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 109 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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 - 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 - 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 - NCT07102849Enrolling by invitation
Studying bone marrow failure with biospecimens and data
This study analyzes blood, bone marrow, and medical information from people with bone marrow failure syndromes (BMFS) and healthy volunteers to better understand these conditions and find new ways to help patients.
Bethesda, MarylandAges 2–120 - 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 - 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 - NCT06940297RecruitingPhase 2
Dasatinib and quercetin with Carvykti for advanced multiple myeloma
This trial tests whether adding two drugs (dasatinib and quercetin) before CAR-T therapy (Carvykti) can improve outcomes for people with multiple myeloma that has come back or not responded to at least three prior treatments. It aims to see if this combination is safe and effective.
Rochester, MinnesotaAges 18 years+ - NCT06172296RecruitingPhase 3
Dinutuximab plus transplant for high-risk neuroblastoma
This trial tests adding a targeted drug called dinutuximab to standard chemotherapy, surgery, and a stem cell transplant for children and young adults with a new diagnosis of high-risk neuroblastoma. The goal is to see if this combination helps more patients stay in remission.
Birmingham, AlabamaAges Up to 30 years - NCT04269902RecruitingPhase 3
Early treatment check for high-risk CLL/SLL
This Phase 3 trial studies an early treatment approach for people with high-risk CLL or SLL who have not yet needed standard therapy. It aims to see if treating earlier can improve outcomes while keeping side effects manageable.
Birmingham, AlabamaAges 18 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 - 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+ - 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+ - NCT06675123RecruitingPhase 1
Pacritinib with a BTK inhibitor for mantle cell lymphoma
This trial tests a new combination of two targeted drugs (pacritinib plus a BTK inhibitor like ibrutinib) for people with mantle cell lymphoma that has come back or stopped responding to treatment. It may help if your current BTK inhibitor is no longer working well.
Duarte, CaliforniaAges 18 years+ - NCT03739814RecruitingPhase 2
Test inotuzumab, blinatumomab, and possibly ponatinib for CD22 ALL
This Phase 2 study tests a combination of medicines (inotuzumab ozogamicin and blinatumomab), with or without ponatinib, for people with CD22-positive B-cell acute lymphoblastic leukemia (ALL). It aims to improve remission and control leukemia cells, including in the bone marrow.
Birmingham, AlabamaAges 18 years+ - NCT04195633RecruitingPhase 2
Haploidentical donor stem cell transplant to treat blood cancers
This Phase 2 trial tests whether a specific stem cell transplant method (from a related “half-matched” donor) plus conditioning chemo and radiation can improve outcomes for several blood cancers. It may help people whose cancer is in remission but still considered high risk, or those who have had limited response after prior treatment.
Seattle, WashingtonAges 6 months+ - NCT04375631RecruitingPhase 1
Study of chemo plus donor stem cell transplant for blood cancers
This Phase 1 trial tests a specific chemotherapy plan followed by a reduced-intensity donor stem cell transplant for people with relapsed or treatment-resistant acute myeloid leukemia (AML), certain myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). It may help by using donor stem cells to rebuild the bone marrow after intensive cancer treatment, in people who are not responding well to standard therapies.
Seattle, WashingtonAges 18 years+ - NCT04546399RecruitingPhase 2
Blinatumomab with or without nivolumab for first relapse ALL
This study compares blinatumomab alone versus blinatumomab plus nivolumab in young people whose B-cell ALL has returned for the first time. It aims to see whether adding nivolumab improves control of the leukemia after relapse.
Birmingham, AlabamaAges 1–30 - NCT04797767RecruitingPhase 1/Phase 2
Venetoclax plus CLAG-M for high-risk AML
This trial studies venetoclax combined with CLAG-M chemotherapy to treat adults with high-risk acute myeloid leukemia (AML) or related high-grade blood cancers. It aims to improve control of the disease, especially in people whose AML is considered high risk.
Seattle, WashingtonAges 18 years+ - NCT04892277RecruitingPhase 1
CD19 CAR-T for adults with hard-to-treat B-cell cancers
This Phase 1 trial tests a lab-made immune therapy (CD19 CAR-T) for adults whose B-cell cancers have come back or did not respond to treatment. It may help your immune system better recognize and attack cancer cells, but it is early research, so benefits and risks are still being defined.
Rochester, MinnesotaAges 18 years+ - NCT04941716RecruitingPhase 2
Acalabrutinib plus venetoclax for hard-to-treat CLL/SLL
This Phase 2 trial studies whether combining acalabrutinib and venetoclax can shrink or control chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) that has come back or did not respond to prior treatment. It may help by attacking the cancer using two different medicines together.
Seattle, WashingtonAges 18 years+
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Common questions
- Are there clinical trials for bone marrow failure syndrome?
- Yes. Clin2 currently lists 109 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.