Trials / Completed
CompletedNCT02064569
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
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
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 19 (actual)
- Sponsor
- GenSight Biologics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | GS010 |
Timeline
- Start date
- 2014-02-13
- Primary completion
- 2020-06-25
- Completion
- 2020-06-25
- First posted
- 2014-02-17
- Last updated
- 2026-03-02
Locations
1 site across 1 country: France
Source: ClinicalTrials.gov record NCT02064569. Inclusion in this directory is not an endorsement.