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

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

NCT Number	NCT05600426
Lead Sponsor	Boston Children's Hospital Collaborators: Center for International Blood and Marrow Transplant Research, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), North American Pediatric Aplastic Anemia Consortium, Pediatric Transplantation and Cellular Therapy Consortium, Blood and Marrow Transplant Clinical Trials Network
Conditions	Severe Aplastic Anemia
Enrollment	53 participants
Start Date	2023-01-25
Primary Completion	2029-12 (estimated)
Study Completion	2029-12 (estimated)
Updated on ClinicalTrials.gov	2026-07-30