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CTX001 in transfusion-dependent β-thalassemia
NCT03655678Phase 3Completed
- Sponsor
- Vertex Pharmaceuticals
- Start
- Sep 2018
- Primary completion
- Nov 2025
- Modality
- Gene Silencing, CRISPR-Cas9, Cell Therapies
- Countries
- 5
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in beta thalassemia, sponsored by Vertex Pharmaceuticals. Primary endpoints: Transfusion Independence for at least 12 consecutive months at 24 months, Neutrophil Engraftment at 42 days and 4 more. Started Sep 2018; status: completed.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemia
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