Clinical Trial
Study Details
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.
Outcomes
* 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)
To describe the incidence rate of the following Adverse Events of Special Interest (AESI):
* severe thrombocytopenia
* serious and non-serious bleeding events
* glomerulonephritis
* composite of stroke and/or cervicocephalic arterial dissection
* central nervous system (CNS) vasculitis
* ocular toxicity due to vitamin A deficiency
To describe the time to onset of the following Adverse Events of Special Interest (AESI):
* severe thrombocytopenia
* serious and non-serious bleeding events
* glomerulonephritis
* composite of stroke and/or cervicocephalic arterial dissection
* central nervous system (CNS) vasculitis
* ocular toxicity due to vitamin A deficiency
Study Overview
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.
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, gastrointestinal tract, and other organs. hATTR-PN is a rare disease and there are no large epidemiological studies that reliably provide an indication of its prevalence. The worldwide distribution is unequal, with higher rates in Portugal, Japan, Brazil, Northern Sweden, and the US. Current estimates suggest there may be 10,000 afflicted patients worldwide.
TEGSEDI (inotersen) is an antisense oligonucleotide inhibitor of human TTR protein synthesis. In Europe and Canada, TEGSEDI is indicated for the treatment of Stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In the US, TEGSEDI is indicated for treatment of the polyneuropathy of hereditary TTR-mediated amyloidosis in adults. Efficacy has been demonstrated in patients with hATTR-PN, as reflected by a slowing or reversal of disease progression.
Research Question:
The overarching goal of this study is to further characterize the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.
Population:
Patients in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN. Physicians participating in the study will be instructed to invite all patients who meet study eligibility criteria to enroll until the enrollment period is closed.
Contacts and Locations
Study Center
Anchorage, AK 99508
United States
Study Center
Los Angeles, CA 90033
United States
Study Center
Los Angeles, CA 90073
United States
Study Center
Maywood, IL 60153
United States
Study Center
Indianapolis, IN 46260
United States
Study Centre
Louisville, KY 40202
United States
Study Center
Rochester, MN 55905
United States
Study Center
New York, NY 10032
United States
Study Center
Rosedale, NY 11422
United States
Study Center
Cleveland, OH 44195
United States
Study Center
Abington, PA 19001
United States
Study Center
Philadelphia, PA 19107
United States
Study Center
Dallas, TX 75204
United States
Study Center
Norfolk, VA 23507
United States
Study Center
Toronto ON M2K 0A6
Canada
Study Center
59037 Lille
France
Study Center
06001 Nice
France
Study Centre
44093 Saint-Herblain
France
Study Center
69120 Heidelberg
Germany
Study Center
115 28 Athens
Greece
Study Center
11528 Athens
Greece
Study Center
71500 Heraklion
Greece
Study Center
00168 Lagosanto RM
Italy
Study Centre
00189 Roma
Italy
Study Center
40139 Bologna
Italy
Study Center
16132 Genova
Italy
Study Center
98125 Messina
Italy
Study Center
20133 Milano
Italy
Study Center
80131 Napoli
Italy
Study Center
27100 Pavia
Italy
Study Center
00173 Roma
Italy
Study Center
1649 035 Lisboa
Portugal
Study Center
21005 Huelva
Spain
Study Centre
33011 Oviedo
Spain
Study Centre
08036 Villarreal Barcelona
Spain
Study Centre
08035 Barcelona
Spain
Study Centre
28040 Madrid
Spain
Study Center
07198 Palma De Mallorca
Spain
Study Center
28041 Madrid
Spain
Participation Criteria
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
2. Clinically managed in Canada, Europe, or the US
3. Have provided appropriate written informed consent
Exclusion Criteria:
* None
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.