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)

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)

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.

Study Overview

• Status: Recruiting
• Conditions: Stargardt Disease
• Intervention / Treatment: Genetic: RTx-021

Detailed Description

This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Executive Clinical Director; Phone Number: 858-617-8610; Email: clinicaltrials@raytherapeutics.com

Study Locations

• United States
  • California
    • Bakersfield, California, United States, 93309
      • Recruiting
      • RayTx Clinical Site
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15219
      • Recruiting
      • UPMC Vision Institute
      • Contact: UPMC Vision Institute Clinical Trials Team; Phone Number: 412-642-7888; Email: Clinicaltrialsoph@upmc.edu
  • Texas
    • Bellaire, Texas, United States, 77401
      • Recruiting
      • RayTx Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low Dose RTx-021; Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.	Genetic: RTx-021; Optogenetic gene therapy
Experimental: Middle Dose RTx-021; Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.	Genetic: RTx-021; Optogenetic gene therapy
Experimental: High Dose RTx-021; Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.	Genetic: RTx-021; Optogenetic gene therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events	The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher	6 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Best Corrected Visual Acuity (BCVA)	Change from Baseline to Month 6 after injection with RTx-021 in BCVA	6 Months
Low Luminance Visual Acuity (LLVA)	Change from Baseline to Month 6 after injection with RTx-021 in LLVA	6 Months
MNREAD Reading Assessment	Change from Baseline to Month 6 after injection with RTx-021 in reading acuity and reading speed.	6 Months
Contrast Sensitivity	Change from Baseline to Month 6 after injection with RTx-021 in contrast sensitivity	6 Months
Visual Field	Change from Baseline to Month 6 after injection with RTx-021 in the total area in which objects can be seen	6 Months
Low Vision Quality of Life (VA LV VFQ-48)	Change from Baseline to Month 6 after injection with RTx-021 in low vision quality of life questionnaire VA LV VFQ-48. A total score will be calculated with higher scores indicating an improvement.	6 Months

Collaborators and Investigators

• Sponsor: Ray Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 28, 2026
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2030

Study Registration Dates

• First Submitted: February 18, 2026
• First Submitted That Met QC Criteria: February 23, 2026
• First Posted (Actual): February 27, 2026

Study Record Updates

• Last Update Posted (Actual): April 15, 2026
• Last Update Submitted That Met QC Criteria: April 13, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: AURORA; RTx-021
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Stargardt Disease
• Other Study ID Numbers: RTx-021-CP-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: IPD is not currently being shared as this investigational treatment has not received regulatory approval.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No