Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration

Overview

The study will involve approximately 40 subjects aged 55 or above who have exudative age-related macular degeneration (wet AMD). Patients will be randomized to receive one of two doses of rAAV.sFlt-1 or assigned to the control group.

Full Title of Study: “A Phase I/II Controlled Dose-escalating Trial to Establish the Baseline Safety and Efficacy of a Single Subretinal Injection of rAAV.sFlt-1 Into Eyes of Patients With Exudative Age-related Macular Degeneration (AMD)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Single (Outcomes Assessor)
  • Study Primary Completion Date: May 2017

Detailed Description

A new treatment for exudative age-related macular degeneration (wet AMD) is being investigated. The purpose of this Phase I/II clinical research study is to examine the baseline safety and efficacy of an experimental study drug to treat a complication of the disease which leads to vision loss. The name of the study drug is rAAV.sFlt-1. This experimental study uses a non-pathogenic virus to express a therapeutic protein within the eye. The therapeutic diminishes the growth of abnormal blood vessels under the retina. The duration of effect is thought to be long-term (years) following a single administration. The clinical research study will look at the baseline safety and efficacy of a single injection of rAAV.sFlt-1 injected directly into the eye. Approximately forty (40) subjects will participate in Australia. The primary endpoint of the study is at one month, with extended follow up for 3 years.

Interventions

  • Biological: rAAV.sFlt-1
    • 1 x 10^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
  • Biological: rAAV.sFlt-1
    • 1 x 10^11 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
  • Other: Control (ranibizumab alone)
    • Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).

Arms, Groups and Cohorts

  • Experimental: Low Dose rAAV.sFlt-1
  • Experimental: High Dose rAAV.sFlt-1
  • Active Comparator: Control – ranibizumab only

Clinical Trial Outcome Measures

Primary Measures

  • No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection
    • Time Frame: Primary endpoint at 1 month
    • Ocular examination: Ocular inflammation Intraocular pressure Visual acuity Retinal bleeding Abnormal laboratory data

Secondary Measures

  • Maintenance or improvement of vision without the necessity of ranibizumab re-injections
    • Time Frame: Up to 3 years
    • Best-corrected visual acuity CNV lesion Foveal thickness

Participating in This Clinical Trial

Inclusion Criteria

  • Age greater than or equal to 55 years; – Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 – 6/9 with 6/60 or better in the other eye; – Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or CNV currently under active management with anti-VEGF therapy; – Must be a candidate for anti-VEGF intravitreal injections; – No previous retinal treatment of photodynamic therapy or laser; – Able to provide informed consent; – Able to comply with protocol requirements, including follow-up visits. Exclusion Criteria:

  • Liver enzymes > 2 X upper limit of normal; – Any prior treatment for AMD in the study / control eye, excluding anti-VEGF injections; – Extensive sub-foveal scarring, extensive geographic atrophy, or thick subretinal blood in the study eye as determined by the investigator; – Significant retinal disease other than sub-foveal CNV AMD;

Gender Eligibility: All

Minimum Age: 55 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Lions Eye Institute, Perth, Western Australia
  • Collaborator
    • Adverum Biotechnologies, Inc.
  • Provider of Information About this Clinical Study
    • Principal Investigator: Prof. P. Elizabeth Rakoczy, Principal Scientific Investigator – Lions Eye Institute, Perth, Western Australia
  • Overall Official(s)
    • Ian Constable, Professor, Principal Investigator, Lions Eye Institute

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