Bone Marrow Transplant for Severe Aplastic Anemia

This study is looking at bone marrow transplants as an early treatment for severe aplastic anemia (SAA). SAA is a condition where your body doesn't make enough blood cells. Researchers want to see if using a bone marrow transplant from a partially matched family member (haploidentical donor) or an unrelated donor, along with medicines like Antithymocyte Globulin (ATG), Fludarabine, and Cyclophosphamide, can help restore normal blood cell production. You might be able to join if you are between 3 and 75 years old and have been recently diagnosed with SAA. The main goal is to see how many participants are free from graft-versus-host disease (GVHD) and treatment failure one year after the transplant. The current recruitment status is unclear.

Study design
This is a multicenter study involving 60 participants. It is an interventional study, meaning participants will receive a specific treatment.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for at least one year after the start of their treatment to assess outcomes.

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NCT06517641

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

Recruiting
PHASE2Ages 3–75InterventionalTreatment
Medical College of Wisconsin
~60 participants
Updated 2026-02-17 on ClinicalTrials.gov
What's tested:Haploidentical donor bone marrow transplantUnrelated donor bone marrow transplant

At a glance

Recruiting sites
25 of 25 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Graft versus host disease (GVHD)-free failure-free survival (GFFS) at One year
Measured over 1 year after initiation of conditioning
Severe Aplastic Anemia
25 sites across 19 states
California3
North Carolina3
Georgia2
Massachusetts2
Alabama1
Florida1
Kansas1
Maryland1

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Eligibility criteria

Exclusion

Prior infections must be controlled
  • Graft versus host disease (GVHD)-free failure-free survival (GFFS) at One year1 year after initiation of conditioning

    The primary endpoint is GFFS at 1 year after initiation of conditioning. Events for GFFS include Grade III-IV aGVHD, cGVHD requiring immunosuppression, primary or secondary graft failure requiring second definitive therapy, failure to receive an HSCT infusion, and death. GFFS is defined as the time interval from start of conditioning until the first of these events occurs. For failure to receive an HSCT infusion, the date will be at the time the decision not to proceed to HSCT is made.