Overview

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

Status:
Not yet recruiting

Trial end date:
2029-12-01

Target enrollment:

Participant gender:

Summary

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.

Phase:

Phase 3

Details

Lead Sponsor:

Boston Children's Hospital

Collaborators:

Blood and Marrow Transplant Clinical Trials Network
Center for International Blood and Marrow Transplant Research
National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health (NIH)
North American Pediatric Aplastic Anemia Consortium
Pediatric Transplantation and Cellular Therapy Consortium

Treatments:

Antilymphocyte Serum
Cyclophosphamide
Cyclosporine
Cyclosporins
Fludarabine
Immunosuppressive Agents
Methotrexate
Thymoglobulin