Pediatric study of fludarabine, abatacept, and sirolimus immunosuppression for for high-risk thalassemia patients

The research company The Hospital for Sick Children is conducting the clinical trial Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia.

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.

It is planned to include 20 participants.

Actual study start date is March 22, 2022. The researchers expect to complete the study by December 31, 2026.

One primary outcome measure is Immune reconstitution, Rate of immune reconstitution defined by recovery of CD4 cells post bone marrow transplantation.

The location of the study is as follows (further details can be found here https://ichgcp.net/clinical-trials-registry/NCT05426252 or https://clinicaltrials.gov/ct2/show/NCT05426252.

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