GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

Macular Degeneration Clinical Trial

Official title:

A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03815825
• Other study ID #: ISIS 696844-CS5
• Secondary ID: 2020-005174-94
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: March 4, 2019
• Est. completion date: June 2024

Study information

• Verified date: May 2024
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).

Description:

The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at multiple centers. It is an adaptive design in which three dose levels will be evaluated in a subset of participants (Stage 1) and, following an interim analysis, the number of participants in two of the dose cohorts will be expanded (Stage 2).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 332
• Est. completion date: June 2024
• Est. primary completion date: April 18, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years and older

Eligibility

Inclusion Criteria:

• Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
• Well-demarcated geographic atrophy (GA) due to AMD
• Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
• Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging

Exclusion Criteria:

• Clinically-significant abnormalities in medical history
• A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
• Chronic treatment with steroids, including topically or intravitreally administered
• History or presence of diabetic retinopathy or diabetic macular edema (DME)
• History or presence of a disease other than AMD that could affect vision or safety assessments
• Prior treatment with another investigational drug, biological agent, or device
• Other protocol-specified inclusion/exclusion criteria may apply

Related Conditions & MeSH terms

• Atrophy
• Geographic Atrophy
• Macular Degeneration

Intervention

Drug:
• IONIS-FB-LRx
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
• Placebo
Placebo matching solution, administered subcutaneously every 4 weeks

Locations

Country	Name	City	State
Australia	IONIS Investigative Site	Adelaide	South Australia
Australia	IONIS Investigative Site	Albury	New South Wales
Australia	IONIS Investigative Site	East Melbourne	Victoria
Australia	IONIS Investigative Site	Essendon	Victoria
Australia	IONIS Investigative Site	Glen Iris	Victoria
Australia	IONIS Investigative Site	Hurstville	New South Wales
Australia	IONIS Investigative Site	Malvern	Victoria
Australia	IONIS Investigative Site	Parkville	Victoria
Australia	IONIS Investigative Site	Parramatta	New South Wales
Australia	IONIS Investigative Site	Perth	Western Australia
Australia	IONIS Investigative Site	Sydney	New South Wales
Austria	IONIS Investigative Site	Klagenfurt
Austria	IONIS Investigative Site	Linz	Oberösterreich
Austria	IONIS Investigative Site	Vienna
Austria	IONIS Investigative Site	Vienna
Canada	IONIS Investigative Site	London	Ontario
Canada	IONIS Investigative Site	Ottawa	Ontario
Canada	IONIS Investigative Site	Ottawa	Ontario
Canada	IONIS Investigative Site	Québec	Quebec
Czechia	IONIS Investigative Site	Prague
Netherlands	IONIS Investigative Site	Nijmegen	Gelderland
Netherlands	IONIS Investigative Site	Rotterdam
New Zealand	IONIS Investigative Site	Christchurch
Poland	IONIS Investigative Site	Bydgoszcz
Poland	IONIS Investigative Site	Katowice	Silesian
Spain	IONIS Investigative Site	Barcelona
Spain	IONIS Investigative Site	Barcelona
Spain	IONIS Investigative Site	Burjassot	Valencia
Spain	IONIS Investigative Site	Majadahonda	Madrid
Spain	IONIS Investigative Site	Pamplona	Navarra
Spain	IONIS Investigative Site	Terrassa	Barcelona
Spain	IONIS Investigative Site	Valladolid
Spain	IONIS Investigative Site	Zaragoza
United States	IONIS Investigative Site	Abilene	Texas
United States	IONIS Investigative Site	Albuquerque	New Mexico
United States	IONIS Investigative Site	Arlington	Texas
United States	IONIS Investigative Site	Asheville	North Carolina
United States	IONIS Investigative Site	Augusta	Georgia
United States	IONIS Investigative Site	Austin	Texas
United States	IONIS Investigative Site	Baltimore	Maryland
United States	IONIS Investigative Site	Baltimore	Maryland
United States	IONIS Investigative Site	Bellaire	Texas
United States	IONIS Investigative Site	Beverly Hills	California
United States	IONIS Investigative Site	Clearwater	Florida
United States	IONIS Investigative Site	Cleveland	Ohio
United States	IONIS Investigative Site	Cleveland	Ohio
United States	IONIS Investigative Site	Cleveland	Ohio
United States	IONIS Investigative Site	Colorado Springs	Colorado
United States	IONIS Investigative Site	Dallas	Texas
United States	IONIS Investigative Site	Fort Myers	Florida
United States	IONIS Investigative Site	Greenville	South Carolina
United States	IONIS Investigative Site	Hagerstown	Maryland
United States	IONIS Investigative Site	Hickory	North Carolina
United States	IONIS Investigative Site	Huntington Beach	California
United States	IONIS Investigative Site	La Jolla	California
United States	IONIS Investigative Site	Ladson	South Carolina
United States	IONIS Investigative Site	Lake Worth	Florida
United States	IONIS Investigative Site	Lakeland	Florida
United States	IONIS Investigative Site	Leawood	Kansas
United States	IONIS Investigative Site	Lemont	Illinois
United States	IONIS Investigative Site	Long Beach	California
United States	IONIS Investigative Site	Lynchburg	Virginia
United States	IONIS Investigative Site	McAllen	Texas
United States	IONIS Investigative Site	Melbourne	Florida
United States	IONIS Investigative Site	Nashville	Tennessee
United States	IONIS Investigative Site	Philadelphia	Pennsylvania
United States	IONIS Investigative Site	Phoenix	Arizona
United States	IONIS Investigative Site	Phoenix	Arizona
United States	IONIS Investigative Site	Phoenix	Arizona
United States	IONIS Investigative Site	Pittsburgh	Pennsylvania
United States	IONIS Investigative Site	Poway	California
United States	IONIS Investigative Site	Richmond	Virginia
United States	IONIS Investigative Site	Salt Lake City	Utah
United States	IONIS Investigative Site	San Antonio	Texas
United States	IONIS Investigative Site	San Francisco	California
United States	IONIS Investigative Site	Silverdale	Washington
United States	IONIS Investigative Site	Southaven	Mississippi
United States	IONIS Investigative Site	Southlake	Texas
United States	IONIS Investigative Site	Springfield	Illinois
United States	IONIS Investigative Site	Stuart	Florida
United States	IONIS Investigative Site	Sun City	Arizona
United States	IONIS Investigative Site	Tampa	Florida
United States	IONIS Investigative Site	The Woodlands	Texas
United States	IONIS Investigative Site	Tulsa	Oklahoma
United States	IONIS Investigative Site	West Mifflin	Pennsylvania
United States	IONIS Investigative Site	Willow Park	Texas
United States	IONIS Investigative Site	Winston-Salem	North Carolina
United States	IONIS Investigative Site	Winter Haven	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Canada,  Czechia,  Netherlands,  New Zealand,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging		Week 49
Secondary	Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)		Baseline and up to Week 57
Secondary	Percentage Change from Baseline in Levels of Factor B (FB) in Plasma		Baseline and up to Week 49
Secondary	Percentage Change from Baseline in Levels of Serum AH50 Activity		Baseline and up to Week 49
Secondary	Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)		Baseline and up to Week 49