Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
NCT ID: NCT04704921
Last Updated: 2025-08-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
630 participants
INTERVENTIONAL
2020-12-29
2027-11-30
Brief Summary
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Detailed Description
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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
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ABBV-RGX-314 Dose 1
ABBV-RGX-314 Dose 1 administered via subretinal delivery one time.
ABBV-RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
ABBV-RGX-314 Dose 2
ABBV-RGX-314 Dose 2 administered via subretinal delivery one time.
ABBV-RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
Control Arm
Ranibizumab administered via intravitreal injection approximately every 28 days
Ranibizumab (LUCENTIS®)
0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days
Interventions
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ABBV-RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
ABBV-RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
Ranibizumab (LUCENTIS®)
0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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
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
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
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.
50 Years
89 Years
ALL
No
Sponsors
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REGENXBIO Inc.
INDUSTRY
AbbVie
INDUSTRY
Responsible Party
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Principal Investigators
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ABBVIE INC.
Role: STUDY_DIRECTOR
AbbVie
Locations
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Retinal Research Institute /ID# 256019
Phoenix, Arizona, United States
Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055
Sun City, Arizona, United States
University of Arkansas for Medical Sciences /ID# 271290
Little Rock, Arkansas, United States
Retina Vitreous Assoc Med Grp /ID# 256299
Beverly Hills, California, United States
Retinal Diagnostic Center /ID# 256137
Campbell, California, United States
The Retina Partners - Encino /ID# 256054
Encino, California, United States
Retina Consultants of Orange County /ID# 256152
Fullerton, California, United States
Salehi Retina Institute /ID# 263485
Huntington Beach, California, United States
UC Irvine/Gavin Herbert Eye Institute /ID# 256145
Irvine, California, United States
Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298
Mountain View, California, United States
UCLA Doheny Eye Center /ID# 256120
Pasadena, California, United States
California Eye Specialists Medical Group Inc. /ID# 256079
Pasadena, California, United States
Retina Consultants of San Diego /ID# 256021
Poway, California, United States
Retinal Consultants Medical Group /ID# 256047
Sacramento, California, United States
West Coast Retina /ID# 256448
San Francisco, California, United States
University of California, San Francisco /ID# 256130
San Francisco, California, United States
Orange County Retina Medical Group /ID# 256073
Santa Ana, California, United States
California Retina Consultants - Santa Barbara /ID# 256017
Santa Barbara, California, United States
Retina Consultants of Southern Colorado /ID# 256069
Colorado Springs, Colorado, United States
Southwest Retina Research Center /ID# 256136
Durango, Colorado, United States
Colorado Retina Associates /ID# 256121
Lakewood, Colorado, United States
Retina Group of New England - Waterford /ID# 256071
Waterford, Connecticut, United States
Advanced Research, LLC /ID# 275451
Deerfield Beach, Florida, United States
Vitreoretinal Associates, P.A. /ID# 256150
Gainesville, Florida, United States
Florida Retina Consultants /ID# 265661
Lakeland, Florida, United States
Bascom Palmer Eye Institute - University of Miami /ID# 256072
Miami, Florida, United States
Retina Specialty Institute /ID# 256153
Pensacola, Florida, United States
Retina Vitreous Associates of Florida - St. Petersburg /ID# 256050
St. Petersburg, Florida, United States
Southern Vitreoretinal Associates /ID# 256158
Tallahassee, Florida, United States
Southeast Retina Center /ID# 256022
Augusta, Georgia, United States
Georgia Retina - Marietta /ID# 256142
Marietta, Georgia, United States
Marietta Eye Clinic /ID# 268163
Marietta, Georgia, United States
Thomas Eye Group PC /ID# 268159
Sandy Springs, Georgia, United States
Retina Consultants of Hawaii /ID# 256049
‘Aiea, Hawaii, United States
University of Illinois at Chicago /ID# 256300
Chicago, Illinois, United States
University Retina and Macula Associates /ID# 256078
Lemont, Illinois, United States
University Retina and Macula Associates /ID# 256077
Oak Forest, Illinois, United States
Springfield Clinic /ID# 266225
Springfield, Illinois, United States
Retina Partners Midwest, PC /ID# 256045
Indianapolis, Indiana, United States
John-Kenyon American Eye Institute -New Albany /ID# 256065
New Albany, Indiana, United States
Retina Associates - Lenexa /ID# 256080
Lenexa, Kansas, United States
Cincinnati Eye Institute- Edgewood /ID# 256132
Edgewood, Kentucky, United States
Retina Associates of New Orleans /ID# 272440
Metairie, Louisiana, United States
Ochsner Medical Center - Jefferson Highway /ID# 270524
New Orleans, Louisiana, United States
The Retina Care Center /ID# 256144
Baltimore, Maryland, United States
Johns Hopkins Hospital /ID# 256015
Baltimore, Maryland, United States
The Retina Group Of Washington - Chevy Chase /ID# 276039
Chevy Chase, Maryland, United States
Cumberland Valley Retina Consultants - Hagerstown /ID# 256151
Hagerstown, Maryland, United States
Ophthalmic Consultants of Boston /ID# 256014
Boston, Massachusetts, United States
Retina Associates of Michigan /ID# 266198
Grand Blanc, Michigan, United States
Associated Retinal Consultants /ID# 256156
Royal Oak, Michigan, United States
VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256160
Edina, Minnesota, United States
Mayo Clinic - Minnesota /ID# 256051
Rochester, Minnesota, United States
The Retina Institute /ID# 266587
St Louis, Missouri, United States
Sierra Eye Associates /ID# 256020
Reno, Nevada, United States
Eye Associates of New Mexico /ID# 256075
Albuquerque, New Mexico, United States
Long Island Vitreoretinal Consultants /ID# 256074
Great Neck, New York, United States
Retina-Vitreous Surgeons of Central New York - Liverpool /ID# 266274
Liverpool, New York, United States
Duke Eye Center /ID# 256076
Durham, North Carolina, United States
Atrium Health Wake Forest Baptist Medical Center /ID# 270767
Winston-Salem, North Carolina, United States
Retina Associates of Cleveland-Middleburg Heights /ID# 256052
Cleveland, Ohio, United States
Cleveland Clinic Main Campus /ID# 256064
Cleveland, Ohio, United States
Retina Vitreous Center, Research /ID# 256067
Edmond, Oklahoma, United States
Verum Research, LLC /ID# 266585
Eugene, Oregon, United States
Retina Northwest, PC /ID# 256140
Portland, Oregon, United States
Erie Retina Research /ID# 256154
Erie, Pennsylvania, United States
Retina Vitreous Consultants - Monroeville /ID# 271654
Monroeville, Pennsylvania, United States
Mid Atlantic Retina /ID# 256013
Philadelphia, Pennsylvania, United States
Charleston Neuroscience Institute /ID# 256235
Ladson, South Carolina, United States
Black Hills Regional Eye Institute /ID# 256161
Rapid City, South Dakota, United States
Charles Retina Institute /ID# 256016
Germantown, Tennessee, United States
Retina Research Institute of Texas /ID# 256141
Abilene, Texas, United States
Austin Research Center for Retina /ID# 256148
Austin, Texas, United States
Austin Retina Associates - Austin /ID# 256053
Austin, Texas, United States
Austin Clinical Research, LLC /ID# 256043
Austin, Texas, United States
Retina Consultants of Texas - Beaumont /ID# 266279
Beaumont, Texas, United States
Retina and Vitreous of Texas /ID# 263961
Bellaire, Texas, United States
Texas Retina Associates - Dallas /ID# 256133
Dallas, Texas, United States
Baylor Scott & White Surgicare /ID# 256122
Fort Worth, Texas, United States
Retina Consultants Of Texas /ID# 268611
San Antonio, Texas, United States
Stone Oak Surgery Center /ID# 266199
San Antonio, Texas, United States
Retina Center Of Texas (Rct) - Southlake /ID# 256056
Southlake, Texas, United States
Retina Consultants - The Woodlands /ID# 256018
The Woodlands, Texas, United States
Retina Associates of Utah /ID# 256044
Salt Lake City, Utah, United States
John A. Moran Eye Center /ID# 256068
Salt Lake City, Utah, United States
Wagner Macula & Retina Center - Norfolk /ID# 256134
Norfolk, Virginia, United States
Pacific Northwest Retina /ID# 256155
Bellevue, Washington, United States
Retina Center NW, PLLC /ID# 256157
Silverdale, Washington, United States
University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences /ID# 256159
Madison, Wisconsin, United States
Countries
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Central Contacts
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Facility Contacts
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Site Coordinator
Role: primary
Site Coordinator
Role: primary
Site Coordinator
Role: primary
Site Coordinator
Role: primary
Related Links
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Other Identifiers
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M23-416
Identifier Type: OTHER
Identifier Source: secondary_id
RGX-314-2104
Identifier Type: -
Identifier Source: org_study_id
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