TransIT Protocol: 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
Study ID: STU-2022-0964
This study is designed as a Phase III RCT comparing unrelated donor BMT with IST for newly diagnosed pediatric and young adult patients (less than 25 years old) with SAA. The design used for this study is unblinded, stratified, randomized phase III study design. The study is unblinded because one arm is a transplant arm
Primary: To compare time from randomization to treatment failure or death from any cause of IST versus BMT. Failure of IST is defined as initiation of a second definitive therapy (BMT, second course of ATG) and failure of BMT is defined as initiation of a second definitive therapy (second BMT, course of ATG).