Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
NCT02064569 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 19
Last updated 2026-03-02
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.
Conditions
- Leber Hereditary Optic Neuropathy
Interventions
- GENETIC
-
GS010
Sponsors & Collaborators
-
GenSight Biologics
lead INDUSTRY
Principal Investigators
-
CATHERINE J. VIGNAL, MD · CIC CHNO DES QUINZE VINGTS
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2014-02-13
- Primary Completion
- 2020-06-25
- Completion
- 2020-06-25
Countries
- France
Study Locations
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