Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
NCT ID: NCT01496040
Last Updated: 2015-10-07
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2011-09-30
2014-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
NONE
Study Groups
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rAAV2/4.hRPE65
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.
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* Presence of characteristic abnormalities in fundus
* Dramatic reduction of both rods ans cones ERG responses
* Low visual acuity \<0.32
* inform consent signed
Exclusion Criteria
* 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........
6 Years
50 Years
ALL
No
Sponsors
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Nantes University Hospital
OTHER
Responsible Party
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Principal Investigators
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Michel WEBER, Professor
Role: PRINCIPAL_INVESTIGATOR
Nantes University Hospital
Locations
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CHU Nantes
Nantes, , France
Countries
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Other Identifiers
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2011-000418-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
BRD 07/08-K
Identifier Type: -
Identifier Source: org_study_id
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