Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
This trial is active, not recruiting.
|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.
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Injection of vector
time frame: at intervals up to 12 months
time frame: intervals up to 12 months
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
|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.|
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