RecruitingPhase 1Phase 2NCT07439887

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

Sponsor

Ray Therapeutics, Inc.

Enrollment

18 participants

Start Date

Jan 28, 2026

Study Type

INTERVENTIONAL

Conditions

Stargardt Disease

Summary

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.

Eligibility

Min Age: 16 Years

Inclusion Criteria6

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 Criteria13

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