Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Phase 1
Completed
- Conditions
- Retinal Degeneration
- Interventions
- Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
- Registration Number
- NCT00643747
- Lead Sponsor
- University College, London
- 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 dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 12
Inclusion Criteria
- Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65
Exclusion Criteria
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description A tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) Injection of vector
- Primary Outcome Measures
Name Time Method intraocular inflammation at intervals up to 12 months
- Secondary Outcome Measures
Name Time Method visual function intervals up to 12 months
Trial Locations
- Locations (1)
Moorfields Eye Hospital NHS Foundation Trust
🇬🇧London, United Kingdom