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

August 30, 2017 updated by: Prof. P. Elizabeth Rakoczy, Lions Eye Institute, Perth, Western Australia

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

40

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Lions Eye Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

55 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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;

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low Dose rAAV.sFlt-1
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
Experimental: High Dose rAAV.sFlt-1
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
Active Comparator: Control - ranibizumab only
Other: Control (ranibizumab alone)
Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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

  1. Ocular examination:

    • Ocular inflammation
    • Intraocular pressure
    • Visual acuity
    • Retinal bleeding
  2. Abnormal laboratory data
Primary endpoint at 1 month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Maintenance or improvement of vision without the necessity of ranibizumab re-injections
Time Frame: Up to 3 years
  1. Best-corrected visual acuity
  2. CNV lesion
  3. Foveal thickness
Up to 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lions Eye Institute, Perth, Western Australia

Collaborators

Adverum Biotechnologies, Inc.

Investigators

  • Principal Investigator: Ian Constable, Professor, Lions Eye Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2011

Primary Completion (Actual)

May 1, 2017

Study Completion (Actual)

August 1, 2017

Study Registration Dates

First Submitted

December 14, 2011

First Submitted That Met QC Criteria

December 15, 2011

First Posted (Estimate)

December 19, 2011

Study Record Updates

Last Update Posted (Actual)

September 1, 2017

Last Update Submitted That Met QC Criteria

August 30, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2008-135

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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