Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 (RPE65)
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|ClinicalTrials.gov Identifier: NCT01496040|
Recruitment Status : Completed
First Posted : December 21, 2011
Last Update Posted : October 7, 2015
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|Condition or disease||Intervention/treatment||Phase|
|Leber Congenital Amaurosis||Drug: rAAV2/4.hRPE65||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (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|
|Study Start Date :||September 2011|
|Actual Primary Completion Date :||August 2014|
|Actual Study Completion Date :||August 2014|
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.
- 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 ]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 ]
Recording global ERG (electroretinogram)
Patient efficacy questionnaire
Testing of far and near visual acuity, color vision, pupillometry, microperimetry and dark adaptation.
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|Ages Eligible for Study:||6 Years to 50 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Mutations that code for abnormal RPE65 protein
- Presence of characteristic abnormalities in fundus
- Dramatic reduction of both rods ans cones ERG responses
- Low visual acuity <0.32
- inform consent signed
- Patients with chronic conditions such a haematological, cardiac, renal diseases
- Patients with, within the past 6 months, a clinically significant cardiac disease or known congestive heart failure, cardiac rhytm and conduction abnormalities
- Patients with pulmonaty dysfunction
- Patients with suspected rheumatoid arthritis
- Patients with current systemic infection........
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01496040
|Nantes, France, 44000|
|Principal Investigator:||Michel WEBER, Professor||CHU Nantes|
|Responsible Party:||Nantes University Hospital|
|Other Study ID Numbers:||
2011-000418-21 ( EudraCT Number )
|First Posted:||December 21, 2011 Key Record Dates|
|Last Update Posted:||October 7, 2015|
|Last Verified:||October 2015|
Leber Congenital Amaurosis
Nervous System Diseases
Eye Diseases, Hereditary