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

Phase 1

Completed

• Conditions: Macular Degeneration; Age-related Maculopathies; Maculopathy,Age-related; Age-related Maculopathy; Maculopathies,Age-related; Retinal Degeneration; Eye Diseases; Retinal Neovascularization
• Interventions: Biological: rAAV.sFlt-1; Other: Control (ranibizumab alone)

• Registration Number: NCT01494805
• Lead Sponsor: Lions Eye Institute, Perth, Western Australia

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.

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 Timeline

• Study Start: 2011-12
• Study First Submitted: 2011-12-14
• Study First Submitted QC: 2011-12-15
• Study First Posted: 2011-12-19
• Primary Completion: 2017-05
• Status Verified: 2017-08
• Study Completion: 2017-08
• Last Update Submitted: 2017-08-30
• Last Update Posted: 2017-09-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Low Dose rAAV.sFlt-1	rAAV.sFlt-1	-
High Dose rAAV.sFlt-1	rAAV.sFlt-1	-
Control - ranibizumab only	Control (ranibizumab alone)	-

Primary Outcome Measures

Name	Time	Method
No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection	Primary endpoint at 1 month	1. Ocular examination: * Ocular inflammation; * Intraocular pressure; * Visual acuity; * Retinal bleeding; 2. Abnormal laboratory data

Secondary Outcome Measures

Name	Time	Method
Maintenance or improvement of vision without the necessity of ranibizumab re-injections	Up to 3 years	1. Best-corrected visual acuity; 2. CNV lesion; 3. Foveal thickness

Trial Locations

Locations (1)

Lions Eye Institute; 🇦🇺 Nedlands, Western Australia, Australia