NCT05407636

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

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 660 (estimated)
• Sponsor: AbbVie · Industry
• Sex: All
• Age: 50 Years – 89 Years
• Healthy volunteers: Not accepted

Summary

ABBV-RGX-314 (also known as 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-vascular endothelial growth factor (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. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

Detailed description

This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of ABBV-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 ABBV-RGX-314 relative to aflibercept. Approximately 660 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms. A bilateral treatment substudy conducted at US sites is an open-label, partially randomized, parallel arm study to evaluate the safety and efficacy of subretinal ABBV-RGX-314 administered bilaterally in participants who have bilateral nAMD. Previously treated crossover participants from the control arm of the main study who crossed over and received ABBV-RGX-314 in the study eye will receive the same ABBV-RGX-314 dose in the contralateral eye (ie, same dose as in the study eye), and newcomers (participants who have not been randomized in an ABBV-RGX-314 study) and untreated crossover participants (ongoing control participants in the main study who have completed Week 54 but have not crossed over to receive ABBV-RGX-314 in the main study) will be randomized in a 2:1 ratio to receive ABBV-RGX-314 Dose 1 or ABBV-RGX-314 Dose 2 in both eyes. Up to 15 participants who qualify for the substudy will be enrolled and followed for a minimum of 50 weeks.

Conditions

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

Interventions

Type	Name	Description
GENETIC	ABBV-RGX-314 Dose 1	AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
GENETIC	ABBV-RGX-314 Dose 2	AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
BIOLOGICAL	Aflibercept (EYLEA®)	2.0 mg (0.05 mLsolution) administered by intravitreal injection approximately every 8 weeks after 3 monthly injections

Timeline

• Start date: 2022-01-13
• Primary completion: 2026-10-01
• Completion: 2027-11-01
• First posted: 2022-06-07
• Last updated: 2025-09-25

Locations

181 sites across 10 countries: United States, Canada, France, Germany, Hungary, Italy, Japan, Puerto Rico, Spain, United Kingdom

Regulatory

• FDA-regulated drug study