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Clinical trial

An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

NCT04516369 is registered in ClinicalTrials.gov with Novartis Pharmaceuticals as lead sponsor.

NCT NCT04516369
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Lead sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Overall status
Completed
Start date
November 24, 2020
Completion date
May 19, 2026
Enrollment
4 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

biallelic rpe65 mutation-associated retinal dystrophy