NCT04850105

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 240 (estimated)
• Sponsor: Akcea Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, non-interventional, Long-term, multinational cohort safety study of patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN). The overarching goal of this study is to further characterize the long-term safety of TEGSEDI (inotersen) in patients with hATTR-PN under real-world conditions.

Detailed description

Study Rationale: hATTR-PN is an inherited, progressive, fatal disease caused by misfolded transthyretin (TTR) proteins that accumulate as amyloid fibrils predominantly in the peripheral nerves, heart, gastrointestinal tract, and other organs. hATTR-PN is a rare disease and there are no large epidemiological studies that reliably provide an indication of its prevalence. The worldwide distribution is unequal, with higher rates in Portugal, Japan, Northern Sweden, and the US. Current estimates suggest there may be 10,000 afflicted patients worldwide. TEGSEDI (inotersen) is an antisense oligonucleotide inhibitor of human TTR protein synthesis. In Europe and Canada, TEGSEDI is indicated for the treatment of Stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In the US, TEGSEDI is indicated for treatment of the polyneuropathy of hereditary TTR-mediated amyloidosis in adults. Efficacy has been demonstrated in patients with hATTR-PN, as reflected by a slowing or reversal of disease progression. Research Question: The overarching goal of this study is to further characterize the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions. Population: Patients in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN. Physicians participating in the study will be instructed to invite all patients who meet study eligibility criteria to enroll until the enrollment period is closed.

Conditions

• Hereditary Transthyretin Amyloidosis With Polyneuropthy

Interventions

Type	Name	Description
OTHER	Data Collection	Data on each patient will be collected at study enrollment and at each follow-up visit. No mandatory visits, tests, or assessments are required for this study. All visits will be scheduled and conducted according to the clinical site's normal clinical practice.

Timeline

• Start date: 2021-09-21
• Primary completion: 2036-03-31
• Completion: 2036-03-31
• First posted: 2021-04-20
• Last updated: 2025-09-11

Locations

26 sites across 8 countries: Bulgaria, Cyprus, France, Germany, Greece, Italy, Portugal, Spain