A gene therapy clinical trial for the treatment of Leber hereditary optic neuropathy (LHON) due to the ND4 mutation is currently recruiting participants. The trial is based in Moorfields Eye Hospital in London and open to participants from the Republic of Ireland.
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in patients with LHON due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.
Each participant will have one eye randomly selected to receive a single dose of GS010 via intravitreal injection. The other eye will receive a sham or placebo injection. The primary outcome measure is improvement in visual acuity measured 48 weeks after injection.
Selection and inclusion criteria are outlined below. For more information about the clinical trial and full details of exclusion criteria, please visit https://clinicaltrials.gov/ct2/show/NCT02652767?term=GenSight+Biologics&rank=2
Participants must meet all the following criteria at the Screening Visit (Visit 1) in order to be included into the study.
Participants included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).
Study title: Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)
Lead investigator: Dr Patrick Yu-Wai-Man
Sponsor: GenSight Biologics