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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05667493
Registration number
NCT05667493
Ethics application status
Date submitted
12/07/2022
Date registered
28/12/2022
Date last updated
28/03/2024
Titles & IDs
Public title
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
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Scientific title
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
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Secondary ID [1]
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2022-000826-74
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Secondary ID [2]
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ION-682884-CS12
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
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Condition category
Condition code
Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Eplontersen
Experimental: Eplontersen - Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.
Treatment: Drugs: Eplontersen
Eplontersen will be administered by SC injection.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
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Assessment method [1]
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Timepoint [1]
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Baseline up to 36 months
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Primary outcome [2]
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Change From Baseline in Platelet Count
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Assessment method [2]
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Timepoint [2]
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Baseline up to 36 months
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Primary outcome [3]
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Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
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Assessment method [3]
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Timepoint [3]
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Baseline up to 36 months
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Primary outcome [4]
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Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
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Assessment method [4]
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Timepoint [4]
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Baseline up to 36 months
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Primary outcome [5]
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Change From Baseline in AST
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Assessment method [5]
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Timepoint [5]
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Baseline up to 36 months
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Primary outcome [6]
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Change From Baseline in ALT
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Assessment method [6]
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Timepoint [6]
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Baseline up to 36 months
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Primary outcome [7]
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Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
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Assessment method [7]
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Timepoint [7]
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Baseline up to 36 months
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Primary outcome [8]
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Number of Participants With Clinically Significant Changes From Baseline in Thyroid-Stimulating Hormone (TSH)
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Assessment method [8]
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Timepoint [8]
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Baseline up to 36 months
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Primary outcome [9]
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Percentage of Participants With Anti-Drug Antibodies (ADA)
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Assessment method [9]
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Timepoint [9]
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Baseline up to 36 months
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Secondary outcome [1]
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Change From Baseline in Transthyretin (TTR) Serum Levels
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Assessment method [1]
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Timepoint [1]
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Baseline up to 36 months
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Secondary outcome [2]
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Change From Baseline in 6-minute Walk Test (6MWT)
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Assessment method [2]
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Timepoint [2]
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Baseline up to 36 months
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Secondary outcome [3]
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Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
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Assessment method [3]
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Timepoint [3]
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Baseline up to 36 months
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Secondary outcome [4]
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Change From Baseline in Short Form Health Survey Questionnaire (SF-36)
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Assessment method [4]
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Timepoint [4]
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Baseline up to 36 months
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Secondary outcome [5]
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Change From Baseline in 5 Level EuroQol - 5 Dimension (EQ-5D-5L)
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Assessment method [5]
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Timepoint [5]
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Baseline up to 36 months
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Secondary outcome [6]
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Change From Baseline in Biomarker: N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
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Assessment method [6]
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Timepoint [6]
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Baseline up to 36 months
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Secondary outcome [7]
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Change From Baseline in Biomarker: High Sensitivity Cardiac Troponin T (hs-cTnT)
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Assessment method [7]
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Timepoint [7]
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Baseline up to 36 months
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Eligibility
Key inclusion criteria
1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the
Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on
ISIS 420915- CS101 study as judged by the Investigator and Sponsor.
2. Investigator is willing to treat the participant with open-label eplontersen.
3. Willingness to adhere to vitamin A supplementation per protocol.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Permanently discontinued study drug administration while participating in the Index
Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
2. Have any new condition or worsening of an existing condition that in the opinion of
the Investigator or Sponsor would make the participant unsuitable for enrolment, or
which could interfere with the participant participating in or completing the study,
including the need for treatment with medications disallowed in the Index Study.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/11/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/08/2029
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Actual
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Sample size
Target
1400
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
West AustraliaWA
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Recruitment hospital [1]
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Advara HeartCare Murdoch - Murdoch
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Recruitment hospital [2]
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Advara HeartCare - Joondalup
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Recruitment hospital [3]
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Liverpool Hospital - Liverpool
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Recruitment postcode(s) [1]
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6150 - Murdoch
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Recruitment postcode(s) [2]
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6027 - Joondalup
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Recruitment postcode(s) [3]
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2170 - Liverpool
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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District of Columbia
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Florida
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Georgia
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Missouri
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Washington
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Austria
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Graz
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Austria
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Innsbruck
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Brugge
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Alberta
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Ontario
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La Roche-sur-Yon
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Nantes
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France
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Pessac
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Germany
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Homburg
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Germany
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Münster
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Würzburg
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Athens
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Haifa
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Israel
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Rehovot
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Italy
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Firenze
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Italy
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Pavia
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Portugal
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Guimarães
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Porto
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Barcelona
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Majadahonda
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Spain
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Murcia
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Göteborg
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Sweden
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Skellefteå
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United Kingdom
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State/province [48]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Ionis Pharmaceuticals, Inc.
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Address
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Country
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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AstraZeneca
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with
eplontersen in participants with ATTR-CM.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05667493
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Phone
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Email
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Contact person for public queries
Name
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Ionis Pharmaceuticals
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Address
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Country
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Phone
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(844) 520-3239
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05667493
Download to PDF