Status:
COMPLETED
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Lead Sponsor:
University College, London
Collaborating Sponsors:
Moorfields Eye Hospital NHS Foundation Trust
Targeted Genetics Corporation
Conditions:
Retinal Degeneration
Eligibility:
All Genders
5-30 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.
Detailed Description
The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different d...
Eligibility Criteria
Inclusion
- Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65
Exclusion
- Visual acuity in the study eye better than 6/36 Snellen
- Hypertension
- Diabetes mellitus
- Tuberculosis
- Renal impairment
- Immunocompromise
- Osteoporosis
- Gastric ulceration
- Severe affective disorder)
- Pregnancy or lactation
Key Trial Info
Start Date :
January 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2014
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00643747
Start Date
January 1 2007
End Date
December 1 2014
Last Update
December 7 2015
Active Locations (1)
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1
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD