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 range
15 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial tested a gene therapy called GS010 specifically for people with Leber Hereditary Optic Neuropathy caused by the ND4 mutation — can you test whether my vision loss is actually caused by the ND4 mutation, and if so, does that make me a potential candidate for this type of treatment?
2The trial only enrolled people within 6 months of their vision loss starting — since timing seems to have been a key eligibility factor, how does the stage of my own vision loss affect what treatment options might still be available to me?
3Since this was a completed Phase 3 trial measuring visual acuity changes at 48 weeks, have the full results been published, and what do they tell us about how much vision improvement patients actually experienced compared to a placebo?
4Gene therapy like GS010 is a relatively new approach — based on what's now known from this trial's completed results, what were the main safety concerns or side effects that were reported, and how might those risks apply to my situation?
5Are there currently any approved treatments or other active clinical trials for ND4-related LHON that I should be comparing this gene therapy approach against before deciding on a treatment path with you?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48
. 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).
. Previous vitrectomy in either eye.
. 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.