NCT07482176

Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)

A Multi-center, Randomized, Double-masked, Active-comparator-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 284 (estimated)
• Sponsor: Adverum Biotechnologies, Inc. · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to intravitreal aflibercept (active comparator). The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured as an average at Weeks 52 and 56. Safety, tolerability, and efficacy will be evaluated throughout the study.

Detailed description

The primary objective of this study is to evaluate the non-inferiority in efficacy of a single IVT injection of Ixo-vec 6 x 10\^10 vector genome (vg)/eye compared to an active comparator. Non-inferiority will be evaluated using a pre-specified margin defined in the protocol. Neovascular AMD is a degenerative ocular disease associated with the infiltration of abnormal blood vessels in the retina from the underlying choroid layer and is a leading cause of blindness in patients over 65 years of age. The abnormal angiogenic process in nAMD is stimulated and modulated by vascular endothelial growth factor (VEGF). Treatment of nAMD requires frequent IVT injections of VEGF inhibitors (anti-VEGF) administered every 4-16 weeks. Ixo-vec (also known as ADVM-022 or AAV.7m8-aflibercept) is an adeno-associated virus (AAV)-based gene therapy product being developed for the treatment of nAMD. Ixo-vec is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice. Safety, tolerability, and efficacy will be evaluated throughout this study. The primary endpoint of this study is the mean change in BCVA of Ixo-vec compared to an active comparator measured as an average at Weeks 52 and 56 post-treatment. Due to the long duration of the Screening period, this study will be considered fully enrolled when randomization has been completed.

Conditions

• Neovascular Age-Related Macular Degeneration (nAMD)
• Wet AMD

Interventions

Type	Name	Description
GENETIC	Ixo-vec	Ixo-vec will be administered intravitreally.
DRUG	Aflibercept	Aflibercept will be administered intravitreally.

Timeline

• Start date: 2026-03-16
• Primary completion: 2027-11-15
• Completion: 2031-10-20
• First posted: 2026-03-19
• Last updated: 2026-03-19

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study