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Exa-cel (CTX001) in Severe Sickle Cell Disease
NCT03745287Phase 3Completed
- Sponsor
- Vertex Pharmaceuticals
- Start
- Nov 2018
- Primary completion
- Jul 2025
- Modality
- Gene Silencing, CRISPR-Cas9, Cell Therapies
- Countries
- 7
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoints: Percentage of Participants Free From Severe Vaso-occlusive Crisis for at Least 12 Consecutive Months at 2 years, Percentage of Participants Who Achieve Neutrophil Engraftment at 24 months and 6 more. Started Nov 2018; status: completed.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)
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