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Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease
NCT03282656Phase 1Active, not recruiting
- Sponsor
- David Williams
- Start
- Feb 2018
- Primary completion
- May 2026
- Modality
- Gene Editing, Viral-delivered RNAi, Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of BCL11a shRNAmiR in sickle cell disease (SCD), sponsored by David Williams. Primary endpoint: Rescue of hematopoiesis after conditioning. Started Feb 2018; status: active, not recruiting.
02
Drugs
BCL11a shRNAmiR
03
Conditions
sickle cell disease (SCD)
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