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)

March 5, 2025 updated by: Ionis Pharmaceuticals, Inc.

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)

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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).

Study Type

Interventional

Enrollment (Actual)

332

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Albury, New South Wales, Australia, 2640
        • Ionis Investigative Site
      • Hurstville, New South Wales, Australia, 2228
        • Ionis Investigative Site
      • Parramatta, New South Wales, Australia, 2150
        • Ionis Investigative Site
      • Sydney, New South Wales, Australia, 2000
        • Ionis Investigative Site
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Ionis Investigative Site
    • Victoria
      • East Melbourne, Victoria, Australia, 3002
        • Ionis Investigative Site
      • Essendon, Victoria, Australia, 3040
        • Ionis Investigative Site
      • Glen Iris, Victoria, Australia, 3146
        • Ionis Investigative Site
      • Malvern, Victoria, Australia, 3144
        • Ionis Investigative Site
      • Parkville, Victoria, Australia, 3050
        • Ionis Investigative Site
    • Western Australia
      • Perth, Western Australia, Australia, 6009
        • Ionis Investigative Site
  • Austria
      • Klagenfurt, Austria, 9020
        • Ionis Investigative Site
      • Vienna, Austria, 1090
        • Ionis Investigative Site
      • Vienna, Austria, 1140
        • Ionis Investigative Site
    • Oberösterreich
      • Linz, Oberösterreich, Austria, 4020
        • Ionis Investigative Site
  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 4V2
        • Ionis Investigative Site
      • Ottawa, Ontario, Canada, K1H 8L6
        • Ionis Investigative Site
      • Ottawa, Ontario, Canada, K2B 7E9
        • Ionis Investigative Site
    • Quebec
      • Québec, Quebec, Canada, G1S 4L8
        • Ionis Investigative Site
  • Czechia
      • Prague, Czechia, 15000
        • Ionis Investigative Site
  • Netherlands
      • Rotterdam, Netherlands, 3011 BH
        • Ionis Investigative Site
    • Gelderland
      • Nijmegen, Gelderland, Netherlands, 6525EX
        • Ionis Investigative Site
  • New Zealand
      • Christchurch, New Zealand, 8013
        • Ionis Investigative Site
  • Poland
      • Bydgoszcz, Poland, 85-631
        • Ionis Investigative Site
    • Silesian
      • Katowice, Silesian, Poland, 40-514
        • Ionis Investigative Site
  • Spain
      • Barcelona, Spain, 8022
        • Ionis Investigative Site
      • Barcelona, Spain, 8034
        • Ionis Investigative Site
      • Valladolid, Spain, 47012
        • Ionis Investigative Site
      • Zaragoza, Spain, 50009
        • Ionis Investigative Site
    • Barcelona
      • Terrassa, Barcelona, Spain, 8227
        • Ionis Investigative Site
    • Madrid
      • Majadahonda, Madrid, Spain, 28222
        • Ionis Investigative Site
    • Navarra
      • Pamplona, Navarra, Spain, 31008
        • Ionis Investigative Site
    • Valencia
      • Burjassot, Valencia, Spain, 46100
        • Ionis Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85020
        • Ionis Investigative Site
      • Phoenix, Arizona, United States, 85021
        • Ionis Investigative Site
      • Phoenix, Arizona, United States, 85053
        • Ionis Investigative Site
      • Sun City, Arizona, United States, 85351
        • Ionis Investigative Site
    • California
      • Beverly Hills, California, United States, 90211
        • Ionis Investigative Site
      • Huntington Beach, California, United States, 92697
        • Ionis Investigative Site
      • La Jolla, California, United States, 92093
        • Ionis Investigative Site
      • Long Beach, California, United States, 90807
        • Ionis Investigative Site
      • Poway, California, United States, 92064
        • Ionis Investigative Site
      • San Francisco, California, United States, 94109
        • Ionis Investigative Site
    • Colorado
      • Colorado Springs, Colorado, United States, 80909
        • Ionis Investigative Site
    • Florida
      • Clearwater, Florida, United States, 33761
        • Ionis Investigative Site
      • Fort Myers, Florida, United States, 33907
        • Ionis Investigative Site
      • Lake Worth, Florida, United States, 33461
        • Ionis Investigative Site
      • Lakeland, Florida, United States, 33801
        • Ionis Investigative Site
      • Melbourne, Florida, United States, 32901
        • Ionis Investigative Site
      • Stuart, Florida, United States, 34994
        • Ionis Investigative Site
      • Tampa, Florida, United States, 33612
        • Ionis Investigative Site
      • Winter Haven, Florida, United States, 33880
        • Ionis Investigative Site
    • Georgia
      • Augusta, Georgia, United States, 30909
        • Ionis Investigative Site
    • Illinois
      • Lemont, Illinois, United States, 60439
        • Ionis Investigative Site
      • Springfield, Illinois, United States, 62702
        • Ionis Investigative Site
    • Kansas
      • Leawood, Kansas, United States, 66211
        • Ionis Investigative Site
    • Maryland
      • Baltimore, Maryland, United States, 21204
        • Ionis Investigative Site
      • Baltimore, Maryland, United States, 21209
        • Ionis Investigative Site
      • Hagerstown, Maryland, United States, 21740
        • Ionis Investigative Site
    • Mississippi
      • Southaven, Mississippi, United States, 38671
        • Ionis Investigative Site
    • New Mexico
      • Albuquerque, New Mexico, United States, 87109
        • Ionis Investigative Site
    • North Carolina
      • Asheville, North Carolina, United States, 28803
        • Ionis Investigative Site
      • Hickory, North Carolina, United States, 28602
        • Ionis Investigative Site
      • Winston-Salem, North Carolina, United States, 27157
        • Ionis Investigative Site
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Ionis Investigative Site
      • Cleveland, Ohio, United States, 44130
        • Ionis Investigative Site
      • Cleveland, Ohio, United States, 44195
        • Ionis Investigative Site
    • Oklahoma
      • Tulsa, Oklahoma, United States, 74114
        • Ionis Investigative Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Ionis Investigative Site
      • Pittsburgh, Pennsylvania, United States, 15213
        • Ionis Investigative Site
      • West Mifflin, Pennsylvania, United States, 15122
        • Ionis Investigative Site
    • South Carolina
      • Greenville, South Carolina, United States, 29605
        • Ionis Investigative Site
      • Ladson, South Carolina, United States, 29456
        • Ionis Investigative Site
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Ionis Investigative Site
    • Texas
      • Abilene, Texas, United States, 79606
        • Ionis Investigative Site
      • Arlington, Texas, United States, 76012
        • Ionis Investigative Site
      • Austin, Texas, United States, 78705
        • Ionis Investigative Site
      • Bellaire, Texas, United States, 77401
        • Ionis Investigative Site
      • Dallas, Texas, United States, 75231
        • Ionis Investigative Site
      • McAllen, Texas, United States, 78503
        • Ionis Investigative Site
      • San Antonio, Texas, United States, 78240
        • Ionis Investigative Site
      • Southlake, Texas, United States, 76051
        • Ionis Investigative Site
      • The Woodlands, Texas, United States, 77384
        • Ionis Investigative Site
      • Willow Park, Texas, United States, 76087
        • Ionis Investigative Site
    • Utah
      • Salt Lake City, Utah, United States, 84107
        • Ionis Investigative Site
    • Virginia
      • Lynchburg, Virginia, United States, 24502
        • Ionis Investigative Site
      • Richmond, Virginia, United States, 23235
        • Ionis Investigative Site
    • Washington
      • Silverdale, Washington, United States, 98383
        • Ionis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

50 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: IONIS-FB-LRx
Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
Drug: IONIS-FB-LRx
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
Other Names:
  • ISIS 696844
Experimental: Placebo
Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
Drug: Placebo
Placebo matching solution, administered subcutaneously every 4 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
Time Frame: Week 49
Week 49

Secondary Outcome Measures

Outcome Measure
Time Frame
Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
Time Frame: Baseline and up to Week 49
Baseline and up to Week 49
Percentage Change from Baseline in Levels of Serum AH50 Activity
Time Frame: Baseline and up to Week 49
Baseline and up to Week 49
Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
Time Frame: Baseline and up to Week 49
Baseline and up to Week 49
Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)
Time Frame: Baseline and up to Week 57
Baseline and up to Week 57

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ionis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 4, 2019

Primary Completion (Actual)

April 18, 2024

Study Completion (Actual)

June 12, 2024

Study Registration Dates

First Submitted

January 22, 2019

First Submitted That Met QC Criteria

January 22, 2019

First Posted (Actual)

January 24, 2019

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

March 5, 2025

Last Verified

March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISIS 696844-CS5
  • 2020-005174-94 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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