NCT04514653

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)

A Phase 2, Randomized, Dose-escalation, Ranibizumab-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via One or Two Suprachoroidal Space (SCS) Injections in Participants With Neovascular Age-Related Macular Degeneration (nAMD) (AAVIATE)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 146 (actual)
• Sponsor: AbbVie · Industry
• Sex: All
• Age: 50 Years – 89 Years
• Healthy volunteers: Not accepted

Summary

This interventional study is being conducted with an investigational gene therapy treatment called ABBV-RGX-314 (also known as RGX-314) and is being developed as a potential one-time gene therapy treatment for neovascular (wet) age-related macular degeneration (wet AMD or nAMD). The typical treatment for nAMD is frequent injections of anti-VEGF therapy. Researchers are testing ABBV-RGX-314 to see if it has similar effects as the current approved standard of care, such as Lucentis® or Eylea® injections. The duration of this study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314 after week 52, up to 80 weeks post-randomization. The primary outcome measure for this investigational study is to evaluate the mean change in best-corrected visual acuity (BCVA) for ABBV-RGX-314 compared with ranibizumab monthly at the Week 40 visit.

Detailed description

This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 gene therapy in subjects with nAMD. ABBV-RGX-314 will be injected into the SCS part of the eye. Approximately 140 participants who meet the inclusion/exclusion criteria will be enrolled into one of 7 cohorts. Participants will be randomized in Cohorts 1 and 2 to receive ABBV-RGX-314 or the intravitreal ranibizumab control. Participants enrolled in Cohorts 3 through 5 will receive ABBV-RGX-314. Participants enrolled in Cohort 6 will receive ABBV-RGX-314 and will be randomized to one of two different post-procedural steroid regimens. Participants randomized in Cohort 7 will receive ABBV-RGX-314 with a protocol mandated steroid regimen or the intravitreal ranibizumab control. Cohort 1 will evaluate ABBV-RGX-314 Dose 1, Cohorts 2 and 3 will evaluate ABBV-RGX-314 Dose 2, Cohorts 4, 5, and 6 will evaluate ABBV-RGX-314 Dose 3, and Cohort 7 will evaluate ABBV-RGX-314 Dose 4. The duration of the study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314, up to 80 weeks post-randomization.

Conditions

• Neovascular Age-Related Macular Degeneration (nAMD)

Interventions

Type	Name	Description
BIOLOGICAL	Ranibizumab	Ranibizumab (anti-VEGF agent)
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)
GENETIC	ABBV-RGX-314 Dose 3	AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)
DRUG	Local Steroid	Local steroid
DRUG	Topical Steroid	Topical steroid
GENETIC	ABBV-RGX-314 Dose 4	AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)

Timeline

• Start date: 2020-08-25
• Primary completion: 2026-08-01
• Completion: 2027-02-01
• First posted: 2020-08-17
• Last updated: 2026-02-27

Locations

18 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study