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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-000418-21 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rAAV2/4.hRPE65 | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rAAV2/4.hRPE65 | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| The drug safety evaluation after administration | 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. | After administration of the gene therapy product.The patient will be folloed for the duration of the hospital stay, an average of 7 days |
| Measure | Description | Time Frame |
|---|---|---|
| Different efficacy parameters and immune parameters have to be measured to conclude on the overall amelioration of quality of life of enrolled patients | Recording global ERG (electroretinogram) Patient efficacy questionnaire Testing of far and near visual acuity, color vision, pupillometry, microperimetry and dark adaptation. | Between Day -120 and Day-7, Day 5, Day 14, Day 30 Day 60, Day 90, Day 120, Day 180, Day 360 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michel WEBER, Professor | Nantes University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU Nantes | Nantes | 44000 | France |
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| ID | Term |
|---|---|
| D057130 | Leber Congenital Amaurosis |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012164 | Retinal Diseases |
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