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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

December 14, 2011
September 11, 2012
December 2011
June 2013   (final data collection date for primary outcome measure)
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
Same as current
Complete list of historical versions of study NCT01494805 on ClinicalTrials.gov Archive Site
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
Same as current
Not Provided
Not Provided
 
Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration
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 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.

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.
Interventional
Phase 1
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
  • Macular Degeneration
  • Age-related Maculopathies
  • Age-related Maculopathy
  • Maculopathies,Age-related
  • Maculopathy,Age-related
  • Retinal Degeneration
  • Retinal Neovascularization
  • Eye Diseases
  • 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).
  • Experimental: Low Dose rAAV.sFlt-1
    Intervention: Biological: rAAV.sFlt-1
  • Experimental: High Dose rAAV.sFlt-1
    Intervention: Biological: rAAV.sFlt-1
  • Active Comparator: Control - ranibizumab only
    Intervention: Other: Control (ranibizumab alone)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
48
December 2014
June 2013   (final data collection date for primary outcome measure)

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;
Both
55 Years and older
No
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
Australia
 
NCT01494805
2008-135
Yes
Prof. P. Elizabeth Rakoczy, Lions Eye Institute, Perth, Western Australia
Lions Eye Institute, Perth, Western Australia
Avalanche Biotechnologies, Inc.
Principal Investigator: Ian Constable, Professor Lions Eye Institute
Lions Eye Institute, Perth, Western Australia
September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP