Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to… (NCT02652767) | Clinical Trial Compass
CompletedPhase 3
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
United States, France, Germany39 participantsStarted 2016-02-23
Plain-language summary
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.
Who can participate
Age range15 Years
SexALL
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Inclusion criteria
✓. Age 15 years or older.
✓. Onset of vision loss based on medically documented history or participant testimony, in at least one eye for ≤180 days in duration and if both eyes are affected the duration of vision loss in both eyes must be ≤180 days in duration.
✓. Each eye of the participant maintaining visual ability to allow at least for counting of the examiner's fingers at any distance.
✓. Female participants (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after intravitreal (IVT) injection and male participants must agree to use condoms for up to 6 months after IVT injection.
✓. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.
✓. Signed written informed consent.
✓. Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary Leber Hereditary Optical Neuropathy (LHON)-associated mutations (ND1 or ND6) in the participant's mitochondrial DNA.
✓. Review of all selection criteria to ensure continued compliance.
Exclusion criteria
✕. Any known allergy or hypersensitivity to GS010 or its constituents.
✕. Contraindication to IVT injection.
✕. IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1).
What they're measuring
1
Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48
✕. Narrow angle in either eye contra-indicating pupillary dilation.
✕. Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period.
✕. Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.
✕. Causes of optic neuropathy other than LHON and glaucoma.