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.
Name: GS010
Name: Sham
Description: AEs or SAEs (ocular or systemic) related to IMP or administration procedure, as judged by the Investigator, reported during the long-term follow-up visits (2, 2.5, 3, 4, and 5 years) from the period of 96 weeks up to 5 years post-treatment and summarized descriptively by type, frequency (number, percentage), severity, causal relationship, and seriousness
Measure: Adverse events (AEs) or serious adverse events (SAEs) (ocular or systemic) Time: Up to 5-Year post-treatmentDescription: Change in best BCVA reported with LogMAR
Measure: Best-Corrected Visual Acuity (BCVA) reported with LogMAR Time: Up to 5-Year post-treatmentDescription: Change of parameters measured with HVF 30-2
Measure: HumphreyTM visual field (HVF) 30-2 parameters Time: Up to 5-Year post-treatmentDescription: Change of parameters measured with SD-OCT
Measure: Spectral domain optical coherence tomography (SD-OCT) parameters Time: Up to 5-Year post-treatmentDescription: Response status of eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection (An improvement of at least 15 ETDRS letters/Eyes that lose less than the 15 ETDRS letters)
Measure: Responder Analysis Time: Up to 5-Year post-treatmentDescription: Time course of the response in eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, for the BCVA reported with LogMAR, for parameters measured with HVF 30-2 and SD-OCT
Measure: Time course of the response Time: Up to 5-Year post-treatmentDescription: Visual improvement as measured by LogMAR by analysis of covariance (ANCOVA)
Measure: Visual improvement Time: Up to 5-Year post-treatmentDescription: Change of GCL thickness/volume and topographical map and other parameters measured by SD-OCT using a mixed model of ANCOVA
Measure: Change of ganglion cell layer (GCL) thickness/volume and topographical map and other parameters measured by SD-OCT Time: Up to 5-Year post-treatmentDescription: QOL as measured with VFQ-25 subject-rated instrument
Measure: Quality of Life: Visual Functioning Questionnaire 25 (VFQ-25) Time: Up to 5-Year post-treatmentDescription: QOL as measured with SF-36v2 subject-rated instrument
Measure: Quality of Life: 36-Item Short Form Health Survey, version 2 (SF-36-v2) Time: Up to 5-Year post-treatmentAllocation: Randomized
Parallel Assignment
There is one SNP
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. --- G11778A ---