A Long-term Follow up Study of EXG102-031 in Patients With wAMD (Everest LTFU)

April 3, 2026 updated by: Exegenesis Bio

An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy With EXG102-031 in Participants With Neovascular Age-related Macular Degeneration

In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD. This study is designed to fulfill the long-term safety monitoring of EXG102-031. Participants that enroll in this long-term follow-up study have been treated with EXG102-031 under the main study (EXG102-031-211).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

Age-related macular degeneration (AMD) is a major cause of blindness and visual impairment in older adults. The wet form of AMD, also called neovascular AMD (nAMD) usually causes faster vision loss than the dry form. The most common current treatments of nAMD are products that inhibit vascular endothelial growth factor (VEGF) (including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron) and are delivered by intravitreal injections at 4 to 16 week intervals and continued indefinitely. The Phase I, open-label, multiple-cohort, dose-escalation study designed to evaluate the safety and tolerability of EXG102-031 gene therapy in subjects with previously treated nAMD (EXG102-031-211). In the main study safety was assessed over 52 weeks after the administration of EXG102-031. This long-term follow-up study will assess safety for an additional 36 months for all participants enrolled in the main study. Participants will return to the clinic regularly for ocular assessments including assessment of whether supplemental therapy with aflibercept is needed.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Nevada
      • Reno, Nevada, United States, 89502
        • Sierra Eye Associates
    • Pennsylvania
      • Erie, Pennsylvania, United States, 16507
        • Erie Retina Research

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Must have been previously enrolled in the parent study (EXG102-031-211) and must have received subretinal EXG102-031 in the parent study;
  • Are willing and able to sign the study written informed consent form;
  • Must be willing and able to comply with all study procedures.

There are no exclusion Criteria in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: All participants who completed the main study of EXG102-031
All subjects who enroll in this LTFU study have received the experimental treatment with EXG102-031 in the main study, however NO experimental therapy is being administered in this study. The intervention with Aflibercept IVT PRN in this study is NOT experimental.
Biological: Aflibercept (2.0 mg)
PRN IVT Aflibercept

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Evaluation of long-term safety by measuring frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs) through Month 36 (Year 4) post EXG102-031 administration.
Time Frame: Month 36
Month 36

Secondary Outcome Measures

Outcome Measure
Time Frame
Evaluation of potential efficacy by measuring change from baseline in best corrected visual acuity (BCVA) measured using the ETDRS method and average number of doses of aflibercept supplemental therapy through Month 36 (Year 4)
Time Frame: Month 36
Month 36

Other Outcome Measures

Outcome Measure
Time Frame
Anatomical characteristics of EXG102-031 by measuring the change from baseline in central retinal thickness as measured by SD-OCT at Month 36 (Year 4)
Time Frame: Month 36
Month 36
Pharmacodynamic characteristics of EXG102-031 by measuring the transgene expression in aqueous fluid at Month 36 (Year 4)
Time Frame: Month 36
Month 36

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Exegenesis Bio

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 10, 2024

Primary Completion (Estimated)

February 1, 2029

Study Completion (Estimated)

February 1, 2029

Study Registration Dates

First Submitted

January 30, 2025

First Submitted That Met QC Criteria

February 5, 2025

First Posted (Actual)

February 10, 2025

Study Record Updates

Last Update Posted (Actual)

April 8, 2026

Last Update Submitted That Met QC Criteria

April 3, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EXG102-031-211-LTFU

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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