Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD

A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of Suprachoroidal Administration of RGX-314 for Participants With Neovascular Age-Related Macular Degeneration

This is a prospective study designed to evaluate the long-term safety and efficacy of ABBV-RGX-314. Eligible participants are those who were previously enrolled in a clinical study of nAMD in which they received suprachoroidal space (SCS) administration of ABBV-RGX-314. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early discontinuation visit in the previous (parent) clinical study. Participants will be followed for up to 5 years after ABBV-RGX-314 administration (inclusive of the parent study). As such, the total study duration for each participant may vary depending on when they enroll in the current study following ABBV-RGX-314 administration in the parent study.

Study Overview

• Status: Enrolling by invitation
• Conditions: Neovascular Age-Related Macular Degeneration (nAMD)
• Intervention / Treatment: Drug: Standard of Care; Drug: Ranibizumab

• Study Type: Interventional
• Enrollment (Estimated): 116
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85014
      • Retinal Research Institute, LLC
  • California
    • Bakersfield, California, United States, 93309
      • California Retina Consultants
    • Beverly Hills, California, United States, 90211
      • Retina-Vitreous Associates Medical Group
    • Mountain View, California, United States, 94040
      • Northern California Retina Vitreous Associates Medical Group Inc
    • Poway, California, United States, 92064
      • Retina Consultants San Diego
    • Santa Barbara, California, United States, 93103
      • California Retina Consultants
  • Georgia
    • Augusta, Georgia, United States, 30909
      • Southeast Retina Center PC
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins University
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Ophthalmic Consultants of Boston
  • Minnesota
    • Edina, Minnesota, United States, 55435
      • Vitreoretinal Surgery PLLC
  • Nevada
    • Reno, Nevada, United States, 89502
      • Sierra Eye Associates
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • Vision Research Center Eye Associates of New Mexico
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Retinovitreous Associates, LTD
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Charles Retina Institute, P.C
    • Nashville, Tennessee, United States, 37203
      • Tennessee Retina, PC
  • Texas
    • The Woodlands, Texas, United States, 77384
      • Retina Consultants of Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Study Population

The sample sizes were determined by the parent studies and all participants who received an SCS administration of RGX-314 will be invited to participate in the long-term follow-up study. All Enrolled Population: Includes all participants who are enrolled in the long-term follow-up study.

Description

Inclusion Criteria:

1. Must provide written, signed informed consent for this study.
2. Must have been enrolled in a previous clinical study of ABBV-RGX-314 in the treatment of nAMD and must have received an SCS injection of ABBV-RGX-314 in that study.
3. Must be willing and able to comply with all study procedures.

Exclusion Criteria:

1. None

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Standard of Care; Dose Level (DL) 1-3 participants who received ABBV-RGX- 314 in the parent study will continue to receive standard of care for the study duration.	Drug: Standard of Care; Standard of Care
Other: Supplemental Treatment; DL 4 participants from the parent study will be able to receive supplement ranibizumab based on protocol-specified criteria if they develop worsening nAMD activity.	Drug: Standard of Care; Standard of Care; Drug: Ranibizumab; Injection; Other Names: LUCENTIS

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidences of ocular Adverse Events and any Serious Adverse Events	To evaluate the long-term safety of ABBV-RGX-314	5 years inclusive of parent study

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidences of non-ocular Adverse Events and any Adverse Events of Special Interest	To evaluate the long-term safety of ABBV-RGX-314	5 years inclusive of parent study
Mean change from baseline (ie, prior to ABBV-RGX-314 administration in the parent study) in BCVA based on ETDRS score in the study eye at assessed timepoints	To evaluate the persistence of effect of ABBV-RGX-314 on best corrected visual acuity (BCVA)	5 years inclusive of parent study
Mean change from baseline (ie, prior to ABBV-RGX-314 administration in the parent study) in CRT as measured by SD-OCT in the study eye at assessed timepoints	To evaluate the persistence of effect of ABBV-RGX-314 on central retinal thickness (CRT), as measured by spectral domain-optical coherence tomography (SD-OCT)	5 years inclusive of parent study
Mean number of supplemental anti-VEGF injections through years 2, 3, 4 and 5	To assess the need for supplemental anti-vascular endothelial growth factor (anti-VEGF) therapy	5 years inclusive of parent study
Mean number of supplemental anti-VEGF injections in years 2, 3, 4, and 5	To assess the need for supplemental anti-vascular endothelial growth factor (anti-VEGF) therapy	5 years inclusive of parent study
Mean supplemental anti-VEGF injection annualized rate through years 2, 3, 4, and 5	To assess the need for supplemental anti-vascular endothelial growth factor (anti-VEGF) therapy	5 years inclusive of parent study
Mean supplemental anti-VEGF injection annualized rate in years 2, 3, 4, and 5	To assess the need for supplemental anti-vascular endothelial growth factor (anti-VEGF) therapy	5 years inclusive of parent study
Mean number of retina specialist visits attended for nAMD through years 2, 3, 4, and 5	To assess the need of clinic visits for management of nAMD	5 years inclusive of parent study
Mean number of retina specialist visits attended for nAMD in years 2, 3, 4, and 5	To assess the need of clinic visits for management of nAMD	5 years inclusive of parent study

Collaborators and Investigators

• Sponsor: AbbVie
• Collaborators: REGENXBIO Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2021
• Primary Completion (Estimated): July 1, 2031
• Study Completion (Estimated): July 1, 2031

Study Registration Dates

• First Submitted: January 14, 2022
• First Submitted That Met QC Criteria: January 14, 2022
• First Posted (Actual): January 27, 2022

Study Record Updates

• Last Update Posted (Actual): August 5, 2026
• Last Update Submitted That Met QC Criteria: August 3, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: Gene Therapy; AMD; wet AMD; wAMD; nAMD
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Wet Macular Degeneration; Health Services Administration; Health Care Quality, Access, and Evaluation; Amino Acids, Peptides, and Proteins; Proteins; Quality of Health Care; Quality Indicators, Health Care; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Ranibizumab; Standard of Care
• Other Study ID Numbers: RGX-314-5102 (M23-421); M23-421 (Other Identifier: AbbVie Inc)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.
• IPD Sharing Time Frame: For details on when studies are available for sharing, visit https://vivli.org/ourmember/abbvie/
• IPD Sharing Access Criteria: To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No