Clinical Trial: Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With
Brief Summary: The study is a follow-on to a Phase 1 dose-escalation and safety study.
Detailed Summary: The study is a follow-on to a Phase 1 dose-escalation and safety study (closed to enrollment as of June 2009). Up to twelve adults and children with molecular diagnosis of LCA2, who have participated in the earlier Phase 1 study, and who meet all study eligibility criteria, will receive AAV2-hRPE65v2 vector in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of AAV2-hRPE65v2.
Sponsor: Spark Therapeutics
Current Primary Outcome: Adverse events as a measure of safety and tolerability [ Time Frame: 15 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Visual acuity [ Time Frame: 15 years ]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.
- Visual field [ Time Frame: 15 years ]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.
- Pupillary light response [ Time Frame: 15 years ]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.
- Mobility testing [ Time Frame: 15 years ]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.
- Full-field light threshold sensitivity testing [ Time Frame: 15 years ]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.
- Contrast sensitivity [ Time Frame: 15 years ]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.
Original Secondary Outcome: Same as current
Information By: Spark Therapeutics
Dates:
Date Received: September 22, 2010
Date Started: November 2010
Date Completion: November 2026
Last Updated: March 24, 2017
Last Verified: March 2017