An observational study in Hereditary Transthyretin Amyloidosis With Polyneuropthy, sponsored by Akcea Therapeutics. Active, not recruiting at 26 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-29.
Sponsored by Akcea Therapeutics · Observational
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.
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.
The TEGSEDI-exposed cohort will consist of patients diagnosed with hATTR-PN who are receiving TEGSEDI. Data from the TEGSEDI-exposed cohort will be compared to data collected prospectively from a TEGSEDI-unexposed cohort which will consist of 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 in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN.
Either:
Exclusion Criteria:
This cohort consist of patients diagnosed with hATTR-PN who are receiving any dose of commercial TEGSEDI and who have provided written informed consent to be included into the study.
Other: Data Collection
This cohort which will consist of 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 and who have provided written informed consent to be included into the study.
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.
Further characterization of the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.
* Determination of the incidence rate of thrombocytopenia in patients with hATTR-PN treated with TEGSEDI (TEGSEDI-exposed cohort) * Comparison of the relative rates of thrombocytopenia in hATTR-PN patients treated with TEGSEDI (TEGSEDI exposed) to hATTR-PN patients unexposed to TEGSEDI (TEGSEDI- unexposed)
Time frame: 10 years
Description of the incidence rate of the Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
To describe the incidence rate of the following Adverse Events of Special Interest (AESI): * severe thrombocytopenia (platelet counts \<25 x 109/L and separately, \<50 x 109/L) * serious and non-serious bleeding events * glomerulonephritis * hepatotoxicity/serious hepatobiliary events * composite of stroke and/or cervicocephalic arterial dissection * central nervous system (CNS) vasculitis * ocular toxicity due to vitamin A deficiency
Time frame: 10 years
Description of the time to onset of Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
To describe the time to onset of the following Adverse Events of Special Interest (AESI): * severe thrombocytopenia * serious and non-serious bleeding events * glomerulonephritis * hepatotoxicity/serious hepatobiliary events * composite of stroke and/or cervicocephalic arterial dissection * central nervous system (CNS) vasculitis * ocular toxicity due to vitamin A deficiency
Time frame: 10 years
Plan to share: No
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This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Amyloidosis, Hereditary, Transthyretin-Related→
Akcea Therapeutics