This trial is active, not recruiting.

Condition retinal degeneration
Treatment tgaag76 (raav 2/2.hrpe65p.hrpe65)
Phase phase 1/phase 2
Sponsor University College, London
Collaborator Moorfields Eye Hospital NHS Foundation Trust
Start date January 2007
End date March 2014
Trial size 12 participants
Trial identifier NCT00643747, ALIR1015


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Injection of vector
tgaag76 (raav 2/2.hrpe65p.hrpe65) rAAV 2/2.hRPE65p.hRPE65
Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Primary Outcomes

intraocular inflammation
time frame: at intervals up to 12 months

Secondary Outcomes

visual function
time frame: intervals up to 12 months

Eligibility Criteria

Male or female participants from 5 years up to 30 years old.

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

Additional Information

Official title 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
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.
Trial information was received from ClinicalTrials.gov and was last updated in December 2013.
Information provided to ClinicalTrials.gov by University College, London.