Clinical Trial: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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
Brief 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.
Detailed Summary:
Sponsor: Nantes University Hospital
Current Primary Outcome: The drug safety evaluation after administration [ Time Frame: After administration of the gene therapy product.The patient will be folloed for the duration of the hospital stay, an average of 7 days ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Different efficacy parameters and immune parameters have to be measured to conclude on the overall amelioration of quality of life of enrolled patients [ Time Frame: Between Day -120 and Day-7, Day 5, Day 14, Day 30 Day 60, Day 90, Day 120, Day 180, Day 360 ]
Recording global ERG (electroretinogram)
Patient efficacy questionnaire
Testing of far and near visual acuity, color vision, pupillometry, microperimetry and dark adaptation.
Original Secondary Outcome: Same as current
Information By: Nantes University Hospital
Dates:
Date Received: November 24, 2011
Date Started: September 2011
Date Completion:
Last Updated: October 6, 2015
Last Verified: October 2015
