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Phase 1/2 Study of RTx-021 for Stargardt Disease

This study is testing a new gene therapy called RTx-021 for people with Stargardt Disease, a genetic eye condition that causes vision loss. This is a Phase 1/2 study, meaning it's an early-stage trial to check the safety of RTx-021 and see if it has any effect. You might be able to join if you are at least 16 years old, have Stargardt Disease confirmed by genetic testing, and meet certain vision requirements. The main goal is to see how safe RTx-021 is over 6 months after a single injection into one eye. The study plans to enroll up to 18 participants, and the current recruitment status is unclear.

Study design
This is an open-label (everyone knows what treatment is given), non-randomized study where participants receive a single injection of RTx-021. It will enroll up to 18 patients in different dose groups.
What's involved
You would receive a single injection of RTx-021 into one eye and be followed for 5 years, with more frequent visits in the first year.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 5 years after treatment.

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NCT07439887

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

Recruiting
PHASE1Ages 16+InterventionalTreatment
Ray Therapeutics, Inc.
~18 participants
Updated 2026-04-15 on ClinicalTrials.gov
What's tested:RTx-021

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment-Emergent Adverse Events
Measured over 6 Months

Conditions

Stargardt Disease

Explore related trials

Stargardt Disease

Where it's being run

3 sites across 3 states
California1
Pennsylvania1
Texas1

Who to contact

Executive Clinical Director
[email protected]
858-617-8610
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Incidence of Treatment-Emergent Adverse Events6 Months

    The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher