ADVM-022 Intravitreal Gene Therapy for Wet AMD

NCT ID: NCT03748784

Last Updated: 2023-08-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-11-14
• Study Completion Date: 2022-06-22

Brief Summary

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.

Detailed Description

This open-label, multicenter, dose-ranging study will evaluate 2 dose levels in up to 30 subjects (15 per dose) with active choroidal neovascularization (CNV) secondary to AMD. Subjects who are under active anti-VEGF treatment and have demonstrated a meaningful response to anti-VEGF therapy will be considered for participation in this study. The primary endpoint for this study is safety and tolerability of ADVM-022. All subjects will continue to be assessed for 104 weeks following treatment with ADVM-022.

Conditions

Wet Age-related Macular Degeneration; Neovascular Age-related Macular Degeneration

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Dose 1

6E11 vg of ADVM-022

Group Type: EXPERIMENTAL

ADVM-022

Intervention Type: BIOLOGICAL

ADVM-022 (AAV.7m8-aflibercept) is a recombinant, replication-deficient adeno-associated virus (AAV.7m8) gene therapy vector carrying a coding sequence for aflibercept

Dose 2

2E11 vg of ADVM-022

Group Type: EXPERIMENTAL

ADVM-022

Intervention Type: BIOLOGICAL

ADVM-022 (AAV.7m8-aflibercept) is a recombinant, replication-deficient adeno-associated virus (AAV.7m8) gene therapy vector carrying a coding sequence for aflibercept

Interventions

ADVM-022

ADVM-022 (AAV.7m8-aflibercept) is a recombinant, replication-deficient adeno-associated virus (AAV.7m8) gene therapy vector carrying a coding sequence for aflibercept

Intervention Type: BIOLOGICAL

Other Intervention Names

AAV.7m8-aflibercept

Eligibility Criteria

Inclusion Criteria

• Age ≥ 50
• Diagnosis of neovascular (wet) AMD
• BCVA ETDRS Snellen equivalent between ≤20/32 and ≥20/320 for each cohort
• Subjects must be under active anti-VEGF treatment for wAMD and received a minimum of 2 injections within 4 months prior to screening
• Demonstrated a meaningful response to anti-VEGF therapy
• Willing and able to provide consent

Exclusion Criteria

• History of retinal disease in the study eye other than wet AMD
• Fibrosis or atrophy, retinal epithelial tear in the center of the fovea in the study eye, or any condition preventing visual acuity improvement
• History of retinal detachment (with or without repair) in the study eye
• History of vitrectomy, trabeculectomy, or other filtration surgery in the study eye
• Uncontrolled glaucoma in the study eye
• Any prior treatment with photodynamic therapy or retinal laser for the treatment of wet AMD and any previous therapeutic radiation in the region of the study eye
• Any previous intraocular or periocular surgery on the study eye within 6 months
• Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months
• Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg

• Minimum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Adverum Biotechnologies, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

OPTIC Medical Monitor

Role: STUDY_CHAIR

Adverum Biotechnologies, Inc.

Locations

Adverum Clinical Site

Bakersfield, California, United States

Adverum Clinical Site

Beverly Hills, California, United States

Adverum Clinical Site

Golden, Colorado, United States

Adverum Clinical Site

Deerfield Beach, Florida, United States

Adverum Clinical Site

Reno, Nevada, United States

Adverum Clinical Site

Philadelphia, Pennsylvania, United States

Adverum Clinical Site

West Columbia, South Carolina, United States

Adverum Clinical Site

Nashville, Tennessee, United States

Adverum Clinical Site

Abilene, Texas, United States

Adverum Clinical Site

Houston, Texas, United States

Adverum Clinical Site

The Woodlands, Texas, United States

Countries

United States

References

Khanani AM, Boyer DS, Wykoff CC, Regillo CD, Busbee BG, Pieramici D, Danzig CJ, Joondeph BC, Major JC Jr, Turpcu A, Kiss S. Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study. EClinicalMedicine. 2023 Dec 22;67:102394. doi: 10.1016/j.eclinm.2023.102394. eCollection 2024 Jan.

Reference Type: DERIVED

PMID: 38152412 (View on PubMed)

Other Identifiers

ADVM-022-01

Identifier Type: -

Identifier Source: org_study_id