RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)
AAVIATE
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)
2 other identifiers
interventional
146
1 country
18
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Aug 2020
Longer than P75 for phase_2
18 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 4, 2020
CompletedFirst Posted
Study publicly available on registry
August 17, 2020
CompletedStudy Start
First participant enrolled
August 25, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 1, 2027
February 27, 2026
February 1, 2026
5.9 years
August 4, 2020
February 25, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean change from baseline in BCVA to Week 40 based on the Early Treatment Diabetic Retinopathy Study (ETDRS) score
The scale used is the early treatment diabetic retinopathy study (ETDRS) letter score from 0-100 and higher score being better vision.
40 weeks
Secondary Outcomes (14)
Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs)
52 weeks
Vector shedding analysis in serum, urine, and tears
52 Weeks
Proportion of participants who experience ocular inflammation following SCS ABBV-RGX-314 administration
52 weeks
Mean change from baseline in choroidal neovascularization (CNV) lesion size and leakage area based on fluorescein angiography (FA)
Up to Week 80
Mean change from baseline in BCVA based on ETDRS score
Up to Week 80
- +9 more secondary outcomes
Study Arms (7)
Ranibizumab control
ACTIVE COMPARATORControl treatment arm
ABBV-RGX-314 Treatment Arm (Dose 1)
EXPERIMENTALABBV-RGX-314 Dose 1
ABBV-RGX-314 Treatment Arm (Dose 2)
EXPERIMENTALABBV-RGX-314 Dose 2
ABBV-RGX-314 Treatment Arm (Dose 3)
EXPERIMENTALABBV-RGX-314 Dose 3
ABBV-RGX-314 Treatment Arm (Dose 3) and Local Steroid
EXPERIMENTALABBV-RGX-314 Dose 3 and Local Steroid
ABBV-RGX-314 Treatment Arm (Dose 3) and Topical Steroid
EXPERIMENTALABBV-RGX-314 Dose 3 and Topical Steroid
ABBV-RGX-314 Treatment Arm (Dose 4) and Topical Steroid
EXPERIMENTALABBV-RGX-314 Dose 4 and Topical Steroid
Interventions
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)
Topical steroid
AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)
Eligibility Criteria
You may qualify if:
- Age \>/= 50 and \</= 89
- Diagnosis of CNV secondary to age-related macular degeneration in the study eye.
- Participants must have demonstrated a meaningful response to anti-VEGF therapy.
- Willing and able to provide written, signed informed consent for this study.
You may not qualify if:
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Subfoveal fibrosis or atrophy in study eye.
- Participants who have had a prior vitrectomy.
- Active or history of retinal detachment in the study eye.
- History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry.
- Received any gene therapy.
- Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract.
- History of intraocular surgery in the study eye within 12 weeks of study entry.
- Receipt of any IP within 30 days of study entry or 5 half-lives of the IP.
- Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry.
- Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye.
- COHORT 6 AND 7 ONLY:
- Active or history of glaucoma or ocular hypertension (steroid-induced ocular hypertension (Cohort 7 only)) in the study eye.
- Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AbbVielead
- REGENXBIO Inc.collaborator
Study Sites (18)
Retinal Research Institute /ID# 255925
Phoenix, Arizona, 85053, United States
California Retina Consultants - Bakersfield /ID# 255910
Bakersfield, California, 93309-1677, United States
Retina Vitreous Assoc Med Grp /ID# 255921
Beverly Hills, California, 90211, United States
Retinal Diagnostic Center /ID# 272275
Campbell, California, 95008, United States
Northern California Retina Vitreous Associates Medical Group, Inc /ID# 255920
Mountain View, California, 94040-4101, United States
Retina Consultants of San Diego /ID# 255911
Poway, California, 92064-2530, United States
California Retina Consultants - Santa Barbara /ID# 255923
Santa Barbara, California, 93103, United States
Southeast Retina Center /ID# 255912
Augusta, Georgia, 30909, United States
Springfield Clinic - First /ID# 272274
Springfield, Illinois, 62702, United States
Johns Hopkins Hospital /ID# 255919
Baltimore, Maryland, 21287, United States
Ophthalmic Consultants of Boston /ID# 255917
Boston, Massachusetts, 02114, United States
Sierra Eye Associates /ID# 255908
Reno, Nevada, 89502, United States
Eye Associates of New Mexico /ID# 255915
Albuquerque, New Mexico, 87109, United States
Duke University Medical Center /ID# 267646
Durham, North Carolina, 27710, United States
Mid Atlantic Retina /ID# 255906
Philadelphia, Pennsylvania, 19107, United States
Charles Retina Institute /ID# 255922
Germantown, Tennessee, 38138, United States
Tennessee Retina - Nashville /ID# 255918
Nashville, Tennessee, 37203-1513, United States
Retina Consultants - The Woodlands /ID# 255924
The Woodlands, Texas, 77384, United States
Related Links
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
ABBVIE INC.
AbbVie
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- OUTCOMES ASSESSOR
- Masking Details
- The vision examiners and central reading center (CRC) graders will be masked, meaning they will be unaware of the participants' treatment assignment. All other individuals affiliated with the study (investigators, all study center personnel apart from the vision examiners, the participants, all staff of Sponsor and its research collaborator, all staff affiliated with the contract research organization, and all CRC staff apart from the CRC graders) will have knowledge of the treatment assignment.
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 4, 2020
First Posted
August 17, 2020
Study Start
August 25, 2020
Primary Completion (Estimated)
August 1, 2026
Study Completion (Estimated)
February 1, 2027
Last Updated
February 27, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share