CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 12 target
Drug / intervention
voretigene neparvovec-rzylbiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00516477
NCT00516477Phase 1Completed
CompareCT.gov

A Phase 1 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-101]

Spark Therapeutics, Inc.·interventional·Posted Aug 15, 2007·Updated Nov 2, 2020

In Brief

A Phase 1 clinical trial evaluating voretigene neparvovec-rzyl for Leber Congenital Amaurosis. Completed, enrolled 12 participants across 1 site.

Detailed Summary

The purpose of this study is to determine whether gene transfer will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLeber Congenital Amaurosis
CountriesUnited States
Collaborators--

Timeline

Phase 1CompletedFinished
20082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedAug 15, 2007
Enrollment StartSep 1, 2007
Primary CompletionMar 20, 2018
TodayJul 2, 2026
Enrollment to primary: 10.6 yearsPosted 18.9 years ago

Interventions

voretigene neparvovec-rzylbiological

Subjects will be dosed unilaterally (one eye) beginning with the lowest dose. Subjects will be injected with AAV2-hRPE65v2 by means of a subretinal injection. Dose escalation to the next cohort will be dependent on assessment of the safety data by the DSMB out to at least 4 weeks following the injection. Because there is a delay between time of delivery of AAV2 and the peak transgene expression there will be a delay of six weeks between all subjects.