Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
Public ClinicalTrials.gov record NCT03293524. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected With G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
Study identification
- NCT ID
- NCT03293524
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 98 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 15 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 11, 2018
- Primary completion
- Jul 22, 2024
- Completion
- Jul 22, 2024
- Last update posted
- Apr 15, 2026
2018 – 2024
United States locations
- U.S. sites
- 7
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Doheny Eye Center UCLA Pasadena | Pasadena | California | 91105 | — |
| University of Colorado Health Eye Center | Aurora | Colorado | 80045 | — |
| Emory Healthcare - The Emory Clinic | Atlanta | Georgia | 30322 | — |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | — |
| Department of Ophthalmology, Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | — |
| Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | — |
| Vanderbilt Eye Institute | Nashville | Tennessee | 37232 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 6 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03293524, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 15, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03293524 live on ClinicalTrials.gov.