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NCT05407636PHASE3Recruiting

Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

AbbVie

Start Date

1/13/2022

Completion Date

11/1/2027

Summary

ABBV-RGX-314 (also known as RGX-314 and surabgene lomparvovec (sura-vec)) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). 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 (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

Detailed Description

This randomized, partially masked, controlled, Phase 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 RGX-314 gene therapy relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of ABBV-RGX-314 relative to aflibercept. Approximately 714 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), while 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.

Eligibility Criteria

Age Range: 50 years to 89 years

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 choroidal neovascularization (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 3. Any condition in the investigator's opinion that could limit VA improvement in the study eye 4. Advanced glaucoma or history of secondary glaucoma in the study eye 5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months 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 medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6 8. Prior treatment with gene therapy 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. Advanced glaucoma or history of secondary glaucoma in either eye 5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months 6. History of intraocular surgery in either eye within 12 weeks prior to randomization 7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6. 8. Prior treatment with gene therapy (\*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only. Note: Other inclusion/exclusion criteria apply

Interventions

GENETIC

ABBV-RGX-314 Dose 1

GENETIC

ABBV-RGX-314 Dose 2

BIOLOGICAL

Aflibercept (EYLEA®)

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Conditions

AMDnAMDWet Age-related Macular DegenerationwAMDWetAMDCNV

Locations

Barnet Dulaney Eye Center-Phoenix /ID# 256340

Phoenix, Arizona 85016

United States

Retinal Research Institute /ID# 256238

Phoenix, Arizona 85053

United States

Retina Macula Institute of Arizona /ID# 271026

Scottsdale, Arizona 85255-4134

United States

California Retina Consultants - Bakersfield /ID# 256240

Bakersfield, California 93309-1677

United States

Retina Vitreous Assoc Med Grp /ID# 256246

Beverly Hills, California 90211

United States

Retinal Diagnostic Center /ID# 256262

Campbell, California 95008

United States

The Retina Partners - Encino /ID# 259660

Encino, California 91436

United States

Retina Consultants of Orange County /ID# 256267

Fullerton, California 92835

United States

Jacobs Retina Center at UCSD/ID# 256320

La Jolla, California 92093

United States

California Retina Consultants - Oxnard - North Ventura Road /ID# 262883

Oxnard, California 93036

United States

Byers Eye Institute Stanford /ID# 262853

Palo Alto, California 94303

United States

Retina Consultants of San Diego /ID# 256258

Poway, California 92064-2530

United States

Kaiser Permanente - Riverside Medical Center /ID# 262413

Riverside, California 92505

United States

UC Davis Health Eye Center

Sacramento, California 95817

United States

Retinal Consultants Medical Group, Inc. - Greenback /ID# 256353

Sacramento, California 95841

United States

Orange County Retina Medical Group /ID# 256286

Santa Ana, California 92705

United States

Macula Retina Vitreous Center - Torrance /ID# 270247

Torrance, California 90503

United States

Bay Area Retina Associates - Walnut Creek - Lennon Lane /ID# 268513

Walnut Creek, California 94598

United States

Retina Consultants of Southern Colorado /ID# 256285

Colorado Springs, Colorado 80909

United States

Southwest Retina Research Center /ID# 256261

Durango, Colorado 81303

United States

Colorado Retina Associates /ID# 256378

Lakewood, Colorado 80228

United States

Advanced Vision Research Institute /ID# 256380

Longmont, Colorado 80503

United States

Retina Consultants, P.C. /ID# 273610

Manchester, Connecticut 06042-3556

United States

Rand Eye Institute /ID# 256337

Deerfield Beach, Florida 33064

United States

National Ophthalmic Research Institute /ID# 256344

Fort Myers, Florida 33912-7125

United States

Vitreoretinal Associates, P.A. /ID# 256266

Gainesville, Florida 32607

United States

Florida Retina Consultants /ID# 256321

Lakeland, Florida 33805

United States

Florida Retina Institute - Orlando /ID# 256373

Orlando, Florida 32806-1101

United States

Retina Specialty Institute /ID# 256268

Pensacola, Florida 32503

United States

Fort Lauderdale Eye Institute /ID# 266011

Plantation, Florida 33324

United States

Retina Vitreous Associates of Florida - St. Petersburg /ID# 256279

St. Petersburg, Florida 33711-1141

United States

Center for Retina and Macular Disease /ID# 256335

Winter Haven, Florida 33880

United States

Georgia Retina - Marietta /ID# 256264

Marietta, Georgia 30060

United States

Retina Consultants of Hawaii /ID# 256278

‘Aiea, Hawaii 96701

United States

Northwestern Memorial Hospital - Department of Ophthalmology /ID# 256379

Chicago, Illinois 60611-2987

United States

University Retina and Macula Associates /ID# 256288

Lemont, Illinois 60439

United States

University Retina and Macula Associates /ID# 256287

Oak Forest, Illinois 60452

United States

Illinois Retina Associates - Oak Park /ID# 256375

Oak Park, Illinois 60304

United States

Springfield Clinic - First /ID# 256371

Springfield, Illinois 62702

United States

Retina Partners Midwest, PC /ID# 256277

Indianapolis, Indiana 46290

United States

John-Kenyon American Eye Institute -New Albany /ID# 256283

New Albany, Indiana 47150

United States

Wolfe Eye Clinic - West Des Moines /ID# 256314

West Des Moines, Iowa 50266

United States

Retina Associates - Lenexa /ID# 258296

Lenexa, Kansas 66215

United States

Maine Eye Center - Marginal Way Campus /ID# 256391

Portland, Maine 04101

United States

Retina Specialists /ID# 256392

Baltimore, Maryland 21204

United States

Johns Hopkins Hospital /ID# 256236

Baltimore, Maryland 21287

United States

Mid Atlantic Retina Specialists - Hagerstown /ID# 256310

Hagerstown, Maryland 21740-5965

United States

MA Eye & Ear Infirmary /ID# 262430

Boston, Massachusetts 02114

United States

Ophthalmic Consultants of Boston /ID# 256233

Boston, Massachusetts 02114

United States

Vitreo Retinal Associates - Worcester /ID# 266008

Worcester, Massachusetts 01605

United States

Retina Associates of Michigan /ID# 256393

Grand Blanc, Michigan 48439

United States

Associated Retinal Consultants /ID# 256269

Royal Oak, Michigan 48073

United States

VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256272

Edina, Minnesota 55435

United States

Mayo Clinic - Rochester /ID# 258295

Rochester, Minnesota 55905-0001

United States

Mississippi Retina Associates /ID# 270934

Madison, Mississippi 39110-2028

United States

Mid America Surgery Center /ID# 258993

Chesterfield, Missouri 63017

United States

University of Missouri Hospital /ID# 262474

Columbia, Missouri 65212

United States

Washington University School of Medicine - St. Louis /ID# 256383

St Louis, Missouri 63108

United States

Sierra Eye Associates /ID# 256239

Reno, Nevada 89502

United States

Envision Ocular, LLC /ID# 256367

Bloomfield, New Jersey 07003

United States

Monmouth Retina Consultants /ID# 275895

Little Silver, New Jersey 07739

United States

Columbia University of New York - Edward S. Harkness Eye Institute /ID# 256342

New York, New York 10032

United States

Retina Associates of Western New York /ID# 256374

Rochester, New York 14620

United States

Asheville Eye Associates- Retina Division /ID# 256355

Asheville, North Carolina 28803

United States

Duke University Medical Center /ID# 256245

Durham, North Carolina 27710

United States

Graystone Eye /ID# 256424

Hickory, North Carolina 28602

United States

Cincinnati Eye Institute /ID# 256247

Cincinnati, Ohio 45242-5664

United States

Retina Associates of Cleveland-Middleburg Heights /ID# 256280

Cleveland, Ohio 44130

United States

Cleveland Clinic - Cleveland /ID# 256282

Cleveland, Ohio 44195

United States

Ohio State University /ID# 256275

Columbus, Ohio 43210

United States

Midwest Retina /ID# 276644

Dublin, Ohio 43016

United States

Retina Vitreous Center, Research /ID# 256284

Edmond, Oklahoma 73013

United States

University of Pennsylvania /ID# 258995

Philadelphia, Pennsylvania 19104-5502

United States

Mid Atlantic Retina /ID# 256232

Philadelphia, Pennsylvania 19107

United States

University of Pittsburgh Medical Center /ID# 258994

Pittsburgh, Pennsylvania 15213

United States

Charleston Neuroscience Institute - Beaufort /ID# 256385

Beaufort, South Carolina 29902-6216

United States

Charleston Neurosciences Institute /ID# 256351

Charleston, South Carolina 29414-5896

United States

Palmetto Retina Center-Florence /ID# 256244

Florence, South Carolina 29501

United States

Charleston Neurosciences Institute /ID# 256336

Ladson, South Carolina 29414-5896

United States

Black Hills Regional Eye Institute /ID# 262872

Rapid City, South Dakota 57701

United States

Southeastern Retina Associates - Chattanooga /ID# 258992

Chattanooga, Tennessee 37421

United States

Charles Retina Institute /ID# 256237

Germantown, Tennessee 38138

United States

Southeastern Retina Associates - Johnson City /ID# 256396

Johnson City, Tennessee 37604

United States

Tennessee Retina - Nashville /ID# 256388

Nashville, Tennessee 37203-1513

United States

Vanderbilt University Medical Center /ID# 256372

Nashville, Tennessee 37232-0011

United States

Retina Research Institute of Texas /ID# 256263

Abilene, Texas 79606-1224

United States

Austin Research Center for Retina /ID# 256265

Austin, Texas 78705

United States

Austin Clinical Research, LLC /ID# 256274

Austin, Texas 78750-2298

United States

Retina Consultants of Texas - Bellaire /ID# 256377

Bellaire, Texas 77401

United States

Star Retina - Burleson /ID# 256376

Burleson, Texas 76028

United States

Texas Retina Associates /ID# 256259

Dallas, Texas 75231

United States

Texas Retina Associates - Fort Worth /ID# 256326

Fort Worth, Texas 76104

United States

Austin Retina Associates - Round Rock /ID# 256387

Round Rock, Texas 78681

United States

Retina Consultants of Texas/ID# 259661

San Antonio, Texas 78240-1655

United States

Retina Associates of South Texas

San Antonio, Texas 78240

United States

Retina Consultants of Texas /ID# 256382

San Antonio, Texas 78251

United States

Retina Center Of Texas (Rct) - Southlake /ID# 256281

Southlake, Texas 76092

United States

Rocky Mountain Retina Consultants /ID# 256369

Murray, Utah 84107

United States

Retina Associates of Utah /ID# 256276

Salt Lake City, Utah 84107

United States

Piedmont Eye Center /ID# 256386

Lynchburg, Virginia 24502

United States

Wagner Kapoor Research Institute - Norfolk /ID# 256260

Norfolk, Virginia 23502

United States

Retina Institute of Virginia /ID# 256394

Richmond, Virginia 23235

United States

Vistar Eye Center /ID# 271024

Roanoke, Virginia 24019

United States

Pacific Northwest Retina /ID# 258999

Bellevue, Washington 98004

United States

Retina Center NW, PLLC /ID# 256395

Silverdale, Washington 98383

United States

Spokane Eye Clinic /ID# 256338

Spokane, Washington 99204

United States

Gundersen Health System - La Crosse Medical Center /ID# 276012

La Crosse, Wisconsin 54601

United States

Calgary Retina Consultants /ID# 258997

Calgary, Alberta T2H 0C8

Canada

Alberta Retina Consultants /ID# 265599

Edmonton, Alberta T5H 0X5

Canada

Retina Surgical Associates /ID# 270958

New Westminster, British Columbia V3L 5H1

Canada

University of British Columbia - Eye Care Centre, Vancouver General Hospital /ID# 266014

Vancouver, British Columbia V5Z 3N9

Canada

Vitreous Retina Macula Specialists of Toronto /ID# 258299

Etobicoke, Ontario M8X 2X3

Canada

Ottawa Hospital Research Institute /ID# 258998

Ottawa, Ontario K1H 8L6

Canada

Retina Centre of Ottawa /ID# 259659

Ottawa, Ontario K2B 7E9

Canada

Toronto Retina Institute /ID# 265602

Toronto, Ontario M3C 0G9

Canada

Sunnybrook Research Institute /ID# 270690

Toronto, Ontario M4N 3M5

Canada

CHU de Quebec-Universite Laval /ID# 258996

Québec, Quebec G1R 2J6

Canada

Centre Hospitalier Universitaire de Nice - Hopital Pasteur /ID# 262418

Nice, Alpes-Maritimes 06001

France

Hôpital de la Croix Rousse - Groupement Hospitalier Nord /ID# 262409

Lyon, Auvergne-Rhône-Alpes 69004

France

CHU Dijon /ID# 262410

Dijon, Cote-d Or 21000

France

Chu De Toulouse - Hopital Pierre Paul Ricquet /ID# 262411

Toulouse, Occitanie 31059

France

Hopitaux Universitaires Paris Centre-Hopital Cochin /ID# 262415

Paris, Paris 75679

France

Centre Hospitalier Universitaire de Nantes, Hotel Dieu -HME /ID# 262408

Nantes, Pays de la Loire Region 44000

France

Clinique Juge /ID# 262417

Marseille, Provence-Alpes-Côte d'Azur Region 13008

France

Centre Hosp Intercommunal de Creteil /ID# 262407

Créteil, Val-de-Marne 94000

France

Clinique Honoré Cave /ID# 262225

Montauban, 82017

France

AP-HP - Groupe Hospitalier 10e - Hopital Lariboisiere /ID# 262406

Paris, 75010

France

Universitaetsklinikum Freiburg /ID# 262495

Freiburg im Breisgau, Baden-Wurttemberg 79106

Germany

Universitaetsklinikum Ulm /ID# 262494

Ulm, Baden-Wurttemberg 89081

Germany

Universitaetsklinikum Bonn /ID# 262498

Bonn, North Rhine-Westphalia 53127

Germany

Universitaetsklinikum Muenster /ID# 262499

Münster, North Rhine-Westphalia 48149

Germany

Klinikum Ludwigshafen /ID# 262497

Ludwigshafen am Rhein, Rhineland-Palatinate 67063

Germany

Universitaetsklinikum Hamburg-Eppendorf /ID# 262493

Hamburg, 20246

Germany

Knappschaftsklinikum Saar /ID# 262496

Püttlingen, 66346

Germany

Debreceni Egyetem-Klinikai Kozpont /ID# 262646

Debrecen, Hajdú-Bihar 4032

Hungary

Budapest Retina Intezet /ID# 262647

Budapest, 1133

Hungary

Ganglion Orvosi Központ /ID# 264550

Pécs, 7621

Hungary

Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale S.Anna /ID# 262896

Cona, Ferrara 44124

Italy

IRCCS Ospedale San Raffaele /ID# 263035

Milan, Milano 20132

Italy

ASST Fatebenefratelli Sacco /ID# 263273

Milan, Milano 20157

Italy

Azienda Ospedaliera Universitaria Luigi Vanvitelli /ID# 264074

Naples, Napoli 80138

Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS-Università Cattolica /ID# 263037

Rome, Roma 00168

Italy

IRCCS Fondazione G.B. Bietti per lo studio e la ricerca in Oftalmologia- ONLUS /ID# 262897

Rome, Roma 00184

Italy

IRCCS Ospedale Sacro Cuore Don Calabria /ID# 263536

Negrar, Verona 37024

Italy

A.O.U. Consorziale Policlinico di Bari /ID# 262500

Bari, 70124

Italy

ASL 2 Abruzzo Lanciano-Vasto-Chieti /ID# 263923

Chieti, 67100

Italy

ASU FC - P.O. Universitario Santa Maria della Misericordia /ID# 263036

Udine, 33100

Italy

Nagoya University Hospital /ID# 267350

Nagoya, Aichi-ken 466-8560

Japan

Fukushima Medical University Hospital /ID# 258411

Fukushima, Fukushima 960-1295

Japan

Kobe City Eye Hospital /ID# 256948

Kobe, Hyōgo 650-0047

Japan

Hyogo Medical University Hospital /ID# 257893

Nishinomiya-shi, Hyōgo 663-8501

Japan

Kagoshima University Hospital /ID# 257053

Kagoshima, Kagoshima-ken 890-8520

Japan

Kyoto University Hospital /ID# 267351

Kyoto, Kyoto 606-8507

Japan

University of Miyazaki Hospital /ID# 262879

Miyazaki, Miyazaki 889-1692

Japan

Shiga University of Medical Science /ID# 266862

Ōtsu, Shiga 520-2192

Japan

Juntendo University Hospital /ID# 258715

Bunkyo-ku, Tokyo 113-8431

Japan

Emanuelli Research & Development Center LLC /ID# 256231

Arecibo, 00612

Puerto Rico

Complejo Hospitalario Universitario de Santiago (CHUS) /ID# 262632

Santiago de Compostela, A Coruna 15706

Spain

Hospital Universitari de Bellvitge /ID# 262641

L'Hospitalet de Llobregat, Barcelona 08907

Spain

Hospital Arruzafa /ID# 262634

Córdoba, Cordoba 14012

Spain

Complejo Hospitalario Universitario Insular-Materno Infantil de Gran Canaria /ID# 262633

Las Palmas de Gran Canaria, Las Palmas 35016

Spain

Hospital Universitario Puerta de Hierro - Majadahonda /ID# 262635

Majadahonda, Madrid 28222

Spain

Institut Clínic d'Oftalmologia (ICOF). Hospital Clínic d'Oftalmologia. /ID# 262644

Barcelona, 08028

Spain

Instituto de Microcirugía Ocular /ID# 262642

Barcelona, 08035

Spain

Hospital Santa Creu i Sant Pau /ID# 262637

Barcelona, 08041

Spain

Hospital Universitario 12 de Octubre /ID# 262636

Madrid, 28041

Spain

Clinica Baviera /ID# 270146

Madrid, 28046

Spain

Stoke Mandeville Hospital /ID# 277343

Aylesbury, Buckinghamshire HP21 8AL

United Kingdom

Western Eye Hospital /ID# 271621

London, England NW1 5QH

United Kingdom

Gloucestershire Royal Hospital /ID# 277362

Gloucester, Gloucestershire GL1 3NN

United Kingdom

Moorfields Eye Hospital /ID# 263038

London, Greater London EC1V 2PD

United Kingdom

The Retina Clinic London /ID# 262304

London, Greater London W1G 7LB

United Kingdom

University Hospital Southampton NHS Foundation Trust /ID# 262307

Southampton, Hampshire SO16 6YD

United Kingdom

Oxford University Hospitals NHS Foundation Trust /ID# 262306

Oxford, Oxfordshire OX3 9DU

United Kingdom

Leeds Teaching Hospital /ID# 277366

Leeds, West Yorkshire LS9 7TF

United Kingdom

Bradford Royal Infirmary /ID# 273164

Bradford, BD9 6RJ

United Kingdom

Bristol Eye Hospital /ID# 271619

Bristol, BS1 2LX

United Kingdom

Liverpool University Hospitals NHS Foundation Trust /ID# 262305

Liverpool, L7 8XP

United Kingdom

Manchester University NHS Foundation Trust /ID# 262404

Manchester, M9 2AA

United Kingdom

Royal Victoria Infirmary /ID# 277368

Newcastle upon Tyne, NE1 4LP

United Kingdom

Sunderland Eye Infirmary /ID# 271620

Sunderland, SR2 9HP

United Kingdom