This trial is active, not recruiting.

Condition leber hereditary optic neuropathy
Treatment gs010
Phase phase 1/phase 2
Sponsor GenSight Biologics
Start date February 2014
End date June 2020
Trial size 21 participants
Trial identifier NCT02064569, GS-LHON/CLIN/01


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Intervention model single group assignment
Primary purpose treatment
Masking no masking

Primary Outcomes

Incidence of local and general adverse events and Serious Adverse Events
time frame: Up to 48 weeks

Eligibility Criteria

All participants at least 18 years old.

Inclusion Criteria

    Exclusion Criteria

      Additional Information

      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 NADH Dehydrogenase 4 Gene
      Principal investigator CATHERINE VIGNAL, MD
      Trial information was received from ClinicalTrials.gov and was last updated in February 2017.
      Information provided to ClinicalTrials.gov by GenSight Biologics.