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Gene therapy trial aims to save sight in boys and men with rare retinal disease

NCT ID NCT05878860

Disease control Sponsor: Atsena Therapeutics Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 09, 2026 · Updated 8 times

Summary

This study tests a gene therapy called ATSN-201 in males aged 6 and older with X-linked retinoschisis, a genetic eye condition that leads to vision loss. The therapy delivers a working copy of the RS1 gene to the retina. The trial has three parts: first, finding the right dose; second, expanding to more people; and third, a randomized phase to see if it improves vision compared to no treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ATSN-201 gene therapy
What this could lead to
If successful, this could become a treatment that improves vision and slows vision loss in people with X-linked retinoschisis.
What could go wrong
This is an early-to-mid stage trial with a small number of participants. Gene therapy carries risks like inflammation or immune reactions, and it may not work for everyone.

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

About 97 people

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

Started

Aug 2023

Expected to finish

Apr 2033

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

6 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.

Part A and B: Inclusion Criteria: 1. Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5. 2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1. 3. Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40). 4. Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator. Exclusion Criteria: 1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants). 2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening or any intraocular surgery anticipated in the study eye during the first 12 months of the study. 3. Treatment in a prior ocular gene or cell therapy study. Part C: Inclusion Criteria: Note: For patients ineligible for bilateral dosing based on the ocular exclusion criteria, the same eye must meet all ocular inclusion criteria, but none of the ocular exclusion criteria, to be eligible for unilateral dosing. General All of the following criteria must be met for unilateral or bilateral dosing. 1. Age ≥ 6 years. 2. Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1. Ocular At least 1 eye must meet all of the following criteria for both unilateral and bilateral dosing. 3. BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40). 4. Presence of foveal schisis on SD-OCT. Exclusion Criteria: General None of the following criteria can be met for unilateral or bilateral dosing. 1. Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening. 2. Treatment in a prior ocular gene or cell therapy study. 3. Absence of macular schisis. 4. BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32). 5. Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants). 6. Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study.

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Conditions

The condition(s) this trial relates to.

retinoschisis X-linked retinoschisis

As listed by the trial registrant

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

X-linked retinoschisis

How to take part

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

  1. The places running it

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

  2. 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 ↗

  3. 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

Locations

  • Associated Retina Consultants

    RECRUITING

    Phoenix, Arizona, 85020, United States

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

  • Boston Children's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University

    RECRUITING

    New York, New York, 100032, United States

  • Duke University

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Flaum Eye Institute, University of Rochester

    NOT_YET_RECRUITING

    Rochester, New York, 14642, United States

  • Massachusetts Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • McGill University (MUHC)

    NOT_YET_RECRUITING

    Montreal, Quebec, H4A 3J1, Canada

  • Moran Eye Center

    RECRUITING

    Salt Lake City, Utah, 84132, United States

  • Oregon Health Sciences University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Oxford Eye Hospital

    NOT_YET_RECRUITING

    Oxford, England, OX3 9DU, United Kingdom

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • Shiley Eye Institute

    NOT_YET_RECRUITING

    La Jolla, California, 92093, United States

  • Stanford University

    NOT_YET_RECRUITING

    Palo Alto, California, 94303, United States

  • The Hospital for Sick Children

    NOT_YET_RECRUITING

    Toronto, Ontario, M5G 0A4, Canada

  • UC San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

  • University of Michigan

    NOT_YET_RECRUITING

    Ann Arbor, Michigan, 48105, United States

  • University of Wisconsin

    NOT_YET_RECRUITING

    Madison, Wisconsin, 53705, United States

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