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RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)

NCT ID: NCT04514653

Last Updated: 2025-11-14

Study Results

Results pending

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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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

140 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-08-25

Study Completion Date

2027-02-28

Brief Summary

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This interventional study is being conducted with an investigational gene therapy treatment called ABBV-RGX-314 (also known as RGX-314) and is being developed as a potential one-time gene therapy treatment for neovascular (wet) age-related macular degeneration (wet AMD or nAMD). The typical treatment for nAMD is frequent injections of anti-VEGF therapy. Researchers are testing ABBV-RGX-314 to see if it has similar effects as the current approved standard of care, such as Lucentis® or Eylea® injections.

The duration of this study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314 after week 52, up to 80 weeks post-randomization.

The primary outcome measure for this investigational study is to evaluate the mean change in best-corrected visual acuity (BCVA) for ABBV-RGX-314 compared with ranibizumab monthly at the Week 40 visit.

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Detailed Description

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This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 gene therapy in subjects with nAMD.

ABBV-RGX-314 will be injected into the SCS part of the eye. Approximately 140 participants who meet the inclusion/exclusion criteria will be enrolled into one of 7 cohorts. Participants will be randomized in Cohorts 1 and 2 to receive ABBV-RGX-314 or the intravitreal ranibizumab control. Participants enrolled in Cohorts 3 through 5 will receive ABBV-RGX-314. Participants enrolled in Cohort 6 will receive ABBV-RGX-314 and will be randomized to one of two different post-procedural steroid regimens. Participants randomized in Cohort 7 will receive ABBV-RGX-314 with a protocol mandated steroid regimen or the intravitreal ranibizumab control. Cohort 1 will evaluate ABBV-RGX-314 Dose 1, Cohorts 2 and 3 will evaluate ABBV-RGX-314 Dose 2, Cohorts 4, 5, and 6 will evaluate ABBV-RGX-314 Dose 3, and Cohort 7 will evaluate ABBV-RGX-314 Dose 4.

The duration of the study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314, up to 80 weeks post-randomization.

Conditions

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Neovascular Age-Related Macular Degeneration (nAMD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors
The vision examiners and central reading center (CRC) graders will be masked, meaning they will be unaware of the participants' treatment assignment. All other individuals affiliated with the study (investigators, all study center personnel apart from the vision examiners, the participants, all staff of Sponsor and its research collaborator, all staff affiliated with the contract research organization, and all CRC staff apart from the CRC graders) will have knowledge of the treatment assignment.

Study Groups

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Ranibizumab control

Control treatment arm

Group Type ACTIVE_COMPARATOR

Ranibizumab

Intervention Type BIOLOGICAL

Ranibizumab (anti-VEGF agent)

ABBV-RGX-314 Treatment Arm (Dose 1)

ABBV-RGX-314 Dose 1

Group Type EXPERIMENTAL

ABBV-RGX-314 Dose 1

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

ABBV-RGX-314 Treatment Arm (Dose 2)

ABBV-RGX-314 Dose 2

Group Type EXPERIMENTAL

ABBV-RGX-314 Dose 2

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

ABBV-RGX-314 Treatment Arm (Dose 3)

ABBV-RGX-314 Dose 3

Group Type EXPERIMENTAL

ABBV-RGX-314 Dose 3

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

ABBV-RGX-314 Treatment Arm (Dose 3) and Local Steroid

ABBV-RGX-314 Dose 3 and Local Steroid

Group Type EXPERIMENTAL

ABBV-RGX-314 Dose 3

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

Local Steroid

Intervention Type DRUG

Local steroid

ABBV-RGX-314 Treatment Arm (Dose 3) and Topical Steroid

ABBV-RGX-314 Dose 3 and Topical Steroid

Group Type EXPERIMENTAL

ABBV-RGX-314 Dose 3

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

Topical Steroid

Intervention Type DRUG

Topical steroid

ABBV-RGX-314 Treatment Arm (Dose 4) and Topical Steroid

ABBV-RGX-314 Dose 4 and Topical Steroid

Group Type EXPERIMENTAL

Topical Steroid

Intervention Type DRUG

Topical steroid

ABBV-RGX-314 Dose 4

Intervention Type GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)

Interventions

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Ranibizumab

Ranibizumab (anti-VEGF agent)

Intervention Type BIOLOGICAL

ABBV-RGX-314 Dose 1

AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

Intervention Type GENETIC

ABBV-RGX-314 Dose 2

AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

Intervention Type GENETIC

ABBV-RGX-314 Dose 3

AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

Intervention Type GENETIC

Local Steroid

Local steroid

Intervention Type DRUG

Topical Steroid

Topical steroid

Intervention Type DRUG

ABBV-RGX-314 Dose 4

AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)

Intervention Type GENETIC

Other Intervention Names

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Combination Product Combination Product Combination Product Combination Product

Eligibility Criteria

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Inclusion Criteria

* Age \>/= 50 and \</= 89
* Diagnosis of CNV secondary to age-related macular degeneration in the study eye.
* Participants must have demonstrated a meaningful response to anti-VEGF therapy.
* Willing and able to provide written, signed informed consent for this study.

Exclusion Criteria

* CNV or macular edema in the study eye secondary to any causes other than AMD.
* Subfoveal fibrosis or atrophy in study eye.
* Participants who have had a prior vitrectomy.
* Active or history of retinal detachment in the study eye.
* History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry.
* Received any gene therapy.
* Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract.
* History of intraocular surgery in the study eye within 12 weeks of study entry.
* Receipt of any IP within 30 days of study entry or 5 half-lives of the IP.
* Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry.
* Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye.

COHORT 6 AND 7 ONLY:

* Active or history of glaucoma or ocular hypertension (steroid-induced ocular hypertension (Cohort 7 only)) in the study eye.
* Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1.
Minimum Eligible Age

50 Years

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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REGENXBIO Inc.

INDUSTRY

Sponsor Role collaborator

AbbVie

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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ABBVIE INC.

Role: STUDY_DIRECTOR

AbbVie

Locations

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Retinal Research Institute /ID# 255925

Phoenix, Arizona, United States

Site Status RECRUITING

California Retina Consultants - Bakersfield /ID# 255910

Bakersfield, California, United States

Site Status RECRUITING

Retina Vitreous Assoc Med Grp /ID# 255921

Beverly Hills, California, United States

Site Status RECRUITING

Retinal Diagnostic Center /ID# 272275

Campbell, California, United States

Site Status RECRUITING

Northern California Retina Vitreous Associates Medical Group, Inc /ID# 255920

Mountain View, California, United States

Site Status RECRUITING

Retina Consultants of San Diego /ID# 255911

Poway, California, United States

Site Status RECRUITING

California Retina Consultants - Santa Barbara /ID# 255923

Santa Barbara, California, United States

Site Status COMPLETED

Southeast Retina Center /ID# 255912

Augusta, Georgia, United States

Site Status RECRUITING

Springfield Clinic - First /ID# 272274

Springfield, Illinois, United States

Site Status RECRUITING

Johns Hopkins Hospital /ID# 255919

Baltimore, Maryland, United States

Site Status RECRUITING

Ophthalmic Consultants of Boston /ID# 255917

Boston, Massachusetts, United States

Site Status RECRUITING

Sierra Eye Associates /ID# 255908

Reno, Nevada, United States

Site Status RECRUITING

Eye Associates of New Mexico /ID# 255915

Albuquerque, New Mexico, United States

Site Status RECRUITING

Duke University Medical Center /ID# 267646

Durham, North Carolina, United States

Site Status RECRUITING

Mid Atlantic Retina /ID# 255906

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Charles Retina Institute /ID# 255922

Germantown, Tennessee, United States

Site Status COMPLETED

Tennessee Retina - Nashville /ID# 255918

Nashville, Tennessee, United States

Site Status COMPLETED

Retina Consultants - The Woodlands /ID# 255924

The Woodlands, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Patient Advocacy

Role: CONTACT

[email protected]
1-866-860-0117

Related Links

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https://www.abbvieclinicaltrials.com/study/?id=RGX-314-2102

Related Info

Other Identifiers

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M23-411

Identifier Type: OTHER

Identifier Source: secondary_id

RGX-314-2102 (M23-411)

Identifier Type: -

Identifier Source: org_study_id

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