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

This study is currently recruiting participants.
Verified September 2012 by Lions Eye Institute, Perth, Western Australia
Sponsor:
Collaborator:
Avalanche Biotechnologies, Inc.
Information provided by (Responsible Party):
Prof. P. Elizabeth Rakoczy, Lions Eye Institute, Perth, Western Australia
ClinicalTrials.gov Identifier:
NCT01494805
First received: December 14, 2011
Last updated: September 11, 2012
Last verified: September 2012
History of Changes
  Purpose

The study will involve 48 patients 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.


Condition Intervention Phase
Macular Degeneration
Age-related Maculopathies
Age-related Maculopathy
Maculopathies,Age-related
Maculopathy,Age-related
Retinal Degeneration
Retinal Neovascularization
Eye Diseases
 Biological: rAAV.sFlt-1
Other: Control (ranibizumab alone)
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)

Resource links provided by NLM:

Drug Information available for: Ranibizumab
U.S. FDA Resources

Further study details as provided by Lions Eye Institute, Perth, Western Australia:

Primary Outcome 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 ] [ Designated as safety issue: Yes ]

    1. Ocular examination:

      • Ocular inflammation
      • Intraocular pressure
      • Visual acuity
      • Retinal bleeding
    2. Abnormal laboratory data


Secondary Outcome Measures:
  • Maintenance or improvement of vision without the necessity of ranibizumab re-injections [ Time Frame: Up to 3 years ] [ Designated as safety issue: No ]
    1. Best-corrected visual acuity
    2. CNV lesion
    3. Foveal thickness


Estimated Enrollment: 48
Study Start Date: December 2011
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Low Dose rAAV.sFlt-1 Biological: rAAV.sFlt-1
1 x 10^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
Experimental: High Dose rAAV.sFlt-1 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).

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.

Forty-eight (48) patients will participate in Australia. The primary endpoint of the study is at one month, with extended follow up for 3 years.

  Eligibility

Ages Eligible for Study:   55 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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;
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01494805

Contacts
Contact: Cora Pierce, RN +61 8 9381 0750 corapierce@lei.org.au
Contact: Clinical Network Services +61(0)7 3719 6000 cns@clinical.net.au

Locations
Australia, Western Australia
Lions Eye Institute Recruiting
Nedlands, Western Australia, Australia, 6009
Principal Investigator: Ian Constable, Professor            
Sponsors and Collaborators
Lions Eye Institute, Perth, Western Australia
Avalanche Biotechnologies, Inc.
Investigators
Principal Investigator: Ian Constable, Professor Lions Eye Institute
  More Information

No publications provided

Responsible Party: Prof. P. Elizabeth Rakoczy, Principal Scientific Investigator, Lions Eye Institute, Perth, Western Australia
ClinicalTrials.gov Identifier: NCT01494805     History of Changes
Other Study ID Numbers: 2008-135
Study First Received: December 14, 2011
Last Updated: September 11, 2012
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by Lions Eye Institute, Perth, Western Australia:
Age-related Macular Degeneration
AMD
Wet AMD
Neovascular AMD
Gene Therapy
Ocular
Ocular Gene Therapy
 Eye diseases
Macular Degeneration
Retinal Degeneration
Retinal Neovascularization
Wet Macular Degeneration
Retinal Diseases

Additional relevant MeSH terms:
Eye Diseases
Macular Degeneration
Neovascularization, Pathologic
Retinal Degeneration
Retinal Neovascularization
 Wet Macular Degeneration
Retinal Diseases
Metaplasia
Pathologic Processes

ClinicalTrials.gov processed this record on May 16, 2013