Single intravitreal RTx-021 injection for Stargardt disease
Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)
PHASE1; PHASE2 · Ray Therapeutics, Inc. · NCT07439887
This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 18 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Ray Therapeutics, Inc. (industry) |
| Drugs / interventions | radiation |
| Locations | 3 sites (Bakersfield, California and 2 other locations) |
| Trial ID | NCT07439887 on ClinicalTrials.gov |
What this trial studies
This is a first-in-human, open-label, non-randomized dose-escalation program enrolling 9–18 patients across up to three dose cohorts. Each participant will receive a single unilateral intravitreal injection of the optogenetic gene therapy RTx-021 in the study eye, with cohorts initially of three patients and potential cohort expansion based on safety. Participants will be followed with frequent visits in the first 12 months and continued monitoring out to five years to characterize safety and preliminary signs of efficacy. The design supports selection of a dose for later-stage development.
Who should consider this trial
Good fit: Ideal candidates are people aged 16 or older with genetically confirmed Stargardt disease, adequate retinal ganglion cell or retinal nerve fiber layer on OCT, qualifying visual acuity in the study eye, good general health, and ability to comply with long-term follow-up.
Not a fit: Patients with very advanced degeneration lacking retinal ganglion cells or retinal nerve fiber layer, prior gene therapy/retinal implants, or other significant ocular conditions that would confound outcomes are unlikely to benefit.
Why it matters
Potential benefit: If successful, RTx-021 could preserve or restore some visual function in treated eyes and slow vision loss for people with Stargardt disease.
How similar studies have performed: Other optogenetic gene therapies for inherited retinal disorders have entered early clinical testing with preliminary safety and limited functional signals in small cohorts, but using RTx-021 in Stargardt disease is a first-in-human application.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Bakersfield, California and 2 other locations
- RayTx Clinical Site — Bakersfield, California, United States (RECRUITING)
- UPMC Vision Institute — Pittsburgh, Pennsylvania, United States (RECRUITING)
- RayTx Clinical Site — Bellaire, Texas, United States (RECRUITING)
Study contacts
- Study coordinator: Executive Clinical Director
- Email: clinicaltrials@raytherapeutics.com
- Phone: 858-617-8610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Stargardt Disease, AURORA, RTx-021