NCT01494805

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)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Lions Eye Institute, Perth, Western Australia · Academic / Other
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Macular Degeneration
• Age-related Maculopathies
• Age-related Maculopathy
• Maculopathies,Age-related
• Maculopathy,Age-related
• Retinal Degeneration
• Retinal Neovascularization
• Eye Diseases

Interventions

Type	Name	Description
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).

Timeline

• Start date: 2011-12-01
• Primary completion: 2017-05-01
• Completion: 2017-08-01
• First posted: 2011-12-19
• Last updated: 2017-09-01

Locations

1 site across 1 country: Australia