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Showing 25–25 of 1 matching trial from the live ClinicalTrials.gov search.
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Filters: Condition: Leber's Congenital Amaurosis Clear all
Completed Phase 1 Interventional

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

NCT00481546
View directory record Official record
Conditions
Amaurosis of Leber, Retinal Diseases
Interventions
rAAV2-CBSB-hRPE65
Genetic
Lead sponsor
University of Pennsylvania
Other
Eligibility
8 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
2
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 21, 2026, 6:51 PM EDT