RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

NCT ID: NCT04832724

Last Updated: 2024-09-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-02-22
• Study Completion Date: 2024-03-18

Brief Summary

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration

Detailed Description

Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long repeated intraocular injections to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. This phase 2, open label study will explore the pharmacodynamics of two doses in two formulations of RGX-314 gene therapy via subretinal delivery in patients with neovascular Age-related Macular Degeneration. Approximately 60 patients (15 per cohort) who meet the inclusion/exclusion criteria will be enrolled in 4 sequential dose cohorts. A dose cohort will be comprised of 1 of 2 doses of RGX-314 in 1 of 2 formulations in order to explore the pharmacodynamics of RGX-314 based on aqueous humor transgene product (TP) concentrations. If the participants meet the study criteria and choose to participate in the study, their participation in the study will last about 12 months.

Conditions

Neovascular Age-related Macular Degeneration; Wet Macular Degeneration; Wet Age-related Macular Degeneration

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Commercial Formulation Dose 1

Dose 1 of RGX-314

Group Type: EXPERIMENTAL

RGX-314

Intervention Type: GENETIC

RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Clinical Formulation Dose 1

Dose 1 of RGX-314

Group Type: EXPERIMENTAL

RGX-314

Intervention Type: GENETIC

RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Commercial Formulation Dose 2

Dose 2 of RGX-314

Group Type: EXPERIMENTAL

RGX-314

Intervention Type: GENETIC

RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Clinical Formulation Dose 2

Dose 2 of RGX-314

Group Type: EXPERIMENTAL

RGX-314

Intervention Type: GENETIC

RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Interventions

RGX-314

RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Intervention Type: GENETIC

Eligibility Criteria

Inclusion Criteria

1. Males or females, aged ≥ 50 years and ≤ 89 years.

2. An Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score between ≤ 78 and ≥ 40 in the study eye at Screening.

3. Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye previously treated with anti-VEGF.

4. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry.

5. Willing and able to provide written, signed informed consent for this study.

6. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.

Exclusion Criteria

1. CNV or macular edema in the study eye secondary to any causes other than AMD.

2. Subfoveal fibrosis or atrophy in study eye.

3. Any condition in the investigator's opinion that could limit visual acuity improvement in the study eye.

4. Active or history of retinal detachment or retinal tear in the study eye.

5. Advanced glaucoma in the study eye.

6. Prior treatment with gene therapy.

7. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 89 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AbbVie

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Retinal Consultants of Arizona

Phoenix, Arizona, United States

Barnet Dulaney Perkins Eye Center

Sun City, Arizona, United States

Retina Consultants of San Diego

San Diego, California, United States

California Retina Consultants

Santa Barbara, California, United States

Retina Vitreous Associates of Florida

St. Petersburg, Florida, United States

Wilmer Eye Institute

Baltimore, Maryland, United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States

Sierra Eye Associates

Reno, Nevada, United States

Eye Associates of New Mexico

Albuquerque, New Mexico, United States

Wills Eye Hospital

Philadelphia, Pennsylvania, United States

Charles Retina Institute

Germantown, Tennessee, United States

Texas Retina Associates

Dallas, Texas, United States

Retina Consultants of Texas

The Woodlands, Texas, United States

Countries

United States

Other Identifiers

RGX-314-2103

Identifier Type: -

Identifier Source: org_study_id