Gene therapy injection aims to restore sight in rare eye diseases

NCT ID NCT06460844

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This early-phase trial tests a single injection of a gene therapy called RTx-015 in 10 adults with retinitis pigmentosa or choroideremia, two inherited conditions that cause vision loss. The therapy delivers a gene that makes retinal cells produce a light-sensitive protein, potentially helping them respond to light. The main goal is to check safety, not yet to prove it improves vision.

What this could mean

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

Active substance
RTx-015 (gene therapy injection into the eye)
What this could lead to
If it works, this could point toward a treatment that helps people with certain inherited eye diseases see better by making light-sensitive proteins in the retina.
What could go wrong
This is a very early, small safety study with only 10 people. It is not designed to prove the treatment works, and there may be side effects from the injection or the gene therapy.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

10 people

The number who actually took part.

Started

Aug 2024

Expected to finish

Oct 2030

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

18 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: * Adults ≥18 years of age * Informed consent obtained from the patient * Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical diagnosis of Choroideremia with known causative mutation * Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria * Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center * Adequate organ function and general good health Exclusion Criteria: * Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months * Concurrent participation in another interventional clinical ocular study * Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy * Pre-existing eye conditions in either eye that would preclude the planned treatment or, in the opinion of the Investigator, are significant enough to interfere with the interpretation of study endpoints or procedural complications * Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications * Complicating systemic diseases; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or Central Nervous System (CNS) function (e.g., radiation treatment of the orbit; leukemia with CNS/optic nerve involvement) * Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others * Cataract or other ocular (including refractive) surgery, intraocular and/or peri-ocular injection in either eye within the prior four months (i.e., 120 days) prior to screening * Prior vitrectomy or aphakia in the study eye * Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g., povidone-iodine to prep for intravitreal injection) * Known contraindication to prophylactic steroid regimen * Current pregnancy or breastfeeding * Any other condition that would not allow the patient to complete follow-up examinations during the study

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Retina Consultants of Texas Research Centers

    Bellaire, Texas, 77401, United States

  • Retina Vitreous Associates Medical Group

    Beverly Hills, California, 90211, United States

  • UCI Alpha Clinic

    Orange, California, 92868, United States

  • UPMC Vision Institute

    Pittsburgh, Pennsylvania, 15221, United States

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