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 29, 2017 (Actual)
- Primary completion
- Nov 24, 2019 (Actual)
- Completion
- Jun 17, 2021 (Actual)
- Last update posted
- May 16, 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.
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 16, 2023 · Synced Oct 27, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03066258 live on ClinicalTrials.gov.