voretigene neparvovec
voretigene neparvovec: 2 clinical trials tracked.
voretigene neparvovec evidence translation
What we can confirm
2 tracked trials
What it means
What to watch
Prepared research question
What is verified about voretigene neparvovec, and what remains unknown?
Traceable citations · Unknowns marked
See linked entities and source evidence1 indications · 1 study sponsors
Related indications
Study sponsors
Clinical Trial Registry Records (2)
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
Primary endpoint: Change from Baseline in full-field light sensitivity threshold
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 Study
Primary endpoint: Adverse events as a measure of safety and tolerability