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Purpose

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.

Conditions

Eligibility

Eligible Ages
Between 50 Years and 89 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 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 Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
2 ABBV-RGX-314 treatment arms, 1 control arm (ranibizumab)
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
The administration of ABBV-RGX-314 requires an outpatient surgical procedure performed in an operating room, while the active control, ranibizumab, is administered via intravitreal injection in an office setting. This study will be partially masked which will include masking of key study assessors and study drug dose.

Arm Groups

ArmDescriptionAssigned Intervention
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:
    • surabgene lomparvovec
    • RGX-314
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 2)
    Other names:
    • surabgene lomparvovec
    • RGX-314
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)

Recruiting Locations

Retinal Research Institute /ID# 256019
Phoenix 5308655, Arizona 5551752 85053
Contact:
Site Coordinator
602-222-2221

Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055
Sun City 5316201, Arizona 5551752 85351

University of Arkansas for Medical Sciences /ID# 271290
Little Rock 4119403, Arkansas 4099753 72205

Retina Vitreous Assoc Med Grp /ID# 256299
Beverly Hills 5328041, California 5332921 90211

Retinal Diagnostic Center /ID# 256137
Campbell 5333689, California 5332921 95008

The Retina Partners - Encino /ID# 256054
Encino 5346649, California 5332921 91436

Retina Consultants of Orange County /ID# 256152
Fullerton 5351247, California 5332921 92835

Salehi Retina Institute /ID# 263485
Huntington Beach 5358705, California 5332921 92647

Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298
Mountain View 5375480, California 5332921 94040-4101

UCLA Doheny Eye Center /ID# 256120
Pasadena 5381396, California 5332921 91105

California Eye Specialists Medical Group Inc. /ID# 256079
Pasadena 5381396, California 5332921 91107

Retina Consultants of San Diego /ID# 256021
Poway 5384690, California 5332921 92064-2530

Retinal Consultants Medical Group /ID# 256047
Sacramento 5389489, California 5332921 95825

West Coast Retina /ID# 256448
San Francisco 5391959, California 5332921 94107

University of California, San Francisco /ID# 256130
San Francisco 5391959, California 5332921 94143

Orange County Retina Medical Group /ID# 256073
Santa Ana 5392900, California 5332921 92705

Retina Consultants of Southern Colorado /ID# 256069
Colorado Springs 5417598, Colorado 5417618 80909
Contact:
Site Coordinator
(719) 473-9595

Southwest Retina Research Center /ID# 256136
Durango 5420241, Colorado 5417618 81303

Colorado Retina Associates /ID# 256121
Lakewood 5427946, Colorado 5417618 80228

Advanced Research, LLC /ID# 275451
Deerfield Beach 4153071, Florida 4155751 33064-1342

Vitreoretinal Associates, P.A. /ID# 256150
Gainesville 4156404, Florida 4155751 32607

Florida Retina Consultants /ID# 265661
Lakeland 4161438, Florida 4155751 33805

Retina Specialty Institute /ID# 256153
Pensacola 4168228, Florida 4155751 32503
Contact:
Site Coordinator
(850) 476-6759

Retina Vitreous Associates of Florida - St. Petersburg /ID# 256050
St. Petersburg 4171563, Florida 4155751 33711-1141

Southern Vitreoretinal Associates /ID# 256158
Tallahassee 4174715, Florida 4155751 32308

Southeast Retina Center /ID# 256022
Augusta 4180531, Georgia 4197000 30909

Georgia Retina - Marietta /ID# 256142
Marietta 4207783, Georgia 4197000 30060

Marietta Eye Clinic /ID# 268163
Marietta 4207783, Georgia 4197000 30060

Thomas Eye Group PC /ID# 268159
Sandy Springs 4221333, Georgia 4197000 30328-4411

Retina Consultants of Hawaii /ID# 256049
‘Aiea 5856430, Hawaii 5855797 96701

University Retina and Macula Associates /ID# 256078
Lemont 4899581, Illinois 4896861 60439

University Retina and Macula Associates /ID# 256077
Oak Forest 4904286, Illinois 4896861 60452

Springfield Clinic /ID# 266225
Springfield 4250542, Illinois 4896861 62702-3749

Retina Partners Midwest, PC /ID# 256045
Indianapolis 4259418, Indiana 4921868 46290

John-Kenyon American Eye Institute -New Albany /ID# 256065
New Albany 4262045, Indiana 4921868 47150-3620

Retina Associates - Lenexa /ID# 256080
Lenexa 4274356, Kansas 4273857 66215

Cincinnati Eye Institute- Edgewood /ID# 256132
Edgewood 4290873, Kentucky 6254925 41017-3415

Retina Associates of New Orleans /ID# 272440
Metairie 4333177, Louisiana 4331987 70006-2940

Ochsner Medical Center - Jefferson Highway /ID# 270524
New Orleans 4335045, Louisiana 4331987 70121

The Retina Care Center /ID# 256144
Baltimore 4347778, Maryland 4361885 21209

Johns Hopkins Hospital /ID# 256015
Baltimore 4347778, Maryland 4361885 21287

The Retina Group Of Washington - Chevy Chase /ID# 276039
Chevy Chase 4351335, Maryland 4361885 20815

Cumberland Valley Retina Consultants - Hagerstown /ID# 256151
Hagerstown 4357141, Maryland 4361885 21740

Ophthalmic Consultants of Boston /ID# 256014
Boston 4930956, Massachusetts 6254926 02114

Retina Associates of Michigan /ID# 266198
Grand Blanc 4994320, Michigan 5001836 48439

Associated Retinal Consultants /ID# 256156
Royal Oak 5007804, Michigan 5001836 48073

VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256160
Edina 5025264, Minnesota 5037779 55435

Mayo Clinic - Minnesota /ID# 256051
Rochester 5043473, Minnesota 5037779 55905

The Retina Institute /ID# 266587
St Louis 4407066, Missouri 4398678 46214

Sierra Eye Associates /ID# 256020
Reno 5511077, Nevada 5509151 89502

Eye Associates of New Mexico /ID# 256075
Albuquerque 5454711, New Mexico 5481136 87109

Retina-Vitreous Surgeons of Central New York - Liverpool /ID# 266274
Liverpool 5124925, New York 5128638 13088

Duke Eye Center /ID# 256076
Durham 4464368, North Carolina 4482348 27705

Atrium Health Wake Forest Baptist Medical Center /ID# 270767
Winston-Salem 4499612, North Carolina 4482348 27157

Retina Associates of Cleveland-Middleburg Heights /ID# 256052
Cleveland 5150529, Ohio 5165418 44130

Cleveland Clinic Main Campus /ID# 256064
Cleveland 5150529, Ohio 5165418 44195

Retina Vitreous Center, Research /ID# 256067
Edmond 4535740, Oklahoma 4544379 73013

Verum Research, LLC /ID# 266585
Eugene 5725846, Oregon 5744337 97401

Retina Northwest, PC /ID# 256140
Portland 5746545, Oregon 5744337 97221

Erie Retina Research /ID# 256154
Erie 5188843, Pennsylvania 6254927 16507

Retina Vitreous Consultants - Monroeville /ID# 271654
Monroeville 5201734, Pennsylvania 6254927 15146
Contact:
Site Coordinator
(412) 683-5300

Mid Atlantic Retina /ID# 256013
Philadelphia 4560349, Pennsylvania 6254927 19107

Charleston Neuroscience Institute /ID# 256235
Ladson 4584277, South Carolina 4597040 29456

Black Hills Regional Eye Institute /ID# 256161
Rapid City 5768233, South Dakota 5769223 57701

Charles Retina Institute /ID# 256016
Germantown 4624601, Tennessee 4662168 38138

Retina Research Institute of Texas /ID# 256141
Abilene 4669635, Texas 4736286 79606-1224

Austin Research Center for Retina /ID# 256148
Austin 4671654, Texas 4736286 78705

Austin Retina Associates - Austin /ID# 256053
Austin 4671654, Texas 4736286 78705

Austin Clinical Research, LLC /ID# 256043
Austin 4671654, Texas 4736286 78750-2298

Retina Consultants of Texas - Beaumont /ID# 266279
Beaumont 4672989, Texas 4736286 77707

Retina and Vitreous of Texas /ID# 263961
Bellaire 4673353, Texas 4736286 77401

Texas Retina Associates - Dallas /ID# 256133
Dallas 4684888, Texas 4736286 75231

Baylor Scott & White Surgicare /ID# 256122
Fort Worth 4691930, Texas 4736286 76104

Retina Consultants Of Texas /ID# 268611
San Antonio 4726206, Texas 4736286 78240

Retina Center Of Texas (Rct) - Southlake /ID# 256056
Southlake 4733313, Texas 4736286 76092

Retina Consultants - The Woodlands /ID# 256018
The Woodlands 4736476, Texas 4736286 77384

Retina Associates of Utah /ID# 256044
Salt Lake City 5780993, Utah 5549030 84107

John A. Moran Eye Center /ID# 256068
Salt Lake City 5780993, Utah 5549030 84132

Wagner Macula & Retina Center - Norfolk /ID# 256134
Norfolk 4776222, Virginia 6254928 23502

Retina Center NW, PLLC /ID# 256157
Silverdale 5810490, Washington 5815135 98383

More Details

NCT ID
NCT04704921
Status
Recruiting
Sponsor
AbbVie

Study Contact

Patient Advocacy
833-711-0349
patientadvocacy@regenxbio.com

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.