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 ]

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 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