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Phase 3 Study of Voretigene Neparvovec in Leber Congenital Amaurosis
NCT00999609Phase 3Active, not recruiting
- Sponsor
- Spark Therapeutics
- Start
- Oct 2012
- Primary completion
- Jul 2015
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 3, randomised, open-label trial of voretigene neparvovec in Leber congenital amaurosis (LCA) and inherited retinal dystrophy (IRD) (2 arms), sponsored by Spark Therapeutics. Primary endpoint: Multi-luminance Mobility Testing at 1 year. Started Oct 2012; status: active, not recruiting.
02
Drugs
voretigene neparvovec
03
Conditions
Leber congenital amaurosis (LCA)inherited retinal dystrophy (IRD)
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