Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
NCT05426252 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20
Last updated 2023-12-19
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.
Conditions
- Thalassemia in Children
Interventions
- DRUG
-
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.
Sponsors & Collaborators
-
Thalassemia Foundation of Canada
collaborator UNKNOWN -
The Hospital for Sick Children
lead OTHER
Principal Investigators
-
Yogi Chopra, MD · The Hospital for Sick Children
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 1 Year
- Max Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-03-22
- Primary Completion
- 2025-12-31
- Completion
- 2026-12-31
Countries
- Canada
Study Locations
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