NCT00481546View on ClinicalTrials.gov

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

PHASE1Active, not recruitingINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

July 31, 2007

Primary Completion Date

June 30, 2026

Study Completion Date

June 30, 2026

Conditions
Amaurosis of LeberRetinal Diseases
Interventions
GENETIC

rAAV2-CBSB-hRPE65

One or two, uniocular, subretinal injections; relative doses: 0.3X (Cohort 1), 0.6X (Cohort 2), 0.45X (Cohort 3), 0.9X (Cohorts 4 and 5)

Trial Locations (2)

19104

Scheie Eye Institute, University of Pennsylvania, Philadelphia

32610

Shands Children's Hospital, University of Florida, Gainesville

All Listed Sponsors
collaborator

National Eye Institute (NEI)

NIH

lead

University of Pennsylvania

OTHER