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Trial record 1 of 9 for:    RGX-314
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RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

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ClinicalTrials.gov Identifier: NCT04832724
Recruitment Status : Recruiting
First Posted : April 6, 2021
Last Update Posted : February 1, 2023
Sponsor:
Information provided by (Responsible Party):
REGENXBIO Inc.

Brief Summary:
RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration

Condition or disease Intervention/treatment Phase
Neovascular Age-related Macular Degeneration Wet Macular Degeneration Wet Age-related Macular Degeneration Genetic: RGX-314 Phase 2

Detailed Description:
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 repeated intraocular injections 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. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. This phase 2, open label study will explore the pharmacodynamics of two doses in two formulations of RGX-314 gene therapy via subretinal delivery in patients with neovascular Age-related Macular Degeneration. Approximately 60 patients (15 per cohort) who meet the inclusion/exclusion criteria will be enrolled in 4 sequential dose cohorts. A dose cohort will be comprised of 1 of 2 doses of RGX-314 in 1 of 2 formulations in order to explore the pharmacodynamics of RGX-314 based on aqueous humor transgene product (TP) concentrations. If the participants meet the study criteria and choose to participate in the study, their participation in the study will last about 12 months.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label Study to Explore the Pharmacodynamics of Two Doses in Two Formulations of RGX-314 Gene Therapy Administered Via Subretinal Delivery in Participants With Neovascular Age-related Macular Degeneration
Actual Study Start Date : February 22, 2021
Estimated Primary Completion Date : March 31, 2023
Estimated Study Completion Date : March 31, 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Commercial Formulation Dose 1
Dose 1 of RGX-314
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Experimental: Clinical Formulation Dose 1
Dose 1 of RGX-314
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Experimental: Commercial Formulation Dose 2
Dose 2 of RGX-314
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Experimental: Clinical Formulation Dose 2
Dose 2 of RGX-314
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein




Primary Outcome Measures :
  1. RGX-314 target protein concentration in aqueous humor [ Time Frame: At Week 24 ]

Secondary Outcome Measures :
  1. Incidence and severity of ocular Adverse Events (AEs) and overall AEs [ Time Frame: Through Week 48 ]
    Evaluate the safety and tolerability of RGX-314

  2. Changes in Best Corrected Visual Acuity (BCVA) [ Time Frame: At Week 24 and 48 ]
    BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)

  3. Changes in Central Retinal Thickness (CRT) [ Time Frame: At Week 24 and 48 ]
    CRT is measured by spectral domain optical coherence tomography (SD-OCT)

  4. Mean Supplemental anti-VEGF injection annualized rate through week 24 and week 48 [ Time Frame: Through Week 24 and week 48 ]
    To assess the need for supplemental anti-VEGF therapy over 48 weeks



Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years to 89 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or females, aged ≥ 50 years and ≤ 89 years.
  2. An Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score between ≤ 78 and ≥ 40 in the study eye at Screening.
  3. Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye previously treated with anti-VEGF.
  4. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry.
  5. Willing and able to provide written, signed informed consent for this study.
  6. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.

Exclusion Criteria:

  1. CNV or macular edema in the study eye secondary to any causes other than AMD.
  2. Subfoveal fibrosis or atrophy in study eye.
  3. Any condition in the investigator's opinion that could limit visual acuity improvement in the study eye.
  4. Active or history of retinal detachment or retinal tear in the study eye.
  5. Advanced glaucoma in the study eye.
  6. Prior treatment with gene therapy.
  7. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04832724


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

Locations
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United States, Arizona
Retinal Consultants of Arizona Recruiting
Phoenix, Arizona, United States, 85014
Contact: Principal Investigator         
Barnet Dulaney Perkins Eye Center Recruiting
Sun City, Arizona, United States, 85351
Contact: Principal Investigator         
United States, California
Retina Consultants of San Diego Recruiting
San Diego, California, United States, 92064
Contact: Principal Investigator         
California Retina Consultants Active, not recruiting
Santa Barbara, California, United States, 93103
United States, Florida
Retina Vitreous Associates of Florida Recruiting
Saint Petersburg, Florida, United States, 33711
Contact: Principal Investigator         
United States, Maryland
Wilmer Eye Institute Recruiting
Baltimore, Maryland, United States, 21287
Contact: Principal Investigator         
United States, Massachusetts
Ophthalmic Consultants of Boston Recruiting
Boston, Massachusetts, United States, 02114
Contact: Principal Investigator         
United States, Nevada
Sierra Eye Associates Recruiting
Reno, Nevada, United States, 89502
Contact: Principal Investigator         
United States, New Mexico
Eye Associates of New Mexico Recruiting
Albuquerque, New Mexico, United States, 87109
Contact: Principal Investigator         
United States, Pennsylvania
Wills Eye Hospital Recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Tennessee
Charles Retina Institute Recruiting
Germantown, Tennessee, United States, 38138
Contact: Principal Investigator         
United States, Texas
Texas Retina Associates Recruiting
Dallas, Texas, United States, 75231
Contact: Principal Investigator         
Retina Consultants of Texas Recruiting
The Woodlands, Texas, United States, 77384
Contact: Principal Investigator         
Sponsors and Collaborators
REGENXBIO Inc.
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Responsible Party: REGENXBIO Inc.
ClinicalTrials.gov Identifier: NCT04832724    
Other Study ID Numbers: RGX-314-2103
First Posted: April 6, 2021    Key Record Dates
Last Update Posted: February 1, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by REGENXBIO Inc.:
AMD
wet AMD
wAMD
neovascular AMD
nAMD
gene therapy
Additional relevant MeSH terms:
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Macular Degeneration
Wet Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases