Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD — ASCENT  

Wet Age-related Macular Degeneration Clinical Trial

Official title: A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD

Status Recruiting

Clinical Trial Summary

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD.

Clinical Trial Description

This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 gene therapy relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to aflibercept. Approximately 465 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms. ;

Related Conditions & MeSH terms

• AMD
• CNV
• Macular Degeneration
• nAMD
• wAMD
• Wet Age-related Macular Degeneration
• WetAMD

• NCT number: NCT05407636
• Study type: Interventional
• Source: AbbVie
• Contact: Patient Advocacy
• Phone: +(1) 866-860-0117
• Email: Patientadvocacy@regenxbio.com
• Status: Recruiting
• Phase: Phase 3
• Start date: December 28, 2021
• Completion date: December 2025