Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04832724
Recruitment Status : Recruiting
First Posted : April 6, 2021
Last Update Posted : October 20, 2021
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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 : July 30, 2022
Estimated Study Completion Date : July 30, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location contacts
Contact: Patient Advocacy 1-866-860-0117 patientadvocacy@regenxbio.com

Locations
Layout table for location information
United States, Arizona
Phoenix location Recruiting
Phoenix, Arizona, United States, 85014
United States, California
San Diego location Recruiting
San Diego, California, United States, 92064
Santa Barbara location Not yet recruiting
Santa Barbara, California, United States, 93103
Santa Maria location Not yet recruiting
Santa Maria, California, United States, 93309
United States, Florida
Saint Petersburg Location Not yet recruiting
Saint Petersburg, Florida, United States, 33711
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 Recruiting
Reno, Nevada, United States, 89502
United States, New Mexico
Albuquerque location Recruiting
Albuquerque, New Mexico, United States, 87109
United States, Pennsylvania
Philadelphia location Recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Tennessee
Memphis location Recruiting
Germantown, Tennessee, United States, 38138
United States, Texas
Dallas Location Not yet recruiting
Dallas, Texas, United States, 75231
Woodlands location Recruiting
The Woodlands, Texas, United States, 77384
Sponsors and Collaborators
Regenxbio Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
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: October 20, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Macular Degeneration
Wet Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases