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Gene therapy trial aims to restore night vision in rare eye disease

NCT ID NCT06388200

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 gene therapy called OCU400 for people with retinitis pigmentosa, an inherited eye disease that causes vision loss. The treatment is injected under the retina to help light-sensitive cells work better. The trial enrolled 140 people aged 3 and older and measures if they can navigate a maze in dim light after one year.

Why investors are watching

Ocugen is a micro-cap company with few products, so this Phase 3 trial of OCU400 for retinitis pigmentosa is its main value driver. The study has finished enrolling 140 patients, and the results will show whether the gene therapy can slow or stop vision loss across many genetic causes of the disease. A clear readout matters because Ocugen's future depends heavily on this single drug.

If it works: A positive result could support Ocugen's path to regulatory approval for OCU400, giving the company its first marketed product. That outcome would validate its gene therapy platform and could lead to a commercial launch in a large inherited blindness market.

If it fails: Phase 3 trials often fail, and OCU400 could miss its efficacy goals or show safety problems. A negative or unclear result would likely force Ocugen to restart development or abandon the program, leaving the company with few other assets.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

140 people

The number who actually took part.

Started

Jun 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.

Ages

3 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.

Inclusion Criteria: 1. Males or females ≥ 3 years of age 2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP 3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3) 4. BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart 5. Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent 6. Able to perform a Luminance LDNA at certain light intensity at the Screening visit 7. Presence of photoreceptors as determined by SD-OCT Exclusion Criteria: 1. Subject lacks evidence of outer nuclear layer 2. Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year. 3. History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma. 4. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months. 5. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant. 6. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa retinitis pigmentosa 4

As listed by the trial registrant

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

Retinitis pigmentosa

Contacts and locations

Locations

  • Advanced Research, LLC.

    Deerfield Beach, Florida, 33064, United States

  • Associated Retina Consultants

    Phoenix, Arizona, 85020, United States

  • Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine

    Miami, Florida, 33136, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Calgary Retina Consultants

    Calgary, Alberta, T2H OC8, Canada

  • Erie Retina Research LLC

    Erie, Pennsylvania, 16507, United States

  • Gundersen Health System

    La Crosse, Wisconsin, 54601, United States

  • Research Institute of the McGill University Health Centre

    Montreal, Quebec, H2W 1L4, Canada

  • Retina Consultants of Texas

    Bellaire, Texas, 77401, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231, United States

  • Sunnybrook Health Sciences Centre

    Toronto, Ontario, M4N 3M5, Canada

  • The University of British Columbia

    Vancouver, British Columbia, V5Z 3N9, Canada

  • University of Alberta

    Edmonton, Alberta, T6G 3E1, Canada

  • University of Southern Califormia

    La Jolla, California, 92093, United States

  • University of Southern California, Roski Eye Insitute

    Los Angeles, California, 90033, United States

  • Valley Retina Institute

    McAllen, Texas, 78503, United States

  • Vanderbilt Eye Institute

    Nashville, Tennessee, 37232, United States

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