Phase 3
Completed N=62
RESCUE and REVERSE Long-term Follow-up
Leber Hereditary Optic Neuropathy
Source: ClinicalTrials.gov NCT03406104 ↗
Enrolled (actual)
62
Serious AEs
12.9%
Results posted
Jan 2024
Primary outcomePrimary: Ocular Adverse Events (AEs) — 5; 1 eyes
◆ Published Evidence
Established
61citations · ~12 / year
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A <i>ND4</i> Mutation: Systematic Review and Indirect Comparison.
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.
Linked Publications (3)
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Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A <i>ND4</i> Mutation: Systematic Review and Indirect Comparison.
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Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
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Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.
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 | — |
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) and the linked publication. 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. Informational only — not medical advice.