NCT05600426

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

A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Boston Children's Hospital · Academic / Other
• Sex: All
• Age: 0 Years – 25 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This study is a multi-center randomized phase III trial to compare the failure free survival between those randomized to IST vs 9-10/10 HLA matched URD BMT. The study will also address patient-reported outcomes and gonadal function in each arm and explore critical biological correlates including assessing germline genetic mutations associated with pediatric SAA that may lead to a predisposition to the disease and the risk of development of clonal hematopoiesis following IST vs BMT in pediatric and young adult SAA. This clinical trial will randomize 234 children/AYA over 3.3-4.7 years at a 1:1 ratio between initial treatment with immune suppression therapy (IST) with horse ATG (hATG)/cyclosporine (CsA) versus well- matched (9-10/10 allele) unrelated donor (URD) bone marrow transplantation (BMT) using a regimen of rabbit ATG (rATG)/fludarabine/cyclophosphamide and 200 cGy TBI. Duration of subject participation for all study procedures in this study will be up to 2 years after treatment; a single later timepoint between 3 and 5 years will be collected to follow patients for specific protocol defined late effects and survival.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	cyclosporine	cyclosporine
PROCEDURE	Matched Unrelated Donor Hematopoetic Stem Cell Transplant	Matched Unrelated Donor (MUD) Hematopoietic Stem Cell Transplantation (HSCT)
DRUG	horse anti-thymocyte globulin (ATG)	horse anti-thymocyte globulin (ATG)
DRUG	rabbit anti-thymocyte globulin (ATG)	rabbit anti-thymocyte globulin (ATG)
DRUG	Methotrexate	methotrexate
DRUG	Fludarabine	fludarabine
DRUG	Cyclophosphamide	cyclophosphamide
RADIATION	low-dose total body irradiation (TBI)	low-dose total body irradiation (TBI)
PROCEDURE	Immunosuppressive Therapy (IST)	Immunosuppressive Therapy (IST)

Timeline

• Start date: 2023-01-25
• Primary completion: 2029-12-01
• Completion: 2029-12-01
• First posted: 2022-10-31
• Last updated: 2026-02-17

Locations

52 sites across 2 countries: United States, Canada