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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
332

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 4, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 18, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 12, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 22, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 22, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 24, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 5, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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