CompletedPhase 3

Safety and Efficacy of Emixustat in Stargardt Disease

United States, Brazil, Canada194 participantsStarted 2019-01-07

Plain-language summary

The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease. Funding Source -- FDA OOPD

Who can participate

Age range

16 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: * A clinical diagnosis of macular atrophy secondary to Stargardt disease (STGD) * Macular atrophy measured to fall within a defined size range * Two mutations of the ABCA4 gene. If only one mutation, a typical STGD appearance of the retina. * Visual acuity in the study eye of at least 20/320 Exclusion Criteria: * Macular atrophy secondary to a disease other than STGD * Mutations of genes, other than ABCA4, that are associated with retinal degeneration * Surgery in the study eye in the past 3 months * Prior participation in a gene therapy or stem cell clinical trial for STGD * Recent participation in a clinical trial for STGD evaluating a complement inhibitor or vitamin A derivative * Use of certain medications in the past 4 weeks that might interfere with emixustat * An abnormal electrocardiogram (ECG) * Certain abnormalities on laboratory blood testing * Female subjects who are pregnant or nursing

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

Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)

Timeframe: 24 months

Trial details

NCT IDNCT03772665

SponsorKubota Vision Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-06-13

Results submitted2023-07-13

View on ClinicalTrials.gov More Stargardt Disease trials

Who can participate

Age range

16 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.