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

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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