Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an observational cohort study designed to describe the long-term safety of initiating eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplantation or severe hepatic impairment. It will characterize baseline demographics and clinical features, including transplant history and hepatic status, and track safety outcomes such as new clinical events and abnormal lab results over time. Data come from participants in a related observational study (D8450R00003) with consent for secondary research use, plus comparisons to patients who received alternative ATTR therapies. Interim analyses are planned annually from 2027 to 2031, with a final report in 2033. Cohort Event Monitoring will be used to identify important safety signals, with cumulative hazard estimates if events exceed ten for a given outcome.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. D8450R00003 participants who consented to have their data used for future related research studies.
2. D8450R00003 participants who initiated eplontersen treatment up to 1-year prior to enrolment into D8450R00003 study observation period, irrespective of ATTR phenotype or genotype. For the comparative analyses, patients unexposed to eplontersen treatment and who initiated another ATTR treatment during D8450R00003 study observation period will be included

Exclusion Criteria:

1. Patients with exposure to eplontersen more than 1-year prior to enrolment into D8450R00003 study.
2. Patients who participated in an interventional ATTR study in the 12-months prior to enrolment into D8450R00003 study.

Where it's running

• Research Site
  Los Angeles, California, United States Recruiting
• Research Site
  Vancouver, British Columbia, Canada Recruiting
• Research Site
  Beijing, China Recruiting
• Research Site
  Hanover, Germany Recruiting
• Research Site
  Majadahonda, Spain Recruiting
• Research Site
  Birmingham, United Kingdom Recruiting

Study contacts

• Study coordinator: AstraZeneca Clinical Study Information Center
• Email: information.center@astrazeneca.com
• Phone: 1-877-240-9479

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Eplontersen exposure during pregnancy and breastfeeding: outcomes

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• Nucresiran for transthyretin amyloidosis with cardiomyopathy

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• Preventing transthyretin amyloidosis in young carriers of a genetic variant

  This study is testing if a medication called acoramidis can help prevent transthyretin amyloidosis in young adults who carry a specific genetic variant that puts them at risk for the disease.

  Recruiting at 104 sites Phase 3 Eidos Therapeutics, a BridgeBio company · La Jolla, California and 103 other locations · NCT06563895

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.