Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD

Purpose

RGX-314 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-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. RGX-314 is being developed as a potential one-time treatment for wet AMD.

Conditions

  • AMD
  • nAMD
  • Wet Age-related Macular Degeneration
  • wAMD
  • Wet AMD
  • CNV

Eligibility

Eligible Ages
Between 50 Years and 89 Years
Eligible Genders
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

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. Active or history of retinal detachment in the study eye 5. Uncontrolled glaucoma in the study eye 6. History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1 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.

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
2 RGX-314 treatment arms, 1 control arm (ranibizumab)
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
The administration of 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
RGX-314 Dose 1 		RGX-314 Dose 1 administered via subretinal delivery one time.
  • Genetic: RGX-314
    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
Experimental
RGX-314 Dose 2 		RGX-314 Dose 2 administered via subretinal delivery one time.
  • Genetic: RGX-314
    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
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 Consultants of Arizona, Ltd.
Phoenix, Arizona 85014

Barnet Dulaney Perkins Eye Center - Phoenix
Sun City, Arizona 85351

Retina Vitreous Assoc Med Grp
Beverly Hills, California 90211

Retina-Vitreous Associates Medical Group
Beverly Hills, California 90211

Retinal Diagnostic Center
Campbell, California 95008

The Retina Partners - Encino
Encino, California 91436

Retina Consultants of Orange County - Fullerton Office
Fullerton, California 92835

University of California Irvine - School of Medicine - The Gavin Herbert Eye Institute
Irvine, California 92697

Doheny Eye Institute - Doheny Eye Center - Arcadia
Los Angeles, California 90033

Northern California Retina Vitreous Associates Medical Group, Inc. - Mountain View Office
Mountain View, California 94040

California Eye Specialists Medical Group Inc. - Pasadena
Pasadena, California 91107

Retina Consultants San Diego
Poway, California 92064

Retina Consultants
Sacramento, California 95819

West Coast Retina Medical Group - San Francisco Office
San Francisco, California 94109
Contact:
San Francisco 1 Location

UCSF Medical Center - Retina and Vitreous Clinic
San Francisco, California 94143

Orange County Retina Medical Group
Santa Ana, California 92705

California Retina Consultants
Santa Barbara, California 85014

Retina Consultants of Southern Colorado PC- Colorado Springs
Colorado Springs, Colorado 80909

Southwest Retina Consultants
Durango, Colorado 81301

Colorado Retina Associates (CRA) - Red Rocks Medical Center Location
Lakewood, Colorado 80401

Retina Group of New England - New London
Waterford, Connecticut 06385

Vitreo Retinal Associates PA - The Millennium Center
Gainesville, Florida 32607

Vitreoretinal Associates, P.A.
Gainesville, Florida 32607

Bascom Palmer Eye Institute - University of Miami Health System
Miami, Florida 33136

Retina Specialty Institute - Pensacola
Pensacola, Florida 32503

Retina Vitreous Associates of Florida (RVA) - Saint Petersburg
Saint Petersburg, Florida 33711

Georgia Retina PC - Marietta
Marietta, Georgia 30060

Retina Consultants of Hawaii (RCH) - Pali Momi Medical Center Location
'Aiea, Hawaii 96701

Illinois Eye and Ear Infirmary at UI Health UIC
Chicago, Illinois 60612

University Retina and Macula Associates, P.C. - Lemont Office
Lemont, Illinois 60439

University Retina and Macula Associates, PC - Oak Forest
Oak Forest, Illinois 60452

Midwest Eye Institute
Indianapolis, Indiana 46290

John-Kenyon American Eye Institute - New Albany
New Albany, Indiana 47150

Retina Associates, P.A. - Shawnee Mission
Shawnee Mission, Kansas 66204

The Retina Care Center, LLC
Baltimore, Maryland 21209

The Johns Hopkins Hospital - Wilmer Eye Institute - East Baltimore
Baltimore, Maryland 21287

Cumberland Valley Retina Consultants (CVRC)
Hagerstown, Maryland 21740

Ophthalmic Consultants of Boston, Inc.
Boston, Massachusetts 02114

Associated Retinal Consultants P.C.
Royal Oak, Michigan 48073

VitreoRetinal Surgery, PA - Edina Location
Minneapolis, Minnesota 55435
Contact:
Location

Mayo Clinic - Minnesota
Rochester, Minnesota 55905

Sierra Eye Associates
Reno, Nevada 89502

Eye Associates of New Mexico - Albuquerque - Retina Center Location
Albuquerque, New Mexico 87109

Long Island Vitreoretinal Consultants - Great Neck
Great Neck, New York 11021

Duke Eye Center - Erwin Road - Duke University Medical Center
Durham, North Carolina 27705

Cincinnati Eye Institute - Blue Ash
Cincinnati, Ohio 45242

Retina Associates of Cleveland, Inc. - Middleburg Heights Office
Cleveland, Ohio 44130

Cleveland Clinic
Cleveland, Ohio 44195

Retina Vitreous Center (RVC)
Edmond, Oklahoma 73013

Retina Northwest, PC
Portland, Oregon 97221

Erie Retinal Surgery, Inc
Erie, Pennsylvania 16507-1429

Mid Atlantic Retina - Philadelphia
Philadelphia, Pennsylvania 19107

Charleston Neuroscience Institute
Ladson, South Carolina 29456-4118

Black Hills Regional Eye Institute
Rapid City, South Dakota 57701

Charles Retina Institute
Germantown, Tennessee 38138

West Texas Retina Consultants (WTRC) - Abilene Location
Abilene, Texas 79606

Austin Research Center for Retina
Austin, Texas 78705

Austin Retina Associates
Austin, Texas 78705

Retina Consultants of Austin
Austin, Texas 78705

Austin Clinical Research, LLC
Austin, Texas 78750

Star Retina
Burleson, Texas 79606
Contact:
Burleson Location

Texas Retina Associates
Dallas, Texas 75231

Retina Center of Texas - Southlake
Southlake, Texas 76092

Retina Consultants of Texas - The Woodlands
The Woodlands, Texas 77030

Retina Associates of Utah, PC
Salt Lake City, Utah 84107

John A. Moran Eye Center - University of Utah Health Care
Salt Lake City, Utah 84132

Wagner Macula & Retina Center
Virginia Beach, Virginia 23454

Pacific Northwest Retina
Bellevue, Washington 98004-3779

Retina Center Northwest - Silverdale
Silverdale, Washington 98383-7849

University of Wisconsin Health - University Station Clinic
Madison, Wisconsin 53705

More Details

NCT ID
NCT04704921
Status
Recruiting
Sponsor
AbbVie

Study Contact

Patient Advocacy
1-866-860-0117
patientadvocacy@regenxbio.com

Detailed Description

This randomized, partially masked, controlled, Phase 2b/3 clinical study will evaluate the efficacy and safety of RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to ranibizumab. Approximately 300 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.