Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

September 30, 2011

Primary Completion Date

August 31, 2014

Study Completion Date

August 31, 2014

Conditions
Leber Congenital Amaurosis
Interventions
DRUG

rAAV2/4.hRPE65

"One injection in on eye~Cohorte 1 : 3 patients will receive one injection of up to 400 microliters of the IMP~Cohorte 2 : 3 patients will receive one injection of up to 800 microliters of the IMP.~Cohorte 3 : 3 patients under age of eighteen will receive one injection up to 400 or 800 microliters of the IMP."

Trial Locations (1)

44000

CHU Nantes, Nantes

All Listed Sponsors
lead

Nantes University Hospital

OTHER

NCT01496040 - Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 | Biotech Hunter | Biotech Hunter