Gene therapy injection aims to save sight in rare eye disease
NCT ID NCT07439887
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single injection of a gene therapy called RTx-021 in the eye of people with Stargardt disease, a genetic condition that causes vision loss. Up to 18 participants will receive different doses to check safety and tolerability. Researchers will monitor side effects and measure vision changes over 5 years.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- RTx-021 (optogenetic gene therapy)
- What this could lead to
- If it works, this could point toward a treatment that helps preserve or improve vision in people with Stargardt disease.
- What could go wrong
- This is a very early, small trial (18 people) focused on safety, not effectiveness. The gene therapy is new and may not improve vision or could cause side effects.
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Dec 2030
An estimate. End dates often move.
- Lead sponsor
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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
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16 years and older
- Sex
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Anyone
- Healthy volunteers
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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: * Male and female patients \>= 16 years of age * Able to comply with the study visit schedule and all protocol assessments * Diagnosis of Stargardt Disease (genetic testing required) * 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 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 including those in which the disease itself, or the treatment of the disease, can alter ocular and/or central nervous system function (e.g. radiation treatment of the orbit; leukemia with 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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
3 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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RayTx Clinical Site
RECRUITINGBakersfield, California, 93309, United States
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RayTx Clinical Site
RECRUITINGBellaire, Texas, 77401, United States
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UPMC Vision Institute
RECRUITINGPittsburgh, Pennsylvania, 15219, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can stem cells restore vision in stargardt disease? a First-in-Human trial aims to find out
- Could stem cells restore sight in damaged eyes?
- New study tracks stargardt disease to pave way for future treatments
- New eye camera could unlock secrets of inherited blindness
- New cell therapy aims to slow vision loss in retinal disease
- Thousands join fight against blindness by sharing their stories