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RGX-314 Gene Therapy for Neovascular AMD Trial

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ClinicalTrials.gov Identifier: NCT03066258
Recruitment Status : Recruiting
First Posted : February 28, 2017
Last Update Posted : December 6, 2017
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

February 17, 2017
February 28, 2017
December 6, 2017
March 31, 2017
August 2018   (Final data collection date for primary outcome measure)
Safety (incidence of ocular and non-ocular AEs and SAEs) [ Time Frame: 26 weeks ]
incidence of ocular and non-ocular AEs and SAEs
Same as current
Complete list of historical versions of study NCT03066258 on ClinicalTrials.gov Archive Site
  • 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
Same as current
Not Provided
Not Provided
 
RGX-314 Gene Therapy for Neovascular AMD Trial
A Phase I, 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)
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.
This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of RGX-314 gene therapy in subjects with previously treated nAMD. Three doses will be studied in approximately 18 subjects. Subjects who meet the inclusion/exclusion criteria and have an anatomic response to an initial anti VEGF injection will receive a single dose of RGX-314 administered by subretinal delivery. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. Safety will be the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects will continue to be assessed until 104 weeks following treatment with RGX-314.
Interventional
Phase 1
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:
dose escalation
Masking: None (Open Label)
Primary Purpose: Treatment
  • Neovascular Age-related Macular Degeneration
  • Wet Age-related Macular Degeneration
Biological: 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 1
    3E9 GC of RGX-314
    Intervention: Biological: RGX-314
  • Experimental: Dose 2
    1E10 GC of RGX-314
    Intervention: Biological: RGX-314
  • Experimental: Dose 3
    6E10 GC of RGX-314
    Intervention: Biological: RGX-314
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
18
Same as current
February 2020
August 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Patients ≥ 50 years with a diagnosis of subfoveal CNV secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy.
  2. 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.
  3. History of need for and response to anti-VEGF.therapy.
  4. Response to anti-VEGF at trial entry (assessed by SD-OCT at week 1)
  5. Must be pseudophakic (status post cataract surgery) in the study eye.
  6. 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
  7. Must be willing and able to provide written, signed informed consent.

Exclusion Criteria:

  1. CNV or macular edema in the study eye secondary to any causes other than AMD.
  2. Any condition preventing visual acuity improvement in the study eye, eg, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea.
  3. Active or history of retinal detachment in the study eye.
  4. Advanced glaucoma in the study eye.
  5. 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.
  6. Presence of an implant in the study eye at screening (excluding intraocular lens).
  7. Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within the past 6 months.
  8. Uncontrolled hypertension (systolic blood pressure [BP] >180 mmHg, diastolic BP >100 mmHg) despite maximal medical treatment.
Sexes Eligible for Study: All
50 Years and older   (Adult, Older Adult)
No
Contact: Rickey Reinhardt, MD, PhD 2405528181 patientadvocacy@regenxbio.com
United States
 
 
NCT03066258
RGX-314-001
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Regenxbio Inc.
Regenxbio Inc.
Not Provided
Principal Investigator: Jeffrey Heier, MD Ophthalmic Consultants of Boston
Regenxbio Inc.
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP