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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01494805
Recruitment Status : Completed
First Posted : December 19, 2011
Last Update Posted : September 1, 2017
Sponsor:
Collaborator:
Adverum Biotechnologies, Inc.
Information provided by (Responsible Party):
Prof. P. Elizabeth Rakoczy, Lions Eye Institute, Perth, Western Australia

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment 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

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (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)
Actual Study Start Date : December 2011
Actual Primary Completion Date : May 2017
Actual Study Completion Date : August 2017

Resource links provided by the National Library of Medicine

Drug Information available for: Ranibizumab

Arms and Interventions
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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
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).


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   55 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01494805


Locations
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Australia, Western Australia
Lions Eye Institute
Nedlands, Western Australia, Australia, 6009
Sponsors and Collaborators
Lions Eye Institute, Perth, Western Australia
Adverum Biotechnologies, Inc.
Investigators
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Principal Investigator: Ian Constable, Professor Lions Eye Institute
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Prof. P. Elizabeth Rakoczy, Principal Scientific Investigator, Lions Eye Institute, Perth, Western Australia
ClinicalTrials.gov Identifier: NCT01494805    
Other Study ID Numbers: 2008-135
First Posted: December 19, 2011    Key Record Dates
Last Update Posted: September 1, 2017
Last Verified: August 2017
Keywords provided by Prof. P. Elizabeth Rakoczy, Lions Eye Institute, Perth, Western Australia:
Age-related Macular Degeneration
AAV
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:
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Macular Degeneration
Eye Diseases
Retinal Degeneration
Retinal Neovascularization
Neovascularization, Pathologic
Retinal Diseases
Metaplasia
Pathologic Processes
 Eye Diseases, Hereditary
Ranibizumab
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents