Gene therapy injection aims to restore sight in rare eye diseases
NCT ID NCT06460844
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
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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 Hide 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
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UPMC Vision Institute
Pittsburgh, Pennsylvania, 15221, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Stem cell eye transplant aims to halt blindness from rare genetic disease
- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Could stem cells restore sight in damaged eyes?
- Gene therapy injection aims to restore sight in rare blindness