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Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Sponsor: Nantes University Hospital
Summary
The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.
Official title: Prospective Monocentric Open Label Non Randomized Uncontrolled Phase I/II Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Key Details
Gender
All
Age Range
6 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
9
Start Date
2011-09
Completion Date
2014-08
Last Updated
2026-09-14
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (1)
CHU Nantes
Nantes, France