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AAV2-hRPE65v2,voretigene neparvovec-rzyl
BIOLOGICAL
2 trials
Sponsors
Spark Therapeutics, Inc.
Conditions
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Inherited Retinal Dystrophy Due to RPE65 Mutations
Leber Congenital Amaurosis
Phase 3
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Active, not recruiting
NCT00999609
Spark Therapeutics, Inc.
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Start: 2012-10-31
End: 2030-01-31
Updated: 2025-04-23
Unknown Phase
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Completed
NCT03597399
Spark Therapeutics, Inc.
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Start: 2019-01-10
End: 2025-06-30
Updated: 2025-09-22
Related Papers
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Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland.
2022-06-28
5 citations
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
2017-07-14
1370 citations