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)
2 other identifiers
observational
240
8 countries
26
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 this study is to further characterize the long-term safety of TEGSEDI (inotersen) in patients with hATTR-PN under real-world conditions.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2021
Longer than P75 for all trials
26 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 6, 2021
CompletedFirst Posted
Study publicly available on registry
April 20, 2021
CompletedStudy Start
First participant enrolled
September 21, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2036
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2036
September 11, 2025
September 1, 2025
14.5 years
April 6, 2021
September 5, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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)
10 years
Secondary Outcomes (2)
Description of the incidence rate of the Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
10 years
Description of the time to onset of Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
10 years
Study Arms (2)
TEGSEDI-exposed cohort
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.
TEGSEDI-unexposed cohort
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.
Interventions
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.
Eligibility Criteria
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.
You may qualify if:
- 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
You may not qualify if:
- None
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Akcea Therapeuticslead
- United BioSource, LLCcollaborator
Study Sites (26)
Study Centre
Sofia, 1431, Bulgaria
Study Centre
Égkomi, Nicosia, 2371, Cyprus
Study Center
Lille, Cedex, 59037, France
Study Center
Nice, Romaine, 06001, France
Study Centre
Nantes, 44093, France
Study Center
Heidelberg, 69120, Germany
Study Center
Athens, 115 28, Greece
Study Center
Athens, 11528, Greece
Study Center
Heraklion, 71500, Greece
Study Centre
Roma, Rome, 00189, Italy
Study Center
Bologna, 40139, Italy
Study Center
Genova, 16132, Italy
Study Center
Messina, 98125, Italy
Study Center
Milan, 20133, Italy
Study Center
Napoli, 80131, Italy
Study Center
Pavia, 27100, Italy
Study Center
Roma, 00133, Italy
Study Center
Roma, 00168, Italy
Study Center
Lisbon, 1649 035, Portugal
Study Center
Huelva, Andalusia, 21005, Spain
Study Centre
Oviedo, Avenida de Roma, 33011, Spain
Study Center
Palma de Mallorca, Balearic Islands, 07198, Spain
Study Centre
Villarreal, Barcelona, 08036, Spain
Study Centre
Barcelona, Catalonia, 08035, Spain
Study Centre
Madrid, Madrid, 28040, Spain
Study Center
Madrid, 28041, Spain
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 6, 2021
First Posted
April 20, 2021
Study Start
September 21, 2021
Primary Completion (Estimated)
March 31, 2036
Study Completion (Estimated)
March 31, 2036
Last Updated
September 11, 2025
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share