Phase 3
N=62
RESCUE and REVERSE Long-term Follow-up
Leber Hereditary Optic Neuropathy
Bottom Line
View on ClinicalTrials.gov: NCT03406104 ↗Enrolled (actual)
62
Serious AEs
12.9%
Results posted
Jan 2024
Primary outcome: Primary: Ocular Adverse Events (AEs) — 5; 1 eyes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- GS010 (Genetic); Sham Intravitreal Injection (Other)
- Age
- Pediatric, Adult, Older Adult · 15+ yrs
- Sex
- All
- Sponsor
- GenSight Biologics
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Ocular Adverse Events (AEs) |
5; 1 | — |
| SECONDARY Visual Acuity |
-0.44; -0.39 | — |
| SECONDARY Responder Analysis: Clinically Relevant Recovery |
60; 66 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase III clinical studies
- Subject of legal consent age has provided informed consent; subjects that are not of legal consent age have undergone their country-approved clinical trial enrollment consent process
Exclusion Criteria
- Subject is unwilling or unable to comply with the protocol requirements
- Subject has any medical or psychological condition that, in the opinion of the Investigator, may compromise his or her safe participation in the study
- Subject is taking or intending to take idebenone during the long-term follow-up study period
Data sourced from ClinicalTrials.gov (NCT03406104). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.