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Gene Therapy for Alpha 1- Antitrypsin Deficiency
NCT06996756Phase 1Recruiting
- Sponsor
- Weill Cornell Medicine
- Start
- Feb 2025
- Primary completion
- Apr 2028
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label trial of AAV8hAAT(AVL) in alpha-1 antitrypsin deficiency (AATD) (4 arms), sponsored by Weill Cornell Medicine. Primary endpoints: Serious Adverse Events at 1 year, Dose Limiting Toxicities at 2 years and 1 more. Started Feb 2025; status: recruiting.
02
Drugs
AAV8hAAT(AVL)
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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