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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)

Comparing two treatments for severe aplastic anemia in young patients.

Recruiting
25 years and younger
All
Phase 3

Severe Aplastic Anemia (SAA) is a rare disease where the body stops making enough new blood cells. Treatments include Immune Suppressive Therapy (IST) and Bone Marrow Transplant (BMT). Usually, if a patient doesn't have a family donor, IST is given first. If IST fails, an Unrelated Donor (URD) BMT is used. This study will compare IST and URD BMT as first treatments for SAA, checking how long they work and how they affect health and fertility. The study involves medicines and procedures that are standard for SAA. It will run for about 3.3-4.7 years and require up to 2 years of participation, with follow-ups till 5 years.

  • Participants will be randomly assigned to IST or URD BMT.
  • Standard treatments, no experimental drugs involved.
  • Study lasts 3.3-4.7 years, with up to 2 years of active participation.
Study details
    Severe Aplastic Anemia

NCT05600426

Boston Children's Hospital

16 February 2025

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