Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial

Part of paid clinical trials in Santa Barbara, California.

Sponsor
REGENXBIO Inc.
Study ID
NCT03066258
Phase
PHASE1/PHASE2
Status
Completed

Conditions

  • Neovascular Age-related Macular Degeneration
  • Wet Age-related Macular Degeneration

Eligibility Criteria

Sex
ALL
Age
50 Years - 89 Years
Healthy Volunteers
Not accepted

Interventions

  • RGX-314 — GENETIC
    RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Study Details

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.

Key Dates

First listed
Feb 28, 2017
Start date
Mar 29, 2017
Status verified
May 2023
Primary completion
Nov 24, 2019
Completion
Jun 17, 2021

Study Design

Enrollment
42 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    3E9 GC (genome copies)/eye of RGX-314 (E means the exponential constant)
  • Experimental: Cohort 2
    1E10 GC/eye of RGX-314
  • Experimental: Cohort 3
    6E10 GC/eye of RGX-314
  • Experimental: Cohort 4
    1.6E11 GC/eye of RGX-314
  • Experimental: Cohort 5
    2.5E11 GC/eye of RGX-314

Primary Outcome Measure

Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)) [ Time Frame: 26 weeks (24 weeks following RGX-314 administration) ]

Locations (8)

FacilityCityStateZIPSite coordinators
Santa Barbara locationSanta BarbaraCalifornia93103-
Baltimore locationBaltimoreMaryland21287-
Boston locationBostonMassachusetts02114-
Reno locationRenoNevada89502-
Philadelphia location 1PhiladelphiaPennsylvania19104-
Philadelphia location 2PhiladelphiaPennsylvania19107-
Memphis locationGermantownTennessee38138-
Houston locationHoustonTexas77030-

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