Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
Phase 1
Withdrawn
- Conditions
- Complex and Transfusion-dependent HemoglobinopathiesAlpha-ThalassemiaAnemia, Sickle CellBeta-ThalassemiaThalassemiaDiamond-Blackfan AnemiaBone Marrow Failure Syndromes
- Interventions
- Biological: Enriched Hematopoetic Stem Cell Infusion
- Registration Number
- NCT01419704
- Lead Sponsor
- Talaris Therapeutics Inc.
- Brief Summary
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Detailed Description
This proposal is a phase I/II feasibility study to demonstrate that mixed chimerism can be established with minimal risk in recipients with hemoglobinopathies treated with Campath-1H-based nonmyeloablative conditioning and graft engineering to reduce the risk of Graft Versus Host Disease (GVHD), but preserve engraftment of donor cells.
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Hemoglobinopathies diagnosed patients Enriched Hematopoetic Stem Cell Infusion Recipients diagnosed with Hemoglobinopathies are treated with an enriched hematopoetic stem cell infusion from living donor bone marrow
- Primary Outcome Measures
Name Time Method Proportion of Hemoglobin A and S one month to three years Red blood cell contents by hemoglobin electrophoresis
- Secondary Outcome Measures
Name Time Method Enriched Hematopoetic Stem Cell Engraftment One month to three years
Trial Locations
- Locations (3)
University of Louisville
🇺🇸Louisville, Kentucky, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States