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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 : April 6, 2021
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Brief Summary:
RGX-314 is a gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. RGX-314 is being studied for its potential to have a single injection that could allow the eye to make its own supply of anti-VEGF continually. 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 characterized by loss of vision due to new, leaky blood vessel formation in the retina. 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 chooses to participate in the study, their participation in the study will last about 6 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 : September 2022
Estimated Study Completion Date : September 2022

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 24 ]
    Evaluate the safety and tolerability of RGX-314

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

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

  4. Supplemental anti-VEGF injection annualized rate [ Time Frame: Through Week 24 ]
    To assess the need for supplemental anti-VEGF therapy



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 subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye.
  4. Participants must have demonstrated a meaningful response to anti-VEGF therapy.
  5. Willing and able to provide written, signed informed consent for this 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 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. Received any 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
Phoenix location Not yet recruiting
Phoenix, Arizona, United States, 85014
United States, California
Bakersfield location Not yet recruiting
Bakersfield, California, United States, 93309
San Diego location Not yet recruiting
San Diego, California, United States, 92064
Santa Barbara location Not yet recruiting
Santa Barbara, California, United States, 93103
United States, Maryland
Baltimore location Recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston location Recruiting
Boston, Massachusetts, United States, 02114
United States, Nevada
Reno location Not yet recruiting
Reno, Nevada, United States, 89502
United States, New Mexico
Albuquerque location Not yet recruiting
Albuquerque, New Mexico, United States, 87109
United States, Pennsylvania
Philadelphia location Not yet recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Tennessee
Memphis location Not yet recruiting
Germantown, Tennessee, United States, 38138
United States, Texas
Woodlands location Recruiting
The Woodlands, Texas, United States, 77384
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: April 6, 2021
Last Verified: April 2021
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