The trial is taking place at:
R

Retinal Diagnostic Center | Campbell, CA

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RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) Without Center Involved-Diabetic Macular Edema (CI-DME) (ALTITUDE)

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Status and phase

Enrolling
Phase 2

Conditions

Diabetic Retinopathy (DR)

Treatments

Drug: Topical Steroid
Genetic: RGX-314 Dose 1
Genetic: RGX-314 Dose 2
Genetic: RGX-314 Dose 3

Study type

Interventional

Funder types

Industry

Identifiers

NCT04567550
RGX-314-2202

Details and patient eligibility

About

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.

Full description

This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of RGX-314 gene therapy in subjects with DR without center-involved diabetic macular edema (CI-DME). Approximately 100 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 5 cohorts. Participants will be randomized in Cohorts 1, 2, 4 and 5 to receive RGX-314 or to be observed, and participants enrolled in Cohorts 3 will receive RGX-314. Cohort 1 will evaluate RGX-314 Dose 1, Cohorts 2 and 3 will evaluate RGX-314 Dose 2, Cohorts 4 and 5 will evaluate RGX-314 Dose 3. Following SCS RGX-314 administration, participants in Cohorts 4 and 5 will receive a protocol-mandated post-procedure steroid regimen for 7 weeks. Participants who are randomized to be observed in Cohorts 1, 2, 4 and 5 will be offered RGX-314 after completing the study. Efficacy will be the primary focus of the study. Participants will be evaluated for safety and tolerability of RGX-314 throughout the study.

Enrollment

100 estimated patients

Sex

All

Ages

25 to 89 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

* Patients 25-89 years of age with a diabetic retinopathy diagnosis of nonproliferative diabetic retinopathy (NPDR) and proliferative diabetic retinopathy (PDR) secondary to diabetes mellitus Type 1 or 2 for which PRP or anti-VEGF injections can be safely deferred for at least 6 months * Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of ≥69 letters (approximate Snellen equivalent of 20/40 or better) * Prior history of CI-DME in the study eye is acceptable. * Must be willing and able to provide written, signed informed consent.

Exclusion criteria

* Neovascularization in the study eye from a cause other than DR. * Presence of any active CI-DME. * Active or history of retinal detachment in the study eye. * Any evidence or documented history of PRP or retinal laser in the study eye. * Patients who had a prior vitrectomy surgery. * Women of childbearing potential. Note: Other inclusions/exclusions criteria apply

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Single Blind

100 participants in 4 patient groups

Observation Control Arm
No Intervention group
Description:
Observation Control
RGX-314 Treatment Arm (Dose 1)
Experimental group
Description:
RGX-314 Dose 1
Treatment:
Genetic: RGX-314 Dose 1
RGX-314 Treatment Arm (Dose 2)
Experimental group
Description:
RGX-314 Dose 2
Treatment:
Genetic: RGX-314 Dose 2
RGX-314 Treatment Arm (Dose 3) and Topical Steroid
Experimental group
Description:
RGX-314 Dose 3 and Topical Steroid
Treatment:
Genetic: RGX-314 Dose 3
Drug: Topical Steroid

Trial contacts and locations

21

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Central trial contact

Patient Advocacy

Data sourced from clinicaltrials.gov

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