Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON D… (NCT02652780) | Clinical Trial Compass
CompletedPhase 3
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
United States, France, Germany37 participantsStarted 2016-01
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 more than six months and up to one year.
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 participants testimony, in both eyes for 181 and ≤365 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 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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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 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.