Status:
RECRUITING
A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Lead Sponsor:
Akcea Therapeutics
Collaborating Sponsors:
United BioSource, LLC
Conditions:
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Eligibility:
All Genders
18+ years
Brief 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 th...
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...
Eligibility Criteria
Inclusion
- 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
Exclusion
- None
Key Trial Info
Start Date :
September 21 2021
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
March 31 2036
Estimated Enrollment :
240 Patients enrolled
Trial Details
Trial ID
NCT04850105
Start Date
September 21 2021
End Date
March 31 2036
Last Update
September 11 2025
Active Locations (26)
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1
Study Centre
Sofia, Bulgaria, 1431
2
Study Centre
Égkomi, Nicosia, Cyprus, 2371
3
Study Center
Lille, Cedex, France, 59037
4
Study Center
Nice, Romaine, France, 06001