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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03815825




Registration number
NCT03815825
Ethics application status
Date submitted
22/01/2019
Date registered
24/01/2019

Titles & IDs
Public title
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)
Scientific 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)
Secondary ID [1] 0 0
2020-005174-94
Secondary ID [2] 0 0
ISIS 696844-CS5
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Macular Degeneration 0 0
Geographic Atrophy 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - IONIS-FB-LRx
Treatment: Drugs - Placebo

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.

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.


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

Treatment: Drugs: Placebo
Placebo matching solution, administered subcutaneously every 4 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
Timepoint [1] 0 0
Week 49
Secondary outcome [1] 0 0
Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)
Timepoint [1] 0 0
Baseline and up to Week 57
Secondary outcome [2] 0 0
Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
Timepoint [2] 0 0
Baseline and up to Week 49
Secondary outcome [3] 0 0
Percentage Change from Baseline in Levels of Serum AH50 Activity
Timepoint [3] 0 0
Baseline and up to Week 49
Secondary outcome [4] 0 0
Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
Timepoint [4] 0 0
Baseline and up to Week 49

Eligibility
Key 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
Minimum age
50 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/06/2024
Sample size
Target
Accrual to date
Final
332
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
Recruitment hospital [1] 0 0
IONIS Investigative Site - Albury
Recruitment hospital [2] 0 0
IONIS Investigative Site - Hurstville
Recruitment hospital [3] 0 0
IONIS Investigative Site - Parramatta
Recruitment hospital [4] 0 0
IONIS Investigative Site - Sydney
Recruitment hospital [5] 0 0
IONIS Investigative Site - Adelaide
Recruitment hospital [6] 0 0
IONIS Investigative Site - East Melbourne
Recruitment hospital [7] 0 0
IONIS Investigative Site - Essendon
Recruitment hospital [8] 0 0
IONIS Investigative Site - Glen Iris
Recruitment hospital [9] 0 0
IONIS Investigative Site - Malvern
Recruitment hospital [10] 0 0
IONIS Investigative Site - Parkville
Recruitment hospital [11] 0 0
IONIS Investigative Site - Perth
Recruitment postcode(s) [1] 0 0
2640 - Albury
Recruitment postcode(s) [2] 0 0
2228 - Hurstville
Recruitment postcode(s) [3] 0 0
2150 - Parramatta
Recruitment postcode(s) [4] 0 0
2000 - Sydney
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
3002 - East Melbourne
Recruitment postcode(s) [7] 0 0
3040 - Essendon
Recruitment postcode(s) [8] 0 0
3146 - Glen Iris
Recruitment postcode(s) [9] 0 0
3144 - Malvern
Recruitment postcode(s) [10] 0 0
3050 - Parkville
Recruitment postcode(s) [11] 0 0
6009 - Perth
Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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California
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Colorado
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Florida
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United States of America
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Georgia
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United States of America
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Illinois
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Kansas
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United States of America
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Maryland
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United States of America
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Mississippi
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New Mexico
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United States of America
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North Carolina
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Ohio
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Oklahoma
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Pennsylvania
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South Carolina
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Tennessee
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Texas
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United States of America
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Utah
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United States of America
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Virginia
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United States of America
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Washington
Country [21] 0 0
Austria
State/province [21] 0 0
Oberösterreich
Country [22] 0 0
Austria
State/province [22] 0 0
Klagenfurt
Country [23] 0 0
Austria
State/province [23] 0 0
Vienna
Country [24] 0 0
Canada
State/province [24] 0 0
Ontario
Country [25] 0 0
Canada
State/province [25] 0 0
Quebec
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Czechia
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Prague
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Netherlands
State/province [27] 0 0
Gelderland
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Netherlands
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Rotterdam
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New Zealand
State/province [29] 0 0
Christchurch
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Poland
State/province [30] 0 0
Silesian
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Poland
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Bydgoszcz
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Navarra
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Spain
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Valencia
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Spain
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Valladolid
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Spain
State/province [37] 0 0
Zaragoza

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03815825