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New study aims to map how stargardt disease worsens over time

NCT ID NCT06435000

Knowledge-focused Sponsor: Splice Bio Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This observational study will follow 75 people aged 12 to 65 with Stargardt disease type 1, a genetic eye condition that causes vision loss. Researchers will use eye exams and imaging to track how the disease changes over time. The goal is to better understand the disease's progression, which could help in planning future 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 could help researchers design future treatments by providing a clearer picture of how Stargardt disease progresses.
What could go wrong
This is an observational study, not a treatment trial. It will not test any new drug or therapy, so there is no direct benefit to participants. The results may not lead to any immediate medical advances.

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

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2024

Expected to finish

Feb 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participants will be selected that have a genetic and clinical diagnosis of STGD1 and fulfil all other entry criteria.

Ages

12 to 65 years

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Provide written consent 2. Are male or female aged 12-65 years old 3. Have a diagnosis of STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene confirmed genotypically by an accredited genotyping laboratory 4. Have a history of STGD1 progression within the last 2 years, in the opinion of the investigator. 5. Eligible eye(s) must have: 1. BCVA of between 24-88 ETDRS letters, inclusive (20/20 - 20/320 Snellen equivalent, 0.0-1.2 logMAR) at the Screening Visit. 2. Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease. 3. Fundus autofluorescence (FAF) measurement of definitely decreased autofluorescence (DDAF) as measured by the Central Reading Center (CRC). 4. Total lesion must be imaged in its entirety. 5. All total lesion borders must be ≥300 microns from all image edges. 6. Eligible eye(s) must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging. Exclusion Criteria: 1. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel. 2. Have any concurrent ocular disease that would affect study procedures or outcomes (e.g., cataracts; subjects can be enrolled 90 days after successful cataract surgery) in eligible eyes. 3. Have two likely pathogenic or pathogenic variants (not STGD1) in autosomal recessive inherited retinal dystrophy (IRD) genes or a single likely pathogenic or pathogenic variant in autosomal dominant or X-linked IRD genes. 4. Have had any intraocular surgery or thermal laser within 90 days of study entry or any prior thermal laser in the macular region within the eligible eye(s). 5. Have any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the study period. 6. Are unwilling to stop taking the following products at Screening and throughout the study: 1. Supplements containing vitamin A or beta-carotene, liver-based products. 2. Prescription oral retinoids. 7. Have actively participated in an investigational therapy study or have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer. Note: any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal injections exclude the subject from study participation. 8. Have known serious allergies to the fluorescein dye that might be used to measure intraocular pressure (IOP), ocular dilating drops, topical ocular anesthetic, or any history of anaphylaxis reaction. 9. Have a history of amblyopia in the eligible eye(s). 10. Have any significant ocular or non-ocular disease/disorder (or medication and/or laboratory test abnormalities) which, in the opinion of the investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or affect the subject's ability to participate in the study.

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Conditions

The condition(s) this trial relates to.

severe early-childhood-onset retinal dystrophy Stargardt disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Stargardt Stargardt disease Stargardt's disease STGD1

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    20 sites in 3 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

  • CUIMC/Edward S. Harkness Eye Institute

    RECRUITING

    New York, New York, 10032, United States

  • Columbia University Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Duke Eye Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Emory University

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Kellogg Clinical Research Center

    RECRUITING

    Ann Arbor, Michigan, 48105, United States

  • Leeds Teaching Hospitals NHS Trust

    RECRUITING

    Leeds, LS9 7T, United Kingdom

  • Massachusetts Eye and Ear Infirmary

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    RECRUITING

    London, London, EC1V 2PD, United Kingdom

  • Oregon Health & Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Oxford Eye Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • Shiley Eye Institute

    RECRUITING

    San Diego, California, 92093, United States

  • UCHealth Sue Anschutz-Rodgers Eye Center

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University Eye Hospital Tübingen

    RECRUITING

    Tübingen, 72076, Germany

  • University of Manchester - The Old St Mary's Hospital

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • University of Wisconsin, Madison

    RECRUITING

    Madison, Wisconsin, 53715, United States

  • Universitätsklinikum Bonn, Klinik für Augenheilkunde

    RECRUITING

    Bonn, 53127, Germany

  • Vitreo Retinal Associates

    RECRUITING

    Gainesville, Florida, 32607, United States

  • Wilmer Eye Institute, Johns Hopkins University MD 21287

    RECRUITING

    Baltimore, Maryland, 21287, United States

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