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

Macular Degeneration Clinical Trial

— AMD  

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01494805
• Other study ID #: 2008-135
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: December 14, 2011
• Last updated: August 30, 2017
• Start date: December 2011
• Est. completion date: August 2017

Study information

• Verified date: August 2017
• Source: Lions Eye Institute, Perth, Western Australia
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: August 2017
• Est. primary completion date: May 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 55 Years and older

Eligibility

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;

Related Conditions & MeSH terms

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

Intervention

Biological:
• rAAV.sFlt-1
1 x 10^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
• 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).

Locations

Country	Name	City	State
Australia	Lions Eye Institute	Nedlands	Western Australia

Sponsors (2)

Lead Sponsor	Collaborator
Lions Eye Institute, Perth, Western Australia	Adverum Biotechnologies, Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection	Ocular examination: Ocular inflammation; Intraocular pressure; Visual acuity; Retinal bleeding; Abnormal laboratory data	Primary endpoint at 1 month
Secondary	Maintenance or improvement of vision without the necessity of ranibizumab re-injections	Best-corrected visual acuity; CNV lesion; Foveal thickness	Up to 3 years