Clinical Trial: Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

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

Official Title: An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial

Brief Summary: The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.

Detailed Summary:
Sponsor: GenSight Biologics

Current Primary Outcome: Incidence of local and general adverse events and Serious Adverse Events [ Time Frame: Up to 48 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: GenSight Biologics

Dates:
Date Received: February 13, 2014
Date Started: February 2014
Date Completion: June 2020
Last Updated: February 22, 2017
Last Verified: February 2017