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Long-term safety assessment of TEGSEDI in patients with hereditary transthyretin amyloidosis with polyneuropathy

A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (hATTR-PN)

Observational Akcea Therapeutics · NCT04850105

This study is testing the long-term safety of the drug TEGSEDI in people with hereditary transthyretin amyloidosis and nerve damage to see how it works in everyday life.

Quick facts

Study typeObservational
Enrollment240 (estimated)
Ages18 Years and up
SexAll
SponsorAkcea Therapeutics Industry-sponsored
Locations26 sites (Sofia and 25 other locations)
Trial IDNCT04850105 on ClinicalTrials.gov

What this trial studies

This is a prospective, non-interventional cohort safety study focusing on patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The study aims to evaluate the long-term safety of TEGSEDI (inotersen) under real-world conditions across multiple countries, including the US, Canada, and various European nations. Patients will be categorized into two cohorts: those who have been exposed to TEGSEDI and those who have not but are eligible for treatment. The study will collect data from participating centers that manage hATTR-PN patients to better understand the safety profile of the treatment over time.

Who should consider this trial

Good fit: Ideal candidates include adults diagnosed with hATTR-PN who have either been treated with TEGSEDI or are eligible for treatment but have not yet received it.

Not a fit: Patients who do not have a diagnosis of hATTR-PN or those who are not eligible for TEGSEDI treatment will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety of TEGSEDI, potentially improving treatment strategies for patients with hATTR-PN.

How similar studies have performed: While this study focuses on long-term safety, previous studies have shown efficacy for TEGSEDI in treating hATTR-PN, indicating a foundation for further investigation.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Either:

   1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment
   2. TEGSEDI Unexposed Cohort: Patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label. Patients may take other drugs to treat hATTR-PN.
2. Clinically managed in Canada, Europe, or the US
3. Have provided appropriate written informed consent

Exclusion Criteria:

* None

Where this trial is running

Sofia and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hereditary Transthyretin Amyloidosis With PolyneuropthyHereditary Transthyretin AmyloidosishATTR-PNhATTRAmyloidosis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.