RECRUITINGOBSERVATIONAL

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)

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Either: 1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment 2. 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. 2. Clinically managed in Canada, Europe, or the US 3. Have provided appropriate written willing to sign a consent form Who Should NOT Join This Trial: - None Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: 1. Either: 1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment 2. 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. 2. Clinically managed in Canada, Europe, or the US 3. Have provided appropriate written informed consent Exclusion Criteria: * None

Treatments Being Tested

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.

Locations (20)

Study Centre

Sofia, Bulgaria

Study Centre

Égkomi, Nicosia, Cyprus

Study Center

Lille, Cedex, France

Study Center

Nice, Romaine, France

Study Centre

Nantes, France

Study Center

Heidelberg, Germany

Study Center

Athens, Greece

Study Center

Athens, Greece

Study Center

Heraklion, Greece

Study Centre

Roma, Rome, Italy

Study Center

Bologna, Italy

Study Center

Genova, Italy

Study Center

Messina, Italy

Study Center

Milan, Italy

Study Center

Naples, Italy

Study Center

Pavia, Italy

Study Center

Roma, Italy

Study Center

Roma, Italy

Study Center

Lisbon, Portugal

Study Center

Huelva, Andalusia, Spain