Scientists track rare eye disease to pave way for future treatments
NCT ID NCT04591483
First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 2 times
Summary
This study follows 25 people aged 10 and older with Stargardt-like macular dystrophy (STDG3), a rare inherited eye disease that causes vision loss. Over three years, researchers will measure changes in the retina using eye exams, imaging, and vision tests. The goal is to understand how the disease progresses naturally, which is essential for designing future clinical trials for potential treatments.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this study will provide crucial data on how STDG3 progresses, which could help design future treatment trials.
- What could go wrong
- This is an observational study, not a treatment trial. It will not test any therapy, and results may not lead directly to a cure.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 25 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2022
- Expected to finish
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Jul 2028
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
25 patients with Stargardt-like macular dystrophy 3 who are \>= 10 years of age.
- Ages
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10 to 100 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: To be eligible, the following inclusion criteria must be met, where applicable. 1. Stated willingness to comply with all study procedures and availability for the duration of the study. 2. Participant must be at least ten years of age. 3. Ability to perform required functional testing and ophthalmic imaging. 4. A mutation in ELOVL4 with a typical clinical presentation of Stargardt-like macular dystrophy. 5. Participant (or legal guardian) must understand and sign the protocol s informed consent document. EXCLUSION CRITERIA: A participant is not eligible if any of the following exclusion criteria are present. 1. Two or more definitive mutations in ABCA4 and/or one mutation in RDS/peripherin or PROM1. 2. Systemic medical contraindications that are rarely associated with ELOVL4 (e.g., Spinocerebellar Ataxia-34).
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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