Safety and Tolerability of RGX-314 Gene Therapy for Neovascular AMD Trial
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03066258 |
Recruitment Status :
Active, not recruiting
First Posted : February 28, 2017
Last Update Posted : April 2, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Neovascular Age-related Macular Degeneration Wet Age-related Macular Degeneration | Genetic: RGX-314 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 42 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Intervention Model Description: | dose escalation |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase I/IIa, 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) |
Actual Study Start Date : | March 31, 2017 |
Actual Primary Completion Date : | November 24, 2019 |
Estimated Study Completion Date : | May 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Dose 1
3E9 GC/eye of RGX-314
|
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
Experimental: Dose 2
1E10 GC/eye of RGX-314
|
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
Experimental: Dose 3
6E10 GC/eye of RGX-314
|
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
Experimental: Dose 4
1.6E11 GC/eye of RGX-314
|
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
Experimental: Dose 5
2.5E11 GC/eye of RGX-314
|
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
- Safety (incidence of ocular and non-ocular AEs and SAEs) [ Time Frame: 26 weeks ]incidence of ocular and non-ocular AEs and SAEs
- Safety (incidence of ocular and non-ocular AEs and SAEs) [ Time Frame: 106 weeks ]incidence of ocular and non-ocular AEs and SAEs
- Change in best corrected visual acuity [ Time Frame: 106 weeks ]BCVA
- Change in central retinal thickness [ Time Frame: 106 weeks ]CRT as measured by SD-OCT
- Rescue injections (Mean number of rescue injections) [ Time Frame: 106 weeks ]Mean number of rescue injections
- Mean change in area of CNV [ Time Frame: 106 weeks ]Area of CNV and leakage measured by FA

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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 |
Inclusion Criteria:
- Patients ≥ 50 and ≤ 89 years with a diagnosis of subfoveal CNV secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy.
- BCVA between ≤20/63 and ≥20/400 (≤63 and ≥19 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400 (≤73 and ≥19 ETDRS letters) for the rest of the cohort.
- History of need for and response to anti-VEGF therapy.
- Response to anti-VEGF at trial entry (assessed by SD-OCT at week 1)
- Must be pseudophakic (status post cataract surgery) in the study eye.
- AST/ALT < 2.5 × ULN; TB < 1.5 × ULN; PT < 1.5 × ULN; Hb > 10 g/dL (males) and > 9 g/dL (females); Platelets > 100 × 10^3/µL; eGFR > 30 mL/min/1.73 m^2
- Must be willing and able to provide written, signed informed consent.
Exclusion Criteria:
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Any condition preventing visual acuity improvement in the study eye, eg, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea.
- Active or history of retinal detachment in the study eye.
- Advanced glaucoma in the study eye.
- History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening.
- Presence of an implant in the study eye at screening (excluding intraocular lens).
- Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within the past 6 months.
- Uncontrolled hypertension (systolic blood pressure [BP] >180 mmHg, diastolic BP >100 mmHg) despite maximal medical treatment.

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): NCT03066258
United States, California | |
Santa Barbara location | |
Santa Barbara, California, United States, 93103 | |
United States, Maryland | |
Baltimore location | |
Baltimore, Maryland, United States, 21287 | |
United States, Massachusetts | |
Boston location | |
Boston, Massachusetts, United States, 02114 | |
United States, Nevada | |
Reno location | |
Reno, Nevada, United States, 89502 | |
United States, Pennsylvania | |
Philadelphia location 1 | |
Philadelphia, Pennsylvania, United States, 19104 | |
Philadelphia location 2 | |
Philadelphia, Pennsylvania, United States, 19107 | |
United States, Tennessee | |
Memphis location | |
Germantown, Tennessee, United States, 38138 | |
United States, Texas | |
Houston location | |
Houston, Texas, United States, 77030 |
Principal Investigator: | Jeffrey Heier, MD | Ophthalmic Consultants of Boston |
Responsible Party: | REGENXBIO Inc. |
ClinicalTrials.gov Identifier: | NCT03066258 |
Other Study ID Numbers: |
RGX-314-001 |
First Posted: | February 28, 2017 Key Record Dates |
Last Update Posted: | April 2, 2021 |
Last Verified: | April 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
nAMD wet AMD gene therapy |
Macular Degeneration Retinal Degeneration Retinal Diseases Eye Diseases |