ABCA4-related retinopathy
MONDO:0800406An inherited retinopathy caused by bi-allelic variants in the ABCA4 gene.
38 clinical trials for this condition and its sub-types, 7 tagged with ABCA4-related retinopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of ABCA4-related retinopathy
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Cone-rod dystrophy 3 0 trials
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Retinitis pigmentosa 19 0 trials
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Could a common diabetes drug protect eyesight in stargardt disease?
Disease control OngoingThis study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The go…
Phase 1/2 • Sponsor: National Eye Institute (NEI) • Aim: Disease control
Last updated Aug 21, 2026 00:00 UTC
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Gene therapy slows vision loss in stargardt disease trial
Disease control OngoingThis study tests a new gene therapy called OCU410ST for people with Stargardt disease, an inherited eye condition that causes vision loss. Early results show the treatment slowed damage to the retina by nearly half and improved or stabilized eyesight in all treated patients. The …
Phase 2/3 • Sponsor: Ocugen • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
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Scientists track rare eye disease to prepare for future treatments
Knowledge-focused OngoingThis study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. …
Sponsor: National Eye Institute (NEI) • Aim: Knowledge-focused
Last updated Sep 03, 2026 00:00 UTC