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

NCT06817343 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: EXG102-031-211-LTFU
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

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).

Conditions

Neovascular Age-Related Macular Degeneration (nAMD); Neovascular (Wet) Age-related Macular Degeneration (AMD)

Keywords

EXG102-031; wAMD

Outcome Measures

Primary Outcomes (1)

• 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.

  Month 36

Secondary Outcomes (1)

• 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)

  Month 36

Other Outcomes (2)

• 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)

  Month 36

• Pharmacodynamic characteristics of EXG102-031 by measuring the transgene expression in aqueous fluid at Month 36 (Year 4)

  Month 36

Study Arms (1)

All participants who completed the main study of EXG102-031

OTHER

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)

Interventions

Aflibercept (2.0 mg) BIOLOGICAL

PRN IVT Aflibercept

All participants who completed the main study of EXG102-031

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• Exegenesis Bio: lead

Study Sites (2)

Sierra Eye Associates

Reno, Nevada, 89502, United States

Location

Erie Retina Research

Erie, Pennsylvania, 16507, United States

Location

MeSH Terms

Conditions

Macular Degeneration

Interventions

aflibercept

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants from the main study, EXG102-031 (211), will rollover to this study and will be offered Aflibercept IVT PRN to control wAMD symptoms.

Study Record Dates

• First Submitted: January 30, 2025
• First Posted: February 10, 2025
• Study Start: September 10, 2024
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): February 1, 2029
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)