Clinical Trial: Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary: 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.
Sponsor: University College, London

Current Primary Outcome: intraocular inflammation [ Time Frame: at intervals up to 12 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome: visual function [ Time Frame: intervals up to 12 months ]

Original Secondary Outcome: Same as current

Information By: University College, London

Dates:
Date Received: March 20, 2008
Date Started: January 2007
Date Completion:
Last Updated: December 4, 2015
Last Verified: December 2013