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Macular DegenerationPhase 2NCT03815825

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)

A Phase 2 study testing IONIS-FB-LRx in people with Macular Degeneration and Geographic Atrophy, run by Ionis Pharmaceuticals, Inc. at 88 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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). Read more about this study

Who is it for?
Ages 50 years and older
Conditions the study is for
Macular Degeneration, Geographic Atrophy
What is being tested?
IONIS-FB-LRx
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
332 participants
Where
88 sitesin 9 countries
Study dates
4 Mar 2019to 12 Jun 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

88 sites in 9 countries.

88 sites in 9 countries

United States55 sites

  • IONIS Investigative Site

    Abilene, Texas, 79606

  • IONIS Investigative Site

    Albuquerque, New Mexico, 87109

  • IONIS Investigative Site

    Arlington, Texas, 76012

  • IONIS Investigative Site

    Asheville, North Carolina, 28803

  • IONIS Investigative Site

    Augusta, Georgia, 30909

  • IONIS Investigative Site

    Austin, Texas, 78705

  • IONIS Investigative Site

    Baltimore, Maryland, 21204

  • IONIS Investigative Site

    Baltimore, Maryland, 21209

  • IONIS Investigative Site

    Bellaire, Texas, 77401

  • IONIS Investigative Site

    Beverly Hills, California, 90211

  • IONIS Investigative Site

    Clearwater, Florida, 33761

  • IONIS Investigative Site

    Cleveland, Ohio, 44106

  • IONIS Investigative Site

    Cleveland, Ohio, 44130

  • IONIS Investigative Site

    Cleveland, Ohio, 44195

  • IONIS Investigative Site

    Colorado Springs, Colorado, 80909

  • IONIS Investigative Site

    Dallas, Texas, 75231

  • IONIS Investigative Site

    Fort Myers, Florida, 33907

  • IONIS Investigative Site

    Greenville, South Carolina, 29605

  • IONIS Investigative Site

    Hagerstown, Maryland, 21740

  • IONIS Investigative Site

    Hickory, North Carolina, 28602

  • IONIS Investigative Site

    Huntington Beach, California, 92697

  • IONIS Investigative Site

    La Jolla, California, 92093

  • IONIS Investigative Site

    Ladson, South Carolina, 29456

  • IONIS Investigative Site

    Lake Worth, Florida, 33461

  • IONIS Investigative Site

    Lakeland, Florida, 33801

  • IONIS Investigative Site

    Leawood, Kansas, 66211

  • IONIS Investigative Site

    Lemont, Illinois, 60439

  • IONIS Investigative Site

    Long Beach, California, 90807

  • IONIS Investigative Site

    Lynchburg, Virginia, 24502

  • IONIS Investigative Site

    McAllen, Texas, 78503

  • IONIS Investigative Site

    Melbourne, Florida, 32901

  • IONIS Investigative Site

    Nashville, Tennessee, 37203

  • IONIS Investigative Site

    Philadelphia, Pennsylvania, 19104

  • IONIS Investigative Site

    Phoenix, Arizona, 85020

  • IONIS Investigative Site

    Phoenix, Arizona, 85021

  • IONIS Investigative Site

    Phoenix, Arizona, 85053

  • IONIS Investigative Site

    Pittsburgh, Pennsylvania, 15213

  • IONIS Investigative Site

    Poway, California, 92064

  • IONIS Investigative Site

    Richmond, Virginia, 23235

  • IONIS Investigative Site

    Salt Lake City, Utah, 84107

  • IONIS Investigative Site

    San Antonio, Texas, 78240

  • IONIS Investigative Site

    San Francisco, California, 94109

  • IONIS Investigative Site

    Silverdale, Washington, 98383

  • IONIS Investigative Site

    Southaven, Mississippi, 38671

  • IONIS Investigative Site

    Southlake, Texas, 76051

  • IONIS Investigative Site

    Springfield, Illinois, 62702

  • IONIS Investigative Site

    Stuart, Florida, 34994

  • IONIS Investigative Site

    Sun City, Arizona, 85351

  • IONIS Investigative Site

    Tampa, Florida, 33612

  • IONIS Investigative Site

    The Woodlands, Texas, 77384

  • IONIS Investigative Site

    Tulsa, Oklahoma, 74114

  • IONIS Investigative Site

    West Mifflin, Pennsylvania, 15122

  • IONIS Investigative Site

    Willow Park, Texas, 76087

  • IONIS Investigative Site

    Winston-Salem, North Carolina, 27157

  • IONIS Investigative Site

    Winter Haven, Florida, 33880

Other trials in United States

Australia11 sites

  • IONIS Investigative Site

    Adelaide, South Australia, 5000

  • IONIS Investigative Site

    Albury, New South Wales, 2640

  • IONIS Investigative Site

    East Melbourne, Victoria, 3002

  • IONIS Investigative Site

    Essendon, Victoria, 3040

  • IONIS Investigative Site

    Glen Iris, Victoria, 3146

  • IONIS Investigative Site

    Hurstville, New South Wales, 2228

  • IONIS Investigative Site

    Malvern, Victoria, 3144

  • IONIS Investigative Site

    Parkville, Victoria, 3050

  • IONIS Investigative Site

    Parramatta, New South Wales, 2150

  • IONIS Investigative Site

    Perth, Western Australia, 6009

  • IONIS Investigative Site

    Sydney, New South Wales, 2000

Other trials in Australia

Spain8 sites

  • IONIS Investigative Site

    Barcelona, 8022

  • IONIS Investigative Site

    Barcelona, 8034

  • IONIS Investigative Site

    Burjassot, Valencia, 46100

  • IONIS Investigative Site

    Majadahonda, Madrid, 28222

  • IONIS Investigative Site

    Pamplona, Navarre, 31008

  • IONIS Investigative Site

    Terrassa, Barcelona, 8227

  • IONIS Investigative Site

    Valladolid, 47012

  • IONIS Investigative Site

    Zaragoza, 50009

Other trials in Spain

Austria4 sites

  • IONIS Investigative Site

    Klagenfurt, 9020

  • IONIS Investigative Site

    Linz, Upper Austria, 4020

  • IONIS Investigative Site

    Vienna, 1090

  • IONIS Investigative Site

    Vienna, 1140

Other trials in Austria

Canada4 sites

  • IONIS Investigative Site

    London, Ontario, N6A 4V2

  • IONIS Investigative Site

    Ottawa, Ontario, K1H 8L6

  • IONIS Investigative Site

    Ottawa, Ontario, K2B 7E9

  • IONIS Investigative Site

    Québec, Quebec, G1S 4L8

Other trials in Canada

Netherlands2 sites

  • IONIS Investigative Site

    Nijmegen, Gelderland, 6525EX

  • IONIS Investigative Site

    Rotterdam, 3011 BH

Other trials in Netherlands

Poland2 sites

  • IONIS Investigative Site

    Bydgoszcz, 85-631

  • IONIS Investigative Site

    Katowice, Silesian, 40-514

Other trials in Poland

Czechia1 site

  • IONIS Investigative Site

    Prague, 15000

Other trials in Czechia

New Zealand1 site

  • IONIS Investigative Site

    Christchurch, 8013

Other trials in New Zealand

About this study

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)

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).

Read the 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).

Being tested
IONIS-FB-LRx, Placebo
Medical subject headings
Macular Degeneration, Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • IONIS-FB-LRxDrug

    Also called ISIS 696844

    IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks

  • PlaceboDrug

    Placebo matching solution, administered subcutaneously every 4 weeks

Study groups in NCT03815825
Study groupRoleWhat they receive
IONIS-FB-LRxExperimentalStage 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.
Receives: Drug: IONIS-FB-LRx
PlaceboExperimentalStage 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.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging

    Measured: Week 49

Secondary outcomes (4)

  1. Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)

    Measured: Baseline and up to Week 57

  2. Percentage Change from Baseline in Levels of Factor B (FB) in Plasma

    Measured: Baseline and up to Week 49

  3. Percentage Change from Baseline in Levels of Serum AH50 Activity

    Measured: Baseline and up to Week 49

  4. Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)

    Measured: Baseline and up to Week 49

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jan 2019
  2. Study start4 Mar 2019
  3. Primary completion18 Apr 2024
  4. Study completion12 Jun 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
332 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03815825
Sponsor's protocol ID
ISIS 696844-CS5
Other IDs
2020-005174-94
Status last verified
Mar 2025
First submitted
22 Jan 2019
Last updated
7 Mar 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

    PubMed 37314061

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing IONIS-FB-LRx in people with Macular Degeneration and Geographic Atrophy, run by Ionis Pharmaceuticals, Inc. at 88 sites in 9 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 88 sites in United States, Australia, Spain, Austria, Canada and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging (time frame: Week 49). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03815825, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2025.

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