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A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Trial comparing bone marrow transplant and immunosuppressive therapy for severe aplastic anemia.

Recruiting
25 years and younger
All
Phase 3
  • Simplified description (AI rewritten)
  • Technical description (Original)

Severe Aplastic Anemia (SAA) is when the body doesn't make enough new blood cells. It can be treated with immune suppressive therapy (IST) or a bone marrow transplant (BMT). This study compares these treatments in kids and young adults without a family donor. The study will see which treatment helps patients live longer without their disease coming back or getting worse. It will also check how these treatments affect quality of life and fertility and look at certain genes related to SAA.

Participants will be randomly assigned to IST or unrelated donor BMT. This study does not use experimental drugs. The treatments are standard for SAA. Participants will be involved for up to 2 years, with a follow-up between 3 and 5 years.

  • Duration: Participation lasts up to 2 years, with a follow-up between 3 and 5 years.
  • Treatments: Participants receive standard therapies, not experimental drugs.
  • Eligibility: Open to individuals under 25 years with SAA and no family donor.
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Study details
    Severe Aplastic Anemia

NCT05600426

Boston Children's Hospital

11 May 2025

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