Long-term Study of ADVM-022 in Neovascular (Wet) AMD [OPTIC-EXT]

A Long-term Study of ADVM-022 in Neovascular (Wet) AMD - OPTIC Extension

ADVM-022-07 is an observational long-term extension (OPTIC-EXT) study assessing safety and efficacy of ADVM-022 gene therapy product, in participants with neovascular, or exudative (wet), age-related macular degeneration (nAMD).

Study Overview

• Status: Active, not recruiting
• Conditions: Neovascular Age-related Macular Degeneration; Wet Age-related Macular Degeneration
• Intervention / Treatment: Biological: ADVM-022

Detailed Description

ADVM-022 (AAV.7m8-aflibercept) is a gene therapy product developed for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is a serious condition and the leading cause of blindness in the elderly. The available therapies for treating wet AMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy. A one-time IVT administration of ADVM-022 has the potential to treat wet AMD by providing durable expression of therapeutic levels of intraocular anti-VEGF protein (aflibercept) and maintaining the vision of patients. ADVM-022 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with wet AMD receiving anti-VEGF therapy in clinical practice.

ADVM-022-07 is an observational long-term extension (OPTIC-EXT) study assessing safety and efficacy of ADVM-022 gene therapy product in participants with neovascular or exudative (wet), age-related macular degeneration (nAMD) previously treated in the OPTIC parent study (Clinical Protocol No. ADVM-022-01 [OPTIC] - NCT03748784). There is no investigational treatment administered in this study.

In Part 1 of the OPTIC-EXT study participants will roll over from the OPTIC parent study and will be followed for 3 additional years (following 2-years of assessment period in the OPTIC parent study). In Part 2 of the OPTIC-EXT study consenting participants will have 5 annual in-clinic assessments for an additional 5 years of long-term follow-up following the completion of OPTIC-EXT (Part 1). As such participants who complete the parent study as well as Part 1 and Part 2 of the OPTIC-EXT study will have had 10 years of total long-term follow-up.

• Study Type: Observational
• Enrollment (Actual): 23

Contacts and Locations

Study Locations

• United States
  • California
    • Bakersfield, California, United States, 93309
      • Adverum Clinical Site
    • Beverly Hills, California, United States, 90211
      • Adverum Clinical Site
  • Colorado
    • Lakewood, Colorado, United States, 80228
      • Adverum Clinical Site
  • Florida
    • Deerfield Beach, Florida, United States, 33064
      • Adverum Clinical Site
  • Nevada
    • Reno, Nevada, United States, 89502
      • Adverum Clinical Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Adverum Clinical Site
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Adverum Clinical Site
  • Texas
    • Bellaire, Texas, United States, 77401
      • Adverum Clinical Site
    • The Woodlands, Texas, United States, 77384
      • Adverum Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

• Participants who received ADVM-022 at any dose in the OPTIC study
• Up to 30 participants from all cohorts of OPTIC

Description

Inclusion Criteria:

• Participants who received a single dose of ADVM-022 at any dose in the OPTIC study
• Willing and able to provide informed consent

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
1; Participants with wet AMD who received any dose of ADVM-022 in a prior clinical study.	Biological: ADVM-022; Long term follow-up of subjects that received ADVM-022; Other Names: AAV.7m8-aflibercept

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Severity and incidence of ocular and systemic adverse events (AEs).	Severity and incidence of ocular and systemic adverse events	416 weeks (8 years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change in best corrected visual acuity (BCVA) from baseline, over time	Mean change in best corrected visual acuity (BCVA) from baseline, over time	416 weeks (8 years)
Mean change in central subfield thickness (CST) and macular volume from baseline, over time	Mean change in central subfield thickness (CST) and macular volume from baseline, over time	416 weeks (8 years)
Mean number of supplemental aflibercept injections over time	Mean number of supplemental aflibercept injections over time	416 weeks (8 years)
Percentage of participants requiring supplemental bolus aflibercept over time	Percentage of participants requiring supplemental bolus aflibercept over time	416 weeks (8 years)
Time to first supplemental aflibercept requirement	Time to first supplemental aflibercept requirement	416 weeks (8 years)
Percentage of participants without intraretinal fluid (IRF) by spectral domain optical coherence tomography (SD-OCT), over time	Percentage of participants without intraretinal fluid (IRF) by spectral domain optical coherence tomography (SD-OCT), over time	416 weeks (8 years)
Percentage of participants without subretinal fluid (SRF) by SD-OCT over time	Percentage of participants without subretinal fluid (SRF) by SD-OCT over time	416 weeks (8 years)
Time to first dry retina (defined as no IRF or SRF by SD-OCT)	Time to first dry retina (defined as no IRF or SRF by SD-OCT)	416 weeks (8 years)
Duration of fluid free status (defined as no IRF or SRF by SD-OCT)	Duration of fluid free status (defined as no IRF or SRF by SD-OCT)	416 weeks (8 years)
Percentage of participants developing geographic atrophy over time (as assessed by multiple imaging modalities)	Percentage of participants developing geographic atrophy over time (as assessed by multiple imaging modalities)	416 weeks (8 years)
Growth of geographic atrophy over time, as assessed by multiple imaging modalities	Growth of geographic atrophy over time, as assessed by multiple imaging modalities	416 weeks (8 years)
Percentage of participants with CST fluctuations > 50 μm over time	Percentage of participants with CST fluctuations > 50 μm over time	416 weeks (8 years)

Collaborators and Investigators

• Sponsor: Adverum Biotechnologies, Inc.
• Investigators: Study Director: OPTIC-EXT Medical Monitor, Adverum Biotechnologies, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 14, 2020
• Primary Completion (Estimated): June 26, 2030
• Study Completion (Estimated): June 26, 2030

Study Registration Dates

• First Submitted: November 20, 2020
• First Submitted That Met QC Criteria: November 20, 2020
• First Posted (Actual): November 27, 2020

Study Record Updates

• Last Update Posted (Actual): February 10, 2026
• Last Update Submitted That Met QC Criteria: February 6, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Eye Diseases; AMD; AAV; Gene therapy; Age-Related Macular Degeneration; Retinal Diseases; Neovascular age-related macular degeneration; wet AMD; CNV; Blindness; Choroidal Neovascularization; ADVM-022; AAV.7m8; Anti-VEGF therapy; Aflibercept (Eylea); AAV Vector; Adverum; Eye disease; Retinal Degeneration; wAMD; Wet Macular Degeneration; nAMD; OPTIC Study; ADVM-022-01; ADVM-022-07; OPTIC Extension; OPTIC-EXT; Long-term Follow Up; Long-term Extension; Ixoberogene soroparvovec; Ixo-vec; Adeno-associated viruses
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Eye Diseases, Hereditary; Vision Disorders; Sensation Disorders; Uveal Diseases; Choroid Diseases; Metaplasia; Neovascularization, Pathologic; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Retinal Diseases; Eye Diseases; Retinal Degeneration; Macular Degeneration; Choroidal Neovascularization; Wet Macular Degeneration; Blindness; Physiological Effects of Drugs; Antineoplastic Agents; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Aflibercept
• Other Study ID Numbers: ADVM-022-07

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