Status:
COMPLETED
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Lead Sponsor:
Nantes University Hospital
Conditions:
Leber Congenital Amaurosis
Eligibility:
All Genders
6-50 years
Phase:
PHASE1
PHASE2
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 ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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........
Key Trial Info
Start Date :
September 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2014
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT01496040
Start Date
September 1 2011
End Date
August 1 2014
Last Update
October 7 2015
Active Locations (1)
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1
CHU Nantes
Nantes, France, 44000