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An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

An Open Label Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Brice Gary

Ionis Pharmaceuticals, Inc.

2855 Gazelle Court, Carlsbad, CA

ClinicalTrials@ionisph.com

Clinical trial contact

ICON Clinical Research GK

Kyutaromachi 4-chome 1-3, Chuo-ku, Osaka city, Osaka 541-0056, Japan

+81-6-4560-2001

Japan-Chiken@iconplc.com

Recruiting

June. 11, 2025

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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.

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.

Both

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Study drug: Eplontersen(ION-682884)
eplontersen SC once every month for up to 36 months(36 months OR 6 months after eplontersen is health authority-approved and available in the site's country,whichever occurs first).

Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) [Time Frame: Baseline up to 36 months]
Change From Baseline in Platelet Count [Time Frame: Baseline up to 36 months]
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) [Time Frame: Baseline up to 36 months]
Change From Baseline in Urine Protein Creatinine Ratio (UPCR) [Time Frame: Baseline up to 36 months]
Change From Baseline in AST [Time Frame: Baseline up to 36 months]
Change From Baseline in ALT [Time Frame: Baseline up to 36 months]
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters [Time Frame: Baseline up to 36 months]
Number of Participants With Clinically Significant Changes From Baseline in Thyroid-Stimulating Hormone (TSH) [Time Frame: Baseline up to 36 months]
Percentage of Participants With Anti-Drug Antibodies (ADA) [Time Frame: Baseline up to 36 months]

Change From Baseline in Transthyretin (TTR) Serum Levels [Time Frame: Baseline up to 36 months]
Change From Baseline in 6-minute Walk Test (6MWT) [Time Frame: Baseline up to 36 months]
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) [Time Frame: Baseline up to 36 months]
Change From Baseline in Short Form Health Survey Questionnaire (SF-36) [Time Frame: Baseline up to 36 months]
Change From Baseline in 5 Level EuroQol - 5 Dimension (EQ-5D-5L) [Time Frame: Baseline up to 36 months]
Change From Baseline in Biomarker: N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP) [Time Frame: Baseline up to 36 months]
Change From Baseline in Biomarker: High Sensitivity Cardiac Troponin T (hs-cTnT) [Time Frame: Baseline up to 36 months]

+81-92-771-8151

April. 25, 2024

Yes

Ionis may share anonymized individual participant data,aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met:(1)12 months from marketing approval of the study drug in both the United States and European Union; (2)18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website.

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