NCT04850105Active, not recruiting
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)
Source: ClinicalTrials.gov · Last updated 29 Jul 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 202 (actual)
- First submitted
- 6 Apr 2021
- Start date
- 21 Sept 2021
- Primary completion date
- 31 Mar 2036
- Lead sponsor
- Akcea Therapeutics
- Diseases
- Amyloidosis
- NCT number
- NCT04850105
What you commit to
- Study type: Observational
- Participants enrolled: 202
- From 21 Sept 2021 to the primary completion date, 31 Mar 2036
- 24 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- None
Who the study is looking for
- Either:
- TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment
- 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.
- Clinically managed in Canada, Europe, or the US
- Have provided appropriate written informed consent
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (24)
Sites in registry order. Pick your city on the disease page to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.