Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD (ATMOSPHERE)

A Randomized, Partially Masked, Controlled, Phase 2b/3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)

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 or nAMD). 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 (anti-VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to maintain or prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every 4 to 16 weeks in frequency, to maintain efficacy. Due to the burden of these treatments, patients often experience a decline in vision with reduced frequency of treatment over time.

Study Overview

• Status: Active, not recruiting
• Conditions: Choroidal Neovascularization; Neovascular Age-related Macular Degeneration; CNV; AMD; nAMD; Wet AMD; Wet Age-related Macular Degeneration; wAMD; Neovascular AMD
• Intervention / Treatment: Biological: Ranibizumab (LUCENTIS®); Genetic: ABBV-RGX-314; Genetic: ABBV-RGX-314

Detailed Description

This randomized, partially masked, active-controlled, Phase 2b/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 ABBV-RGX-314 relative to an active comparator. The primary endpoint of this study is the mean change from baseline in best-corrected visual acuity (BCVA) of ABBV-RGX-314 relative to ranibizumab at Week 54. Approximately 630 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.

• Study Type: Interventional
• Enrollment (Actual): 671
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85053
      • Retinal Research Institute /ID# 256019
    • Sun City, Arizona, United States, 85351
      • Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences /ID# 271290
  • California
    • Beverly Hills, California, United States, 90211
      • Retina Vitreous Assoc Med Grp /ID# 256299
    • Campbell, California, United States, 95008
      • Retinal Diagnostic Center /ID# 256137
    • Encino, California, United States, 91436
      • The Retina Partners - Encino /ID# 256054
    • Fullerton, California, United States, 92835
      • Retina Consultants of Orange County /ID# 256152
    • Huntington Beach, California, United States, 92647
      • Salehi Retina Institute /ID# 263485
    • Irvine, California, United States, 92697
      • UC Irvine/Gavin Herbert Eye Institute /ID# 256145
    • Mountain View, California, United States, 94040-4101
      • Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298
    • Pasadena, California, United States, 91105
      • UCLA Doheny Eye Center /ID# 256120
    • Pasadena, California, United States, 91107
      • California Eye Specialists Medical Group Inc. /ID# 256079
    • Poway, California, United States, 92064-2530
      • Retina Consultants of San Diego /ID# 256021
    • Sacramento, California, United States, 95825
      • Retinal Consultants Medical Group /ID# 256047
    • San Francisco, California, United States, 94107
      • West Coast Retina /ID# 256448
    • San Francisco, California, United States, 94143
      • University of California, San Francisco /ID# 256130
    • Santa Ana, California, United States, 92705
      • Orange County Retina Medical Group /ID# 256073
    • Santa Barbara, California, United States, 93103
      • California Retina Consultants - Santa Barbara /ID# 256017
  • Colorado
    • Colorado Springs, Colorado, United States, 80909
      • Retina Consultants of Southern Colorado /ID# 256069
    • Durango, Colorado, United States, 81303
      • Southwest Retina Research Center /ID# 256136
    • Lakewood, Colorado, United States, 80228
      • Colorado Retina Associates /ID# 256121
  • Connecticut
    • Waterford, Connecticut, United States, 06385-1215
      • Retina Group of New England - Waterford /ID# 256071
  • Florida
    • Deerfield Beach, Florida, United States, 33064-1342
      • Advanced Research, LLC /ID# 275451
    • Gainesville, Florida, United States, 32607
      • Vitreoretinal Associates, P.A. /ID# 256150
    • Lakeland, Florida, United States, 33805
      • Florida Retina Consultants /ID# 265661
    • Miami, Florida, United States, 33136
      • Bascom Palmer Eye Institute - University of Miami /ID# 256072
    • Pensacola, Florida, United States, 32503
      • Retina Specialty Institute /ID# 256153
    • St. Petersburg, Florida, United States, 33711-1141
      • Retina Vitreous Associates of Florida - St. Petersburg /ID# 256050
    • Tallahassee, Florida, United States, 32308
      • Southern Vitreoretinal Associates /ID# 256158
  • Georgia
    • Augusta, Georgia, United States, 30909
      • Southeast Retina Center /ID# 256022
    • Marietta, Georgia, United States, 30060
      • Georgia Retina - Marietta /ID# 256142
    • Marietta, Georgia, United States, 30060
      • Marietta Eye Clinic /ID# 268163
    • Sandy Springs, Georgia, United States, 30328-4411
      • Thomas Eye Group PC /ID# 268159
  • Hawaii
    • ‘Aiea, Hawaii, United States, 96701
      • Retina Consultants of Hawaii /ID# 256049
  • Illinois
    • Chicago, Illinois, United States, 60607
      • University of Illinois at Chicago /ID# 256300
    • Lemont, Illinois, United States, 60439
      • University Retina and Macula Associates /ID# 256078
    • Oak Forest, Illinois, United States, 60452
      • University Retina and Macula Associates /ID# 256077
    • Springfield, Illinois, United States, 62702-3749
      • Springfield Clinic /ID# 266225
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Retina Partners Midwest, PC /ID# 256045
    • New Albany, Indiana, United States, 47150-3620
      • John-Kenyon American Eye Institute -New Albany /ID# 256065
  • Kansas
    • Lenexa, Kansas, United States, 66215
      • Retina Associates - Lenexa /ID# 256080
  • Kentucky
    • Edgewood, Kentucky, United States, 41017-3415
      • Cincinnati Eye Institute- Edgewood /ID# 256132
  • Louisiana
    • Metairie, Louisiana, United States, 70006-2940
      • Retina Associates of New Orleans /ID# 272440
    • New Orleans, Louisiana, United States, 70121
      • Ochsner Medical Center - Jefferson Highway /ID# 270524
  • Maryland
    • Baltimore, Maryland, United States, 21209
      • The Retina Care Center /ID# 256144
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital /ID# 256015
    • Chevy Chase, Maryland, United States, 20815
      • The Retina Group Of Washington - Chevy Chase /ID# 276039
    • Hagerstown, Maryland, United States, 21740
      • Cumberland Valley Retina Consultants - Hagerstown /ID# 256151
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Ophthalmic Consultants of Boston /ID# 256014
  • Michigan
    • Grand Blanc, Michigan, United States, 48439
      • Retina Associates of Michigan /ID# 266198
    • Royal Oak, Michigan, United States, 48073
      • Associated Retinal Consultants /ID# 256156
  • Minnesota
    • Edina, Minnesota, United States, 55435
      • VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256160
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic - Minnesota /ID# 256051
  • Missouri
    • St Louis, Missouri, United States, 46214
      • The Retina Institute /ID# 266587
  • Nevada
    • Reno, Nevada, United States, 89502
      • Sierra Eye Associates /ID# 256020
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • Eye Associates of New Mexico /ID# 256075
  • New York
    • Great Neck, New York, United States, 11021
      • Long Island Vitreoretinal Consultants /ID# 256074
    • Liverpool, New York, United States, 13088
      • Retina-Vitreous Surgeons of Central New York - Liverpool /ID# 266274
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Duke Eye Center /ID# 256076
    • Winston-Salem, North Carolina, United States, 27157
      • Atrium Health Wake Forest Baptist Medical Center /ID# 270767
  • Ohio
    • Cleveland, Ohio, United States, 44130
      • Retina Associates of Cleveland-Middleburg Heights /ID# 256052
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Main Campus /ID# 256064
  • Oklahoma
    • Edmond, Oklahoma, United States, 73013
      • Retina Vitreous Center, Research /ID# 256067
  • Oregon
    • Eugene, Oregon, United States, 97401
      • Verum Research, LLC /ID# 266585
    • Portland, Oregon, United States, 97221
      • Retina Northwest, PC /ID# 256140
  • Pennsylvania
    • Erie, Pennsylvania, United States, 16507
      • Erie Retina Research /ID# 256154
    • Monroeville, Pennsylvania, United States, 15146
      • Retina Vitreous Consultants - Monroeville /ID# 271654
    • Philadelphia, Pennsylvania, United States, 19107
      • Mid Atlantic Retina /ID# 256013
  • South Carolina
    • Ladson, South Carolina, United States, 29456
      • Charleston Neuroscience Institute /ID# 256235
  • South Dakota
    • Rapid City, South Dakota, United States, 57701
      • Black Hills Regional Eye Institute /ID# 256161
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Charles Retina Institute /ID# 256016
  • Texas
    • Abilene, Texas, United States, 79606-1224
      • Retina Research Institute of Texas /ID# 256141
    • Austin, Texas, United States, 78705
      • Austin Research Center for Retina /ID# 256148
    • Austin, Texas, United States, 78705
      • Austin Retina Associates - Austin /ID# 256053
    • Austin, Texas, United States, 78750-2298
      • Austin Clinical Research, LLC /ID# 256043
    • Beaumont, Texas, United States, 77707
      • Retina Consultants of Texas - Beaumont /ID# 266279
    • Bellaire, Texas, United States, 77401
      • Retina and Vitreous of Texas /ID# 263961
    • Dallas, Texas, United States, 75231
      • Texas Retina Associates - Dallas /ID# 256133
    • Fort Worth, Texas, United States, 76104
      • Baylor Scott & White Surgicare /ID# 256122
    • San Antonio, Texas, United States, 78240
      • Retina Consultants Of Texas /ID# 268611
    • San Antonio, Texas, United States, 78240
      • Stone Oak Surgery Center /ID# 266199
    • Southlake, Texas, United States, 76092
      • Retina Center Of Texas (Rct) - Southlake /ID# 256056
    • The Woodlands, Texas, United States, 77384
      • Retina Consultants - The Woodlands /ID# 256018
  • Utah
    • Salt Lake City, Utah, United States, 84107
      • Retina Associates of Utah /ID# 256044
    • Salt Lake City, Utah, United States, 84132
      • John A. Moran Eye Center /ID# 256068
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Wagner Macula & Retina Center - Norfolk /ID# 256134
  • Washington
    • Bellevue, Washington, United States, 98004
      • Pacific Northwest Retina /ID# 256155
    • Silverdale, Washington, United States, 98383
      • Retina Center NW, PLLC /ID# 256157
  • Wisconsin
    • Madison, Wisconsin, United States, 53705-3644
      • University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences /ID# 256159

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 46 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 50 years and ≤ 89 years
2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
3. Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
5. Willing and able to provide written, signed informed consent for this study
6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry

Inclusion Criteria (Bilateral Treatment Substudy)*:

1. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
2. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
3. Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes
4. Willing and able to provide written, signed informed consent for this study
5. Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study

Exclusion Criteria:

1. CNV or macular edema in the study eye secondary to any causes other than AMD
2. Subfoveal fibrosis or atrophy in the study eye, as determined by CRC
3. Any condition in the investigator's opinion that could limit VA improvement in the study eye
4. Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye
5. Advanced glaucoma or history of secondary glaucoma in the study eye
6. History of intraocular surgery in the study eye within 12 weeks prior to randomization
7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1
8. Prior treatment with gene therapy
9. Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months

Exclusion Criteria (Bilateral Treatment Substudy)*:

1. CNV or macular edema in either eye secondary to any causes other than AMD
2. Subfoveal fibrosis or atrophy in either eye
3. Any condition in the investigator's opinion that could limit VA improvement in either eye
4. Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye
5. Advanced glaucoma or history of secondary glaucoma in either eye
6. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
7. History of intraocular surgery in either eye within 12 weeks prior to randomization (Week -2)
8. History of intravitreal therapy in either eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening
9. Prior treatment with gene therapy (*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Control Arm; Ranibizumab administered via intravitreal injection approximately every 28 days	Biological: Ranibizumab (LUCENTIS®); 0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days; Other Names: Ranibizumab (anti-VEGF agent)
Experimental: ABBV-RGX-314 Dose 1; ABBV-RGX-314 Dose 1 administered via subretinal delivery one time.	Genetic: ABBV-RGX-314; AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1); Other Names: RGX-314; surabgene lomparvovec; Genetic: ABBV-RGX-314; AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2); Other Names: RGX-314; surabgene lomparvovec
Experimental: ABBV-RGX-314 Dose 2; ABBV-RGX-314 Dose 2 administered via subretinal delivery one time.	Genetic: ABBV-RGX-314; AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1); Other Names: RGX-314; surabgene lomparvovec; Genetic: ABBV-RGX-314; AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2); Other Names: RGX-314; surabgene lomparvovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change from baseline in Best Corrected Visual Acuity (BCVA)	BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)	At Week 54
Bilateral Treatment Substudy: Incidence of ocular AEs and any SAEs	Incidence of ocular AEs and any SAEs	Week 50

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidences of ocular and overall AEs over 54 weeks	AEs over 54 weeks	Through Week 54
Incidences of ocular and overall AEs over 98 weeks	AEs over 98 weeks	Through Week 98
Mean change from baseline in BCVA to Week 98 (ABBV-RGX-314 randomized participants) based on the ETDRS score	BCVA measured by ETDRS	Week 98
Proportion of participants with worsened BCVA	Proportion with worsened BCVA	Week 54; Week 98
Proportion of participants with improved BCVA	Proportion with improved BCVA	Week 54; Week 98
Mean change from Week 54 to Week 98 in BCVA (control arm participants who cross over to ABBV-RGX-314)	Mean change in BCVA based on ETDRS score	Week 54 to Week 98
Mean change from baseline in CRT as measured by SD-OCT	Mean change in CRT as measured by SD-OCT	Week 54; (ABBV-RGX-314 randomized participants) Week 98
Mean change from Week 54 to Week 98 in CRT as measured by SD-OCT (control arm participants who cross over to ABBV-RGX-314)	Mean change in CRT as measured by SD-OCT	from Week 54 to Week 98
Mean change from baseline in CPT as measured by SD-OCT	Mean change in CPT as measured by SD-OCT	Week 54; (ABBV-RGX-314 randomized participants) Week 98
Mean change from Week 54 to Week 98 in CPT as measured by SD-OCT (control arm participants who cross over to ABBV-RGX-314)	Mean change in CPT as measured by SD-OCT	from Week 54 to Week 98
Percent reduction in anti-VEGF injection annualized rate after Week 58 through Week 98 relative to the year prior to the study (control arm participants who cross over to ABBV-RGX-314)	Percent reduction in anti-VEGF injection annualized rate	After Week 58 through Week 98
Supplemental anti-VEGF injection annualized rate after Week 58 through Week 98 (control arm participants who cross over to ABBV-RGX-314)	Supplemental anti-VEGF injection annualized rate	After Week 58 to Week 98
Mean change from baseline in NEI-VFQ-25 (composite score) at Week 54 and (for ABBV-RGX-314 randomized participants and control arm participants who cross over to ABBV-RGX-314) Week 98	Mean change in NEI VGQ-25 (composite score) at week 54 (control arm participants who cross over to ABBV-RGX-314)	Week 54; Week 98
Mean change from baseline in MacTSQ (composite score) at Week 54 and (for ABBV-RGX-314 randomized participants and control arm participants who cross over to ABBV-RGX-314) Week 98	Mean change from baseline in MacTSQ (composite score) at week 54 and (control arm participants who cross over to ABBV-RGX-314) at Week 98	Week 54; Week 98
Aqueous ABBV-RGX-314 TP concentrations (ABBV-RGX-314 randomized participants)	Aqueous ABBV-RGX-314 TP concentration	Wk -2, Wk 14, Wk 26, Wk 38, Wk 54, and Wk 98
Aqueous ABBV-RGX-314 TP concentrations (control arm participants who cross over to ABBV-RGX-314)	Aqueous ABBV-RGX-314 TP concentration	Wk 54, Wk 66, Wk 78, Wk 90, and Wk 98
Immunogenicity measurements (ABBV-RGX-314 randomized participants)	Immunogenicity measurements (serum antibodies to AAV8 and serum antibodies to ABBV-RGX-314 TP)	Wk -2, Wk 14, Wk 26, Wk 38, Wk 54, and Wk 98
Immunogenicity measurements (control arm participants who cross over to ABBV-RGX-314)	Immunogenicity measurements (serum antibodies to AAV8 and serum antibodies to ABBV-RGX-314 TP)	Wk 54, Wk 66, Wk 78, Wk 90, and Wk 98
Bilateral Treatment Substudy: Supplemental anti-VEGF injection annualized rate	Supplemental anti-VEGF injection annualized rate	Through Week 50
Proportion of participants (1) gaining > 0 letters; (2) losing > 0 letters; maintaining vision (not losing ≥ 15 letters) compared with baseline as per BCVA	Proportion gaining or losing > 0 letters based on ETDRS score; proportion maintaining vision	Week 54; Week 98
Percent reduction in anti-VEGF injection annualized rate compared with the prior year (ABBV-RGX-314 randomized participants)	Supplemental anti-VEGF injection annualized rate	Through Week 54 and Week 98
Bilateral Treatment Substudy: Incidence of nonocular AEs and any AEs of special interest	Nonocular AEs and AEs of Special interest	Week 50
Bilateral Treatment Substudy: Mean change from Baseline in BCVA at assessed timepoints	BCVA measured by ETDRS	Through Week 50
Bilateral Treatment Substudy: Mean change from Baseline in CRT at assessed timepoints	Mean change in CRT as measured by SD-OCT	Through Week 50
Bilateral Treatement Substudy: Mean number of supplemental anti-VEGF injections	Mean supplemental anti-VEGF injections	Through Week 50
Bilateral Treatment Substudy: Aqueous humor and serum ABBV-RGX-314 TP concentrations	Aqueous humor and serum ABBV-RGX-314 TP Concentrations	Wk 26, Wk 34, Wk 50
Bilateral Treatment Substudy: Immunogenicity measurements (serum anti-ABBV-RGX-314 TP antibodies, serum anti-AAV8 antibodies) and enzyme-linked immunospot at assessed time points	Immunogenicity measurements	Wk 18, Wk 34, Wk 50
Mean change from baseline in BCVA for participants who received 0 or more supplemental anti-VEGF injections (ABBV-RGX-314 randomized participants)	Mean change in BCVA based on ETDRS score for participants who received 0 or more supplemental anti-VEGF injection	Week 54; Week 98
Mean number of supplemental anti-VEGF injections from Baseline through Week 54 (ABBV-RGX-314 randomized participants) and from Week 54 through Week 98 (ABBV-RGX-314 randomized participants and control arm participants who cross over to ABBV-RGX-314)	Mean number of supplemental anti-VEGF injections	Through Week 98
Proportion of participants with 0, 1, 2, and 3 supplemental injections through Week 54 and Week 98 (ABBV-RGX-314 randomized participants) and from Week 54 through Week 98 (control arm participants who cross over to ABBV-RGX-314)	Proportion of participants with 0, 1, 2, and 3 supplemental injections	Through Week 98
Proportion of participants with ≤ 1, ≤ 2, and ≤ 3 supplemental injections through Week 54 and Week 98 (ABBV-RGX-314 randomized participants) and from Week 54 through Week 98 (control arm participants who cross over to ABBV-RGX-314)	Proportion of participants with ≤ 1, ≤ 2, and ≤ 3 supplemental injections	Through Week 98
Proportion of participants that received 1 or 2 injections through Week 54 and Week 98 (ABBV-RGX-314 randomized participants) and from Week 54 through Week 98 (control arm participants who cross over to ABBV-RGX-314)	In the subset of participants who were given supplemental anti-VEGF injections, proportion of participants that received 1 or 2 injections	Through Week 98
Proportion of participants with a reduction of ≥ 50% in anti-VEGF injection annualized rate through Week 54 and Week 98 compared with the prior year (ABBV-RGX-314 randomized participants)	Proportion of participants with a reduction of ≥ 50% in anti-VEGF injection annualized rate	Through Week 98
Proportion of participants with a reduction of ≥ 75% in anti-VEGF injection annualized rate through Week 54 and Week 98 compared with the prior year (ABBV-RGX-314 randomized participants)	Proportion of participants with a reduction of ≥ 75% in anti-VEGF injection annualized rate	Through Week 98
Supplemental anti-VEGF injection annualized rate through Week 54 and Week 98 (ABBV-RGX-314 randomized participants)	Supplemental anti-VEGF injection annualized rate	Through Week 54 and Week 98
Time to first supplemental anti-VEGF injection after the Week 2 injection (ABBV-RGX-314 randomized participants)	Time to first supplemental anti-VEGF injection	Week 98
Time to first supplemental anti-VEGF injection after the Week 58 injection (control arm participants who cross over to ABBV-RGX-314)	Time to first supplemental anti-VEGF injection	After Week 58 to Week 98
Bilateral Treatment Substudy: Proportion of participants with 0, ≤ 1, ≤ 2, and ≤ 3 supplemental anti-VEGF injections	Proportion of participants with 0, ≤ 1, ≤ 2, and ≤ 3 supplemental anti-VEGF injections	Through Week 50

Collaborators and Investigators

• Sponsor: AbbVie
• Collaborators: REGENXBIO Inc.
• Investigators: Study Director: ABBVIE INC., AbbVie

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): December 29, 2020
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): November 1, 2027

Study Registration Dates

• First Submitted: January 8, 2021
• First Submitted That Met QC Criteria: January 8, 2021
• First Posted (Actual): January 12, 2021

Study Record Updates

• Last Update Posted (Actual): April 28, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Eye Diseases; Ranibizumab; Antineoplastic Agents; Physiological Effects of Drugs; Gene therapy; Age-Related Macular Degeneration; Retinal Diseases; Macular Degeneration; Choroidal Neovascularization; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Anti-VEGF therapy; Retinal Degeneration; Wet Macular Degeneration; Neovascular Age-Related Macular Degeneration; Growth Substances; Growth Inhibitors; Anti-vascular endothelial grown factory therapy
• Additional Relevant MeSH Terms: Pathologic Processes; Eye Diseases, Hereditary; Uveal Diseases; Choroid Diseases; Metaplasia; Neovascularization, Pathologic; Pathological Conditions, Signs and Symptoms; Retinal Diseases; Eye Diseases; Retinal Degeneration; Macular Degeneration; Choroidal Neovascularization; Wet Macular Degeneration; Amino Acids, Peptides, and Proteins; Proteins; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Ranibizumab
• Other Study ID Numbers: RGX-314-2104; M23-416 (Other Identifier: AbbVie Inc)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No