RGX-314 Gene Therapy for Diabetic Retinopathy (ALTITUDE Trial)

Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: REGENXBIO, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?RGX-314 is being developed as a novel, potential one-time gene therapy treatment for the treatment of diabetic retinopathy, a chronic and progressive complication of diabetes mellitus. Diabetic retinopathy (DR) is a sight-threatening disease characterized in the early stages by neuronal and vascular dysfunction in the retina, and later by neovascularization that leads to further deterioration of functional vision. Despite the availability of current treatments, diabetic retinopathy remains the leading cause of vision loss in working-age adults, those between the ages of 20 and 74. Existing treatment with anti-VEGF agents, although shown to be effective, are limited by short therapeutic half-lives, which then require frequent intravitreal injections over the patient's lifetime, resulting in increased risk of associated adverse events and significant treatment burden. Due to the burden of treatment, patients often do not closely adhere to treatment regimens and experience sub-optimal outcomes and a decline in vision. RGX-314 is being developed as a potential one time treatment for diabetic retinopathy, which may deliver advantages over conventional treatments, such as potentially providing a longer duration of therapeutic effect and intervening at an earlier stage of the disease.

Eligibility Criteria

This trial is for adults aged 25-89 with diabetic retinopathy, who have good vision (20/40 or better) and can delay other treatments like PRP or anti-VEGF injections for at least 6 months. It's not for those with active macular edema, prior eye surgeries, potential pregnancy, or retinal issues not caused by diabetes.

Exclusion Criteria

I have active diabetic macular edema.

I have had PRP or retinal laser treatment in my study eye.

My eye condition is not due to diabetic retinopathy.

I have had or currently have a detached retina in one eye.

I have had surgery to remove the vitreous gel from my eye.

Treatment Details

The study tests RGX-314 gene therapy as a one-time treatment for diabetic retinopathy. Participants will receive different doses of RGX-314 administered into the suprachoroidal space of the eye to see if it can improve their condition without frequent treatments.

4Treatment groups

Experimental Treatment

Active Control

Group I: RGX-314 Treatment Arm (Dose 3) and Topical SteroidExperimental Treatment2 Interventions

RGX-314 Dose 3 and Topical Steroid

Group II: RGX-314 Treatment Arm (Dose 2)Experimental Treatment1 Intervention

RGX-314 Dose 2

Group III: RGX-314 Treatment Arm (Dose 1)Experimental Treatment1 Intervention

RGX-314 Dose 1

Group IV: Observation Control ArmActive Control1 Intervention

Observation Control