Overview

This trial is active, not recruiting.

Conditions macular degeneration, age-related maculopathies, age-related maculopathy, maculopathies,age-related, maculopathy,age-related, retinal degeneration, retinal neovascularization, eye diseases
Treatments raav.sflt-1, control (ranibizumab alone)
Phase phase 1/phase 2
Sponsor Lions Eye Institute, Perth, Western Australia
Collaborator Avalanche Biotechnologies, Inc.
Start date December 2011
End date May 2015
Trial size 40 participants
Trial identifier NCT01494805, 2008-135

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking single blind (outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
raav.sflt-1
1 x 10^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
(Experimental)
raav.sflt-1
1 x 10^11 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
(Active Comparator)
control (ranibizumab alone)
Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).

Primary Outcomes

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

Secondary Outcomes

Measure
Maintenance or improvement of vision without the necessity of ranibizumab re-injections
time frame: Up to 3 years

Eligibility Criteria

Male or female participants at least 55 years old.

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;

Additional Information

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)
Principal investigator Ian Constable, Professor
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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2014.
Information provided to ClinicalTrials.gov by Lions Eye Institute, Perth, Western Australia.