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NCT03406104 · ClinicalTrials.gov registry record · Phase 3
RESCUE and REVERSE Long-term Follow-up
A Phase 3 study of Leber Hereditary Optic Neuropathy, sponsored by GenSight Biologics.
- Completed
- Registry status
- Phase 3
- Development phase
- 62
- Enrollment target
- 7
- Study locations
NCT03406104: Completed Phase 3 study of Leber Hereditary Optic Neuropathy, sponsored by GenSight Biologics.
NCT03406104 is a Phase 3 study of Leber Hereditary Optic Neuropathy that has completed, run by GenSight Biologics. The registered enrollment target is 62 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (92% lower). The trial reports 7 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03406104, a Phase 3 study of Leber Hereditary Optic Neuropathy, has completed, sponsored by GenSight Biologics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 62 participants
- Enrollment target
- 7
- Study locations
Study Summary
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.
Primary Outcome
Number of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment
Conditions Studied
Interventions
- GENETIC GS010
- OTHER Sham Intravitreal Injection
Study Locations (7)
Other
- CHNO Les Quinze Vingts - Paris
- LMU Klinikum der Universität München / Friedrich-Baur-Institut - Munich
- Ospedale Bellaria - Bologna
California
- Doheny Eye Center UCLA - Pasadena
Georgia
- Emory University Hospital - Atlanta
Pennsylvania
- Wills Eye Institute - Philadelphia
Greater London
- Moorfields Eye Hospital - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 62 participants |
| Start Date | 2018-01-09 |
| Est. Completion | 2022-07-04 |
| Phase | Phase 3 |
What the finished NCT03406104 record still lists
NCT03406104 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 62 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (92% lower).
The record links to 1 condition, with Leber Hereditary Optic Neuropathy appearing as the primary indexed condition, and to 2 interventions - of which GS010 is the first listed.
NCT03406104 lists 7 locations in 5 states (Other, California, Georgia).
Frequently Asked Questions
What is clinical trial NCT03406104 about?
NCT03406104 is a clinical study titled "RESCUE and REVERSE Long-term Follow-up". The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.
What is the current status of trial NCT03406104?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 62 participants. The study started on 2018-01-09. Estimated completion is 2022-07-04.
What conditions does trial NCT03406104 study?
This clinical trial studies the following conditions: Leber Hereditary Optic Neuropathy.
What interventions are being tested in trial NCT03406104?
The interventions under investigation include: GS010 (GENETIC), Sham Intravitreal Injection (OTHER).
Who is sponsoring clinical trial NCT03406104?
This trial is sponsored by GenSight Biologics, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03406104 being conducted?
This trial has 7 study locations across California, Georgia, Pennsylvania, Greater London. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT03406104's enrollment target sits among peer trials
62 1802nd of 2000 higher than 198 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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