NCT05426252

Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

Thal-FabS: Novel Transplant Strategy for High-risk Thalassemia Patients - a Phase I/II Trial of Early Fludarabine Followed by Abatacept and Sirolimus Immunosuppression

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: The Hospital for Sick Children · Academic / Other
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Thalassemia in Children

Interventions

Type	Name	Description
DRUG	Abatacept	Abatacept, co-stimulation blockade, to be given for GVHD prophylaxis in combination with sirolimus post allogeneic hematopoietic stem cell transplantation.
DRUG	Sirolimus	Sirolimus, mTOR inhibitor, to be given for GVHD prophylaxis in combination with abatacept post allogeneic hematopoietic stem cell transplantation.

Timeline

• Start date: 2022-03-22
• Primary completion: 2025-12-31
• Completion: 2026-12-31
• First posted: 2022-06-21
• Last updated: 2023-12-19

Locations

1 site across 1 country: Canada