Transthyretin Amyloid Cardiomyopathy Clinical Trial
A Non-interventional Cohort Safety Study of Patients With hATTR-PN
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.
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.
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.
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.
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
Clinically managed in Canada, Europe, or the US
Have provided appropriate written informed consent
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There are 37 Locations for this study
Anchorage Alaska, 99508, United States
Los Angeles California, 90033, United States
Los Angeles California, 90073, United States
Maywood Illinois, 60153, United States
Indianapolis Indiana, 46260, United States
Louisville Kentucky, 40202, United States
Rochester Minnesota, 55905, United States
New York New York, 10032, United States
Rosedale New York, 11422, United States
Cleveland Ohio, 44195, United States
Abington Pennsylvania, 19001, United States
Philadelphia Pennsylvania, 19107, United States
Dallas Texas, 75204, United States
Norfolk Virginia, 23507, United States
Toronto , ON M2, Canada
Lille Cedex, 59037, France
Nice Romaine, 06001, France
Saint-Herblain , 44093, France
Heidelberg , 69120, Germany
Athens , 115 2, Greece
Athens , 11528, Greece
Heraklion , 71500, Greece
Lagosanto Roma, 00168, Italy
Roma Rome, 00189, Italy
Bologna , 40139, Italy
Genova , 16132, Italy
Messina , 98125, Italy
Milano , 20133, Italy
Pavia , 27100, Italy
Roma , 00173, Italy
Huelva Andalucia, 21005, Spain
Oviedo Avenida De Roma, 33011, Spain
Villarreal Barcelona, 08036, Spain
Barcelona Cataluna, 08035, Spain
Madrid Comunidad De Madrid, 28040, Spain
Palma De Mallorca Islas Baleares, 07198, Spain
Madrid , 28041, Spain
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