RecruitingPhase 1 / Phase 2NCT07002398
Ophthalmological
Stargardt Disease
Also known as Stargardt macular dystrophy, ABCA4 retinopathy, fundus flavimaculatus
Stargardt Disease is the most common inherited macular dystrophy, caused by mutations in the ABCA4 gene which encodes an ATP-binding cassette transporter essential for clearing toxic vitamin A byproducts from photoreceptor cells. Accumulati
ORPHA:827 ↗Gene ABCA4Prevalence 1 per 8,000–10,000Onset Childhood to early adulthoodAutosomal recessive
22
studies recruiting now
as of 7 Sept 2026
67
studies registered in total
as of 7 Sept 2026
2
countries with a recruiting site
as of 7 Sept 2026
29 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT01145196
Genotype-Phenotype Study of Patients With Plaquenil -Induced Retinal Toxicity, With Evaluation of the ABCA4 Gene
RecruitingNCT02617966
Rod and Cone Mediated Function in Retinal Disease
RecruitingPhase 1NCT07734064
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
RecruitingPhase 3NCT07419334
Study of ALK-001 on the Progression of Stargardt Disease
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 22 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Stargardt Disease
Stargardt Disease is the most common inherited macular dystrophy, caused by mutations in the ABCA4 gene which encodes an ATP-binding cassette transporter essential for clearing toxic vitamin A byproducts from photoreceptor cells. Accumulation of bisretinoid compounds, particularly A2E, leads to progressive retinal pigment epithelium and photoreceptor degeneration centred on the macula. The disease typically presents in the first two decades of life with central vision loss, although the rate of progression is highly variable even among individuals carrying the same mutations.
Common clinical features
Progressive central vision lossDifficulty reading or recognising facesColour vision disturbancesPhotophobia and photostressYellow-white flecks at the level of the RPE on fundus examinationDark adaptation delaysParacentral or central scotomasReduced visual acuity (typically 20/200 or worse at advanced stages)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
4 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Tinlarebant
Phase 3Emixustat (Acu-4429)
Phase 2Stg-001
Phase 2Gildeuretinol
Before you apply
Things trial teams commonly ask about for Stargardt Disease. Not eligibility rules; those are set by each study.
- Molecular confirmation of biallelic ABCA4 pathogenic variants is mandatory for most gene therapy and antisense oligonucleotide trials; full-field sequencing including deep intronic variants is recommended.
- Many trials exclude patients with best-corrected visual acuity below a specified threshold (e.g., worse than 20/800), so enrolment during earlier disease stages may expand eligibility.
- Avoiding vitamin A supplementation is sometimes required pre-trial; discuss current supplement use with your ophthalmologist before screening.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).