An Observational Study in Children and Adults With Stargardt Disease (NCT06591806) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
An Observational Study in Children and Adults With Stargardt Disease
United States, Norway80 participantsStarted 2024-05-01
Plain-language summary
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
Who can participate
Age range
8 Years – 50 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
. Male and female subjects between 8 and 50 years of age at the time of enrolment.
. Willingness to adhere to the protocol as evidenced by written informed consent if the subject is 18 years or older. If the subject is under 18 years of age, written assent must be obtained from the subject and written informed consent must be obtained from the subject's legally authorized representative (parent or legal guardian).
. Confirmed mutation in the ABCA4 gene.
Exclusion criteria
. History of uveitis.
. Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
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.
1Since this is an observational study for Stargardt Disease, does it involve any experimental treatments, or would it simply involve monitoring and imaging my eyes over time — and how might that affect my day-to-day care?
2This trial is currently active but no longer recruiting new participants — is there a similar observational or interventional study for Stargardt Disease that I might still be eligible to join?
3The study is measuring changes in Fundus Autofluorescence from a baseline — can you explain what that imaging test shows about how Stargardt Disease is progressing, and whether you're already tracking this as part of my standard care?
4Because this is a Phase NA observational study rather than a treatment trial, would participating in something like this help build the kind of evidence base that could lead to future therapies for Stargardt Disease, and is that something worth factoring into my decisions?
5Are there any interventional clinical trials for Stargardt Disease currently recruiting that you think might be worth discussing as an alternative or complement to an observational approach like this one?
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
To measure the change from baseline in Fundus Autofluorescence