Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 (RPE65)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
|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|
- 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 ] [ Designated as safety issue: Yes ]Biodistribution : Urine sampling and nasal secretion will be collected at several time points after administration of the gene therapy product during all the duration of hospital stay, an average of 7 days.
- 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 ] [ Designated as safety issue: No ]
Recording global ERG (electroretinogram)
Patient efficacy questionnaire
Testing of far and near visual acuity, color vision, pupillometry, microperimetry and dark adaptation.
|Study Start Date:||September 2011|
|Study Completion Date:||August 2014|
|Primary Completion Date:||August 2014 (Final data collection date for primary outcome measure)|
3 cohortes of 3 patients each.
All the patients enrolled in the study will receive a single subretinal injection in one eye. The eye, that will be injected, will be the eye with the poorest visual acuity.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01496040
|Nantes, France, 44000|
|Principal Investigator:||Michel WEBER, Professor||CHU Nantes|