Clinical Trial: Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial

Brief Summary:

Hypotheses:

The primary hypothesis being tested is that there will be no toxicity resulting in loss of vision to no light perception in injected eyes.


Detailed Summary:
Sponsor: John Guy

Current Primary Outcome: Assessment of Primary Endpoint - Toxicity [ Time Frame: 1 year ]

Incidence of local and general adverse events and Serious Adverse Events


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University of Miami

Dates:
Date Received: June 6, 2014
Date Started: July 2014
Date Completion: March 2019
Last Updated: January 10, 2017
Last Verified: January 2017