Clinical trial NCT03815825: 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)
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Macular Degeneration
Geographic Atrophy
Lead sponsor: Ionis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-01-24
Enrollment: 332
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

Detailed 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).

Eligibility criteria: 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

Primary outcomes:
measure: Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
timeFrame: Week 49
Secondary outcomes:
measure: Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)
timeFrame: Baseline and up to Week 57
measure: Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
timeFrame: Baseline and up to Week 49
measure: Percentage Change from Baseline in Levels of Serum AH50 Activity
timeFrame: Baseline and up to Week 49
measure: Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
timeFrame: Baseline and up to Week 49
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: IONIS Investigative Site
state: Arizona
city: Phoenix
country: United States
facility: IONIS Investigative Site
state: Arizona
city: Phoenix
country: United States
facility: IONIS Investigative Site
state: Arizona
city: Sun City
country: United States
facility: IONIS Investigative Site
state: Arizona
city: Beverly Hills
country: United States
facility: IONIS Investigative Site
state: California
city: Huntington Beach
country: United States
facility: IONIS Investigative Site
state: California
city: La Jolla
country: United States
facility: IONIS Investigative Site
state: California
city: Long Beach
country: United States
facility: IONIS Investigative Site
state: California
city: Poway
country: United States
facility: IONIS Investigative Site
state: California
city: San Francisco
country: United States
facility: IONIS Investigative Site
state: California
city: Colorado Springs
country: United States
facility: IONIS Investigative Site
state: Colorado
city: Clearwater
country: United States
facility: IONIS Investigative Site
state: Florida
city: Fort Myers
country: United States
facility: IONIS Investigative Site
state: Florida
city: Lake Worth
country: United States
facility: IONIS Investigative Site
state: Florida
city: Lakeland
country: United States
facility: IONIS Investigative Site
state: Florida
city: Melbourne
country: United States
facility: IONIS Investigative Site
state: Florida
city: Stuart
country: United States
facility: IONIS Investigative Site
state: Florida
city: Tampa
country: United States
facility: IONIS Investigative Site
state: Florida
city: Winter Haven
country: United States
facility: IONIS Investigative Site
state: Florida
city: Augusta
country: United States
facility: IONIS Investigative Site
state: Georgia
city: Lemont
country: United States
facility: IONIS Investigative Site
state: Illinois
city: Springfield
country: United States
facility: IONIS Investigative Site
state: Illinois
city: Leawood
country: United States
facility: IONIS Investigative Site
state: Kansas
city: Baltimore
country: United States
facility: IONIS Investigative Site
state: Maryland
city: Baltimore
country: United States
facility: IONIS Investigative Site
state: Maryland
city: Hagerstown
country: United States
facility: IONIS Investigative Site
state: Maryland
city: Southaven
country: United States
facility: IONIS Investigative Site
state: Mississippi
city: Albuquerque
country: United States
facility: IONIS Investigative Site
state: New Mexico
city: Asheville
country: United States
facility: IONIS Investigative Site
state: North Carolina
city: Hickory
country: United States
facility: IONIS Investigative Site
state: North Carolina
city: Winston-Salem
country: United States
facility: IONIS Investigative Site
state: North Carolina
city: Cleveland
country: United States
facility: IONIS Investigative Site
state: Ohio
city: Cleveland
country: United States
facility: IONIS Investigative Site
state: Ohio
city: Cleveland
country: United States
facility: IONIS Investigative Site
state: Ohio
city: Tulsa
country: United States
facility: IONIS Investigative Site
state: Oklahoma
city: Philadelphia
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: West Mifflin
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Greenville
country: United States
facility: IONIS Investigative Site
state: South Carolina
city: Ladson
country: United States
facility: IONIS Investigative Site
state: South Carolina
city: Nashville
country: United States
facility: IONIS Investigative Site
state: Tennessee
city: Abilene
country: United States
facility: IONIS Investigative Site
state: Texas
city: Arlington
country: United States
facility: IONIS Investigative Site
state: Texas
city: Austin
country: United States
facility: IONIS Investigative Site
state: Texas
city: Bellaire
country: United States
facility: IONIS Investigative Site
state: Texas
city: Dallas
country: United States
facility: IONIS Investigative Site
state: Texas
city: McAllen
country: United States
facility: IONIS Investigative Site
state: Texas
city: San Antonio
country: United States
facility: IONIS Investigative Site
state: Texas
city: Southlake
country: United States
facility: IONIS Investigative Site
state: Texas
city: The Woodlands
country: United States
facility: IONIS Investigative Site
state: Texas
city: Willow Park
country: United States
facility: IONIS Investigative Site
state: Texas
city: Salt Lake City
country: United States
facility: IONIS Investigative Site
state: Utah
city: Lynchburg
country: United States
facility: IONIS Investigative Site
state: Virginia
city: Richmond
country: United States
facility: IONIS Investigative Site
state: Virginia
city: Silverdale
country: United States
facility: IONIS Investigative Site
state: Washington
city: Albury
country: Australia
facility: IONIS Investigative Site
state: New South Wales
city: Hurstville
country: Australia
facility: IONIS Investigative Site
state: New South Wales
city: Parramatta
country: Australia
facility: IONIS Investigative Site
state: New South Wales
city: Sydney
country: Australia
facility: IONIS Investigative Site
state: New South Wales
city: Adelaide
country: Australia
facility: IONIS Investigative Site
state: South Australia
city: East Melbourne
country: Australia
facility: IONIS Investigative Site
state: Victoria
city: Essendon
country: Australia
facility: IONIS Investigative Site
state: Victoria
city: Glen Iris
country: Australia
facility: IONIS Investigative Site
state: Victoria
city: Malvern
country: Australia
facility: IONIS Investigative Site
state: Victoria
city: Parkville
country: Australia
facility: IONIS Investigative Site
state: Victoria
city: Perth
country: Australia
facility: IONIS Investigative Site
state: Western Australia
city: Linz
country: Austria
facility: IONIS Investigative Site
state: Oberösterreich
city: Klagenfurt
country: Austria
facility: IONIS Investigative Site
city: Vienna
country: Austria
facility: IONIS Investigative Site
city: Vienna
country: Austria
facility: IONIS Investigative Site
city: London
country: Canada
facility: IONIS Investigative Site
state: Ontario
city: Ottawa
country: Canada
facility: IONIS Investigative Site
state: Ontario
city: Ottawa
country: Canada
facility: IONIS Investigative Site
state: Ontario
city: Québec
country: Canada
facility: IONIS Investigative Site
state: Quebec
city: Prague
country: Czechia
facility: IONIS Investigative Site
city: Nijmegen
country: Netherlands
facility: IONIS Investigative Site
state: Gelderland
city: Rotterdam
country: Netherlands
facility: IONIS Investigative Site
city: Christchurch
country: New Zealand
facility: IONIS Investigative Site
city: Katowice
country: Poland
facility: IONIS Investigative Site
state: Silesian
city: Bydgoszcz
country: Poland
facility: IONIS Investigative Site
city: Terrassa
country: Spain
facility: IONIS Investigative Site
state: Barcelona
city: Majadahonda
country: Spain
facility: IONIS Investigative Site
state: Madrid
city: Pamplona
country: Spain
facility: IONIS Investigative Site
state: Navarra
city: Burjassot
country: Spain
facility: IONIS Investigative Site
state: Valencia
city: Barcelona
country: Spain
facility: IONIS Investigative Site
city: Barcelona
country: Spain
facility: IONIS Investigative Site
city: Valladolid
country: Spain
facility: IONIS Investigative Site
city: Zaragoza
country: Spain
facility: IONIS Investigative Site
Study officials:
References: