NCT00643747CompletedPhase 1
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration
University College, London1 site in 1 country12 target enrollmentStarted: January 1, 2007Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- University College, London
- Enrollment
- 12
- Locations
- 1
- Primary Endpoint
- intraocular inflammation
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 5 Years to 30 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
A
Experimental
Injection of vector
Intervention: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) (Biological)
Outcomes
Primary Outcomes
intraocular inflammation
Time Frame: at intervals up to 12 months
Secondary Outcomes
- visual function(intervals up to 12 months)
Investigators
Study Sites (1)
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