A Study of EXG102-031 in Patients With wAMD (Everest)

NCT05903794 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: EXG102-031 (211)
• 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.

Conditions

Neovascular (Wet) Age-related Macular Degeneration (nAMD)

Outcome Measures

Primary Outcomes (1)

• Evaluation of safety and tolerability

  Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)

  Throughout 52 weeks

Secondary Outcomes (3)

• Evaluation of potential efficacy

  52 weeks post administration

• Evaluation of potential safety

  Through week 24

• Evaluation of supplementary therapy injections received

  Throughout 52 weeks post administration

Study Arms (1)

EXG102-031

EXPERIMENTAL

Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of four dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into.

Biological: EXG102-031

Interventions

EXG102-031 BIOLOGICAL

EXG102-031 is a recombinant adeno-associated virus (rAAV) expressing an angiopoietin domain and VEGF receptor (ABD-VEGFR) fusion protein. EXG102-031 will be administered by subretinal injecting into one eye of each participant.

EXG102-031

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, age ≥ 50 years of age;
• Diagnosis of nAMD and current active lesion in the study eye at Screening;
• An ETDRS BCVA letter scores between 73 and 19 letters in the study eye;
• Response to anti-VEGF treatment during Screening;
• The study eye must be a pseudophakic lens (post-cataract surgery status); and
• Voluntarily agree to participate in the clinical trial, understand the trial procedures, and be capable of signing the informed consent form before screening.

You may not qualify if:

• Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye;
• Presence in the study eye of CNV or macular edema due to causes other than AMD;
• Presence in the study eye of scarring, fibrosis or atrophy involving the macula;
• Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters;
• Active ocular infection in either eye;
• Presence of advanced glaucoma or uncontrolled glaucoma in the study eye;
• History of intraocular surgery in the study eye within 90 days of screening; or
• Prior receipt of any ocular or systemic gene therapy agent.

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

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Arshad Khanani, MD

  Sierra Eye Associates

  PRINCIPAL INVESTIGATOR

Study Design

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

Model Details: Dose-escalation Study with four cohorts of three participants in each.

Study Record Dates

• First Submitted: June 6, 2023
• First Posted: June 15, 2023
• Study Start: July 24, 2023
• Primary Completion: February 16, 2026
• Study Completion: February 16, 2026
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)