Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

  • STATUS
    Recruiting
  • End date
    Dec 31, 2026
  • participants needed
    20
  • sponsor
    The Hospital for Sick Children
Updated on 15 July 2022

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.

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.

Details
Condition Thalassemia in Children
Treatment Abatacept, Sirolimus
Clinical Study IdentifierNCT05426252
SponsorThe Hospital for Sick Children
Last Modified on15 July 2022

Eligibility

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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