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

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)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Ray Therapeutics, Inc. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Stargardt Disease

Interventions

Type	Name	Description
GENETIC	RTx-021	Optogenetic gene therapy

Timeline

• Start date: 2026-01-28
• Primary completion: 2030-12-01
• Completion: 2030-12-01
• First posted: 2026-02-27
• Last updated: 2026-04-15

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study