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 108 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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+ - 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 - NCT07557056RecruitingPhase 2
Oral combination treatment for untreated chronic lymphocytic leukemia
This clinical trial tests two oral medications, nemtabrutinib and venetoclax, given together to people with untreated CLL or SLL. The goal is to see if this combination effectively kills cancer cells and helps you stay in remission with manageable side effects.
Columbus, OhioAges 18 years+ - NCT07844694RecruitingPhase 1
Low-dose sirolimus for patients with RUNX1 platelet disorder
This trial tests whether a low dose of sirolimus, a drug that calms the immune system, can help improve blood counts and reduce bleeding risk in people with a genetic condition called RUNX1 familial platelet disorder. If it works, it might lower the chance of developing serious blood problems later.
Portland, OregonAges 18 years+ - NCT07734038RecruitingPhase 2
Venetoclax and Zanubrutinib for CLL and SLL
This study tests a combination of two oral medications, venetoclax and zanubrutinib, for people with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). It aims to see if this combination is safe and effective, either as a first treatment or after a previous treatment.
Columbus, OhioAges 18 years+ - NCT07685717RecruitingPhase 2
Pirtobrutinib for older adults with CLL
This trial tests a new pill, pirtobrutinib, for people 75 or older with chronic lymphocytic leukemia (CLL) who have never had treatment before. The goal is to see if it helps control the disease with manageable side effects.
Columbus, OhioAges 75 years+ - 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.
New Haven, ConnecticutAges 18–80 - NCT07548983RecruitingPhase 1
Maintenance therapy after stem cell transplant for AML
This trial tests whether taking two drugs, ruxolitinib and azacitidine, after a reduced-intensity stem cell transplant can help prevent acute myeloid leukemia (AML) from returning. It is for patients who are in remission and at high risk of relapse.
Portland, OregonAges 18 years+ - NCT07420959RecruitingPhase 1/Phase 2
ABBV-383 for Waldenström macroglobulinemia that came back
This trial tests a new drug called ABBV-383 for people with Waldenström macroglobulinemia that has returned or stopped responding to treatment. The drug is given as an infusion and aims to attack the cancer cells. This might help if you've already tried a BTK inhibitor.
Rochester, MinnesotaAges 18 years+ - NCT07293403RecruitingPhase 2
Duvelisib maintenance for peripheral T-cell lymphoma
This trial tests whether the drug duvelisib can help keep T-cell lymphoma in remission after initial chemotherapy. It is for people who have finished first-line treatment with a CHOP-like regimen, achieved a complete response, and cannot or choose not to have a stem cell transplant.
Duarte, CaliforniaAges 18 years+ - NCT07228624RecruitingPhase 2
Ruxolitinib before, during, and after transplant for myelofibrosis
This trial tests whether taking the drug ruxolitinib before, during, and after a stem cell transplant helps older patients with myelofibrosis or certain related blood disorders. The goal is to see if this approach can improve transplant success and reduce side effects.
Seattle, WashingtonAges 18–75 - NCT07225985RecruitingPhase 1/Phase 2
Pralatrexate, bendamustine, and radiation before donor stem cell transplant for T-cell lymphoma
This trial tests a combination of two chemotherapy drugs (pralatrexate and bendamustine) plus total-body radiation before a donor stem cell transplant for people with T-cell lymphoma that has come back or hasn't responded to treatment. The goal is to see if this approach can help destroy the lymphoma and allow the donor cells to take hold safely.
Seattle, WashingtonAges 18 years+ - NCT07181941RecruitingPhase 1/Phase 2
Testing lower doses of linvoseltamab for multiple myeloma
This trial tests if a lower dose of linvoseltamab can still work well for people with multiple myeloma that has come back or not responded to treatment. It aims to reduce side effects while keeping the cancer under control.
Seattle, WashingtonAges 18 years+ - 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+ - 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 - NCT06948786RecruitingPhase 2
Pirtobrutinib and Mosunetuzumab for relapsed follicular lymphoma
This trial tests two new drugs, pirtobrutinib and mosunetuzumab, for people with follicular lymphoma that has come back or not responded to earlier treatments. The goal is to see if this combination can help control the cancer.
Seattle, WashingtonAges 18 years+ - NCT06940297RecruitingPhase 2
Dasatinib and quercetin with CAR-T therapy for relapsed or refractory multiple myeloma
This trial tests whether adding two drugs, dasatinib and quercetin, to CAR-T therapy can help people with multiple myeloma that has come back or not responded to past treatments. It aims to make the CAR-T cells work better and reduce side effects.
Rochester, MinnesotaAges 18 years+ - 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
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Common questions
- Are there clinical trials for bone marrow failure syndrome?
- Yes. Clin2 currently lists 108 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.