Enrolling by invitation NCT05667493

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

Phase III Interventional Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: Eplontersen.
Who it may be relevant to: Registry conditions: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM). Basic parameters: from 18 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: United States, Argentina, Australia, Austria, Belgium +15
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official 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)

Overview

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Detailed description

This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until after eplontersen is approved and available in the site's country, whichever occurs first. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A.

Interventions

Drug Eplontersen
Eplontersen will be administered by SC injection.

Primary outcome measures

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]

Secondary outcome measures (3)

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]

Eligibility criteria

Inclusion criteria

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.
Investigator is willing to treat the participant with open-label eplontersen.
Willingness to adhere to vitamin A supplementation per protocol.

Exclusion criteria

Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation: N/A
Model: Single group
Masking: Open label
Primary purpose: Treatment