Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
Public ClinicalTrials.gov record NCT03066258. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Excessive vascular endothelial growth factor (VEGF) plays a key part in promoting neovascularization and edema in neovascular (wet) age-related macular degeneration (nAMD). VEGF inhibitors (anti-VEGF), including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron), have been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.
Study identification
- NCT ID
- NCT03066258
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 42 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 50 Years to 89 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 28, 2017
- Primary completion
- Nov 23, 2019
- Completion
- Jun 16, 2021
- Last update posted
- May 15, 2023
2017 – 2021
United States locations
- U.S. sites
- 8
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Santa Barbara location | Santa Barbara | California | 93103 | — |
| Baltimore location | Baltimore | Maryland | 21287 | — |
| Boston location | Boston | Massachusetts | 02114 | — |
| Reno location | Reno | Nevada | 89502 | — |
| Philadelphia location 1 | Philadelphia | Pennsylvania | 19104 | — |
| Philadelphia location 2 | Philadelphia | Pennsylvania | 19107 | — |
| Memphis location | Germantown | Tennessee | 38138 | — |
| Houston location | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Recently updated Neovascular Age-related Macular Degeneration trials
Other public records listing Neovascular Age-related Macular Degeneration. Sorted by last update posted; newest first. Not a recommendation.
- NCT07614776: A Study to Evaluate Efficacy, Safety, and Immunogenicity With ABP 938 8 mg Versus EYLEA® HD (Aflibercept) in Participants With Neovascular Age-related Macular Degeneration Recruiting
- NCT06660667: A Safety and Efficacy Study of One-time SAR402663 in Adults With Neovascular Age-related Macular Degeneration Active, not recruiting
- NCT05904028: Home OCT-Guided Treatment Versus Treat and Extend for the Management of Neovascular AMD Recruiting
- NCT04853251: A Study Assessing Corneal Endothelial Cells in Participants With Neovascular Age-related Macular Degeneration (nAMD) Treated With the Port Delivery System With Ranibizumab (PDS) Recruiting
- NCT04704921: Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD Active, not recruiting
- NCT03683251: Extension Study for the Port Delivery System With Ranibizumab (Portal) Recruiting
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03066258, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 15, 2023 · Synced Sep 9, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03066258 live on ClinicalTrials.gov.