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Gene therapy slows vision loss in stargardt disease trial

NCT ID NCT05956626

Disease control Sponsor: Ocugen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 31, 2026 · Updated 1 time

Summary

This 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 trial now enrolls 51 participants aged 5 and older to confirm these benefits and check safety.

Why investors are watching

Ocugen is a micro-cap company whose value depends heavily on its eye disease pipeline. This trial tests OCU410ST, a gene therapy for Stargardt disease, a rare inherited condition that causes vision loss. Phase 1 data showed slower lesion growth and stable vision in treated eyes, and this larger Phase 2/3 study will confirm whether those benefits hold in a controlled setting.

If it works: A positive Phase 2/3 result could establish OCU410ST as a viable treatment for a disease with no approved therapy. That would give Ocugen a clear path toward regulatory approval and a commercial product, which would be a major milestone for a company of this size.

If it fails: Phase 2/3 trials often fail even after promising early data. If OCU410ST does not show a clear benefit over the untreated control group, or if safety problems appear, the company would face a significant setback with no approved product to fall back on.

AI-written from the trial record. Speculative, and not investment advice.

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.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 51 people

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

Started

Aug 2023

Expected to finish

Sep 2026

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.

Ages

5 years and older

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.

Phase 2/3 Inclusion Criteria (applicable for both adult and pediatric subjects): 1. Males or females aged ≥5 years at the time of consent. 2. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies). 3. Adult subjects who have BCVA of 75 letters or less in the study eye (20/32 Snellen equivalent) and Pediatric subjects who have a BCVA of 35 letters or better in the study eye (20/200 Snellen equivalent). 4. The study eye should have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns, and total lesion size must add to less than or equal to ≤ 18 mm\^2 (approximately 7-disc areas). 5. Have detectable outer nuclear layer (ONL) in the macular region Phase 2/3 Exclusion Criteria (applicable for both adult and pediatric subjects): 1. Participation in ongoing antiretroviral therapy treatment. 2. Participation in any investigational therapy study or receipt of investigational treatment within 60 days prior to screening or 5 half-lives (whichever is longer). Any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, intravitreal or subretinal injections, or participation in an Alkeus ALK-001 study within the past 6 months. 3. Macular atrophy secondary to any disease other than Stargardt Disease (STGD). 4. Presence of genetic mutations that mimic Stargardt Disease like ELOVL4, or PROM1. 5. Contraindication to subretinal injection or use of anesthesia (local and/or general). Phase 1 was a multicenter, open-label, dose-ranging/dose escalation study. Enrollment is complete for Phase 1

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Conditions

The condition(s) this trial relates to.

ABCA4-related retinopathy Stargardt disease

As listed by the trial registrant

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

Stargardt disease

Contacts and locations

Locations

  • Advanced Research, LLC

    Pompano Beach, Florida, 33064, United States

  • Associated Retina Consultants

    Phoenix, Arizona, 85020, United States

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Duke Eye Center

    Durham, North Carolina, 27710, United States

  • Erie Retina Research, LLC

    Erie, Pennsylvania, 16505, United States

  • Mississippi Retina Associates

    Jackson, Mississippi, 39202, United States

  • Retina Consultants of Texas

    Bellaire, Texas, 77401, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231, United States

  • Retina Partners Midwest, P.C.

    Carmel, Indiana, 46032, United States

  • Retinal Consultants Medical Group

    Sacramento, California, 95825, United States

  • The Retina Institute

    St Louis, Missouri, 63128, United States

  • Valley Retina Institute

    McAllen, Texas, 78503, United States

  • Vitreo Retinal Associates, P.A.

    Gainesville, Florida, 32607, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.