Clinical Trial: Safety Study in Subjects With Leber Congenital Amaurosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 1 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment E

Brief Summary: The purpose of this study is to determine whether gene transfer will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).

Detailed Summary: Leber Congenital Amaurosis (LCA)is a severe early onset retinal degeneration. Diagnosis is usually made during the first few months of life in infants who present with severely impaired vision, abnormal eye movements (nystagmus) and abnormal electroretinograms (ERG) indicating decreased retinal function. There is an inevitable progression to total blindness in these individuals due to death of photoreceptor cells. There is presently no treatment for this disease. The primary objective of this study is to determine the safety and tolerability of subretinal administration of AAV2-hRPE65v2 to subjects with Leber congenital amaurosis due to RPE65 mutations. The secondary objective is to assess the objective clinical measures of efficacy in human subjects.
Sponsor: Spark Therapeutics

Current Primary Outcome: The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests. [ Time Frame: Visual function will be measured at designated intervals from baseline visits through 5 years as stated in the protocol. ]

Original Primary Outcome: The Primary outcome measure(s) will be change in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests. [ Time Frame: Visual function will be measured at designated intervals from baseline visits through 5 years as stated in the protocol. ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Spark Therapeutics

Dates:
Date Received: August 13, 2007
Date Started: September 2007
Date Completion: July 2024
Last Updated: March 24, 2017
Last Verified: March 2017