Clinical Trial

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

Active, Not Recruiting
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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.
Protocol Amendment History 2 changes
critical Enrollment closed, study ongoing 2026-07-30
notable Enrollment reduced: 240 -> 202 participants 2026-07-30
Trial Details
NCT Number NCT04850105
Lead Sponsor Akcea Therapeutics
Collaborators: United BioSource, LLC
Conditions Hereditary Transthyretin Amyloidosis With Polyneuropthy
Enrollment 202 participants
Start Date 2021-09-21
Primary Completion 2036-03-31 (estimated)
Study Completion 2036-03-31 (estimated)
Updated on ClinicalTrials.gov 2026-07-29