Actively Recruiting

Age: 18Years +
All Genders
NCT04850105

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

Led by Akcea Therapeutics · Updated on 2025-09-11

240

Participants Needed

26

Research Sites

757 weeks

Total Duration

On this page

Sponsors

A

Akcea Therapeutics

Lead Sponsor

U

United BioSource, LLC

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment OR patients diagnosed with hATTR-PN who have not taken TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per product label
  • Clinically managed in Canada, Europe, or the US
  • Have provided appropriate written informed consent
Not Eligible

You will not qualify if you...

History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety

AI-Screening

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Trial Site Locations

Total: 26 locations

1

Study Centre

Sofia, Bulgaria, 1431

Actively Recruiting

2

Study Centre

Égkomi, Nicosia, Cyprus, 2371

Actively Recruiting

3

Study Center

Lille, Cedex, France, 59037

Actively Recruiting

4

Study Center

Nice, Romaine, France, 06001

Actively Recruiting

5

Study Centre

Nantes, France, 44093

Actively Recruiting

6

Study Center

Heidelberg, Germany, 69120

Actively Recruiting

7

Study Center

Athens, Greece, 115 28

Actively Recruiting

8

Study Center

Athens, Greece, 11528

Actively Recruiting

9

Study Center

Heraklion, Greece, 71500

Actively Recruiting

10

Study Centre

Roma, Rome, Italy, 00189

Actively Recruiting

11

Study Center

Bologna, Italy, 40139

Actively Recruiting

12

Study Center

Genova, Italy, 16132

Actively Recruiting

13

Study Center

Messina, Italy, 98125

Actively Recruiting

14

Study Center

Milan, Italy, 20133

Actively Recruiting

15

Study Center

Naples, Italy, 80131

Actively Recruiting

16

Study Center

Pavia, Italy, 27100

Actively Recruiting

17

Study Center

Roma, Italy, 00133

Actively Recruiting

18

Study Center

Roma, Italy, 00168

Actively Recruiting

19

Study Center

Lisbon, Portugal, 1649 035

Actively Recruiting

20

Study Center

Huelva, Andalusia, Spain, 21005

Actively Recruiting

21

Study Centre

Oviedo, Avenida de Roma, Spain, 33011

Actively Recruiting

22

Study Center

Palma de Mallorca, Balearic Islands, Spain, 07198

Actively Recruiting

23

Study Centre

Villarreal, Barcelona, Spain, 08036

Actively Recruiting

24

Study Centre

Barcelona, Catalonia, Spain, 08035

Actively Recruiting

25

Study Centre

Madrid, Madrid, Spain, 28040

Actively Recruiting

26

Study Center

Madrid, Spain, 28041

Actively Recruiting

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Research Team

I

Ionis Pharmaceuticals

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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