RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)
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
1 other identifier
interventional
60
1 country
13
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Feb 2021
Typical duration for phase_2
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 22, 2021
CompletedFirst Submitted
Initial submission to the registry
March 30, 2021
CompletedFirst Posted
Study publicly available on registry
April 6, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 2, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
March 18, 2024
CompletedSeptember 5, 2024
September 1, 2024
2.6 years
March 30, 2021
September 3, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
RGX-314 target protein concentration in aqueous humor
At Week 24
Secondary Outcomes (4)
Incidence and severity of ocular Adverse Events (AEs) and overall AEs
Through Week 48
Changes in Best Corrected Visual Acuity (BCVA)
At Week 24 and 48
Changes in Central Retinal Thickness (CRT)
At Week 24 and 48
Mean Supplemental anti-VEGF injection annualized rate through week 24 and week 48
Through Week 24 and week 48
Study Arms (4)
Commercial Formulation Dose 1
EXPERIMENTALDose 1 of RGX-314
Clinical Formulation Dose 1
EXPERIMENTALDose 1 of RGX-314
Commercial Formulation Dose 2
EXPERIMENTALDose 2 of RGX-314
Clinical Formulation Dose 2
EXPERIMENTALDose 2 of RGX-314
Interventions
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Eligibility Criteria
You may qualify if:
- Males or females, aged ≥ 50 years and ≤ 89 years.
- An Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score between ≤ 78 and ≥ 40 in the study eye at Screening.
- Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye previously treated with anti-VEGF.
- Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry.
- Willing and able to provide written, signed informed consent for this study.
- Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
You may not qualify if:
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Subfoveal fibrosis or atrophy in study eye.
- Any condition in the investigator's opinion that could limit visual acuity improvement in the study eye.
- Active or history of retinal detachment or retinal tear in the study eye.
- Advanced glaucoma in the study eye.
- Prior treatment with gene therapy.
- Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AbbVielead
Study Sites (13)
Retinal Consultants of Arizona
Phoenix, Arizona, 85014, United States
Barnet Dulaney Perkins Eye Center
Sun City, Arizona, 85351, United States
Retina Consultants of San Diego
San Diego, California, 92064, United States
California Retina Consultants
Santa Barbara, California, 93103, United States
Retina Vitreous Associates of Florida
St. Petersburg, Florida, 33711, United States
Wilmer Eye Institute
Baltimore, Maryland, 21287, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114, United States
Sierra Eye Associates
Reno, Nevada, 89502, United States
Eye Associates of New Mexico
Albuquerque, New Mexico, 87109, United States
Wills Eye Hospital
Philadelphia, Pennsylvania, 19107, United States
Charles Retina Institute
Germantown, Tennessee, 38138, United States
Texas Retina Associates
Dallas, Texas, 75231, United States
Retina Consultants of Texas
The Woodlands, Texas, 77384, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 30, 2021
First Posted
April 6, 2021
Study Start
February 22, 2021
Primary Completion
October 2, 2023
Study Completion
March 18, 2024
Last Updated
September 5, 2024
Record last verified: 2024-09
Data Sharing
- IPD Sharing
- Will not share