carfilzomib, daratumumab, and dexamethasone) for people whose multiple myeloma has come back or stopped responding to previous treatment. The goal is to see if adding this drug helps control the cancer better.
Buffalo, New YorkAges 18 years+
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Common questions
- Are there clinical trials for congenital bone marrow failure syndromes?
- Yes. Clin2 currently lists 18 recruiting congenital bone marrow failure syndromes 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 congenital bone marrow failure syndromes 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 congenital bone marrow failure syndromes 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.