ADVM-022 Intravitreal Gene Therapy for Wet AMD (OPTIC)

Adverum Biotechnologies

Status and phase

Completed

Phase 1

Conditions

Wet Age-related Macular Degeneration

Neovascular Age-related Macular Degeneration

Treatments

Biological: ADVM-022

Study type

Interventional

Funder types

Industry

Identifiers

NCT03748784

ADVM-022-01

Details and patient eligibility

About

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.

Full 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.

Enrollment

30 patients

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

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