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NCT03293524 · ClinicalTrials.gov registry record · Phase 3
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
A Phase 3 study, sponsored by GenSight Biologics.
- Completed
- Registry status
- Phase 3
- Development phase
- 98
- Enrollment target
NCT03293524: Completed Phase 3 study, sponsored by GenSight Biologics.
NCT03293524 is a Phase 3 study that has completed, run by GenSight Biologics. The registered enrollment target is 98 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (87% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03293524, a Phase 3 study, has completed, sponsored by GenSight Biologics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 98 participants
- Enrollment target
Study Summary
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.
Primary Outcome
The primary efficacy endpoint was the change from baseline of BCVA reported with LogMAR at 1.5-year post-treatment, in the second-affected/not-yet-affected eyes of ND4 LHON patients with vision loss up to one year. LogMAR BCVA was used to represent BCVA.
Interventions
- DRUG Placebo
- GENETIC GS010
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 98 participants |
| Start Date | 2018-03-12 |
| Est. Completion | 2024-07-23 |
| Phase | Phase 3 |
What the finished NCT03293524 record still lists
NCT03293524 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 98 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (87% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT03293524 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03293524 about?
NCT03293524 is a clinical study titled "Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year". 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.
What is the current status of trial NCT03293524?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 98 participants. The study started on 2018-03-12. Estimated completion is 2024-07-23.
What interventions are being tested in trial NCT03293524?
The interventions under investigation include: Placebo (DRUG), GS010 (GENETIC).
Who is sponsoring clinical trial NCT03293524?
This trial is sponsored by GenSight Biologics, which has 6 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03293524's enrollment target sits among peer trials
98 1714th of 2000 higher than 283 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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