Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Egyéb feltételek

• Main Selection Criteria:
• Age 15 years or older on the date of signed informed consent
• Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye
• Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2); Main Non-Selection Criteria:
• Contraindication to intravitreal injection in any eye
• Subjects refusing to discontinue idebenone
• Previous vitrectomy in either eye
• Narrow angle in any eye contra-indicating pupillary dilation
• Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system
• History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation; Main Inclusion Criteria:
• Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2)
• Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as defined by the study's SOP for visual acuity testing
• Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA; Main Exclusion Criteria:
• Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as defined by the study's standard operating procedure (SOP) for visual acuity testing
• Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye
• Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding nicotine)