NCT07439887 · Ray Therapeutics, Inc.
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)
What this study is about
A Phase 1/2, where both patients and doctors know the treatment given, non-randomly assigned, gradually increasing doses study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021.
View original scientific description
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Bakersfield, California
- Pittsburgh, Pennsylvania
- Bellaire, Texas
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 15, 2026 · Source of record for eligibility and locations