RTx-021 for Stargardt Disease

(AURORA Trial)

Recruiting at 2 trial locations

Overseen By Executive Clinical Director

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ray Therapeutics, Inc.

Must not be taking: Immunosuppressants, Steroids

Disqualifiers: Gene therapy, Eye surgery, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 13

Duration: 1 day

18 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Are You a Good Fit for This Trial?

Inclusion Criteria

I have been diagnosed with Stargardt Disease confirmed by genetic testing.

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

Exclusion Criteria

Current pregnancy or breastfeeding

Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months

I have not had eye surgery or injections in either eye in the past four months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single, unilateral intravitreal injection of RTx-021 in the study eye

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

Frequent visits in the initial 12 months, then periodic visits

What Are the Treatments Tested in This Trial?

Interventions

• RTx-021

How Is the Trial Designed?

Group I: Middle Dose RTx-021 Experimental Treatment 1 Intervention

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.

Group II: Low Dose RTx-021 Experimental Treatment 1 Intervention

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.

Group III: High Dose RTx-021 Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Ray Therapeutics, Inc.

Lead Sponsor

Trials

2

Recruited

140+