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Clinical Trial Summary

The purpose of this study is to evaluate a novel transplant strategy for the long-term benefit of patients with transfusion dependent high-risk thalassemia.


Clinical Trial Description

Patients with high-risk thalassemia meeting the eligibility criteria for this study will be entered sequentially until completion or closure of the study. The hypothesis is that a reduced-toxicity conditioning regimen combined with pre-transplant immunosuppression, followed by abatacept and sirolimus as graft-versus-host disease (GVHD) prophylaxis for allogeneic transplant with either Human Leukocyte Antigen (HLA)-matched sibling donors or haploidentical donors is feasible and safe and can be delivered with less toxicity, durable donor engraftment, and minimal GVHD. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05426252
Study type Interventional
Source The Hospital for Sick Children
Contact Yogi Chopra, MD
Phone +1 (416) 813-7654
Email yogi.chopra@sickkids.ca
Status Recruiting
Phase Phase 1/Phase 2
Start date March 22, 2022
Completion date December 31, 2026

See also
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