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A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Sponsor: Ionis Pharmaceuticals, Inc.
Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Official title: An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
151
Start Date
2022-01-04
Completion Date
2029-08
Last Updated
2025-12-04
Healthy Volunteers
No
Interventions
Eplontersen
Eplontersen will be administered by SC injection.
Locations (33)
Mayo Clinic
Scottsdale, Arizona, United States
Indiana University Health University Hospital
Indianapolis, Indiana, United States
Johns Hopkins University Neurology Research Office
Baltimore, Maryland, United States
Boston University School of Medicine
Boston, Massachusetts, United States
The Neurological Institute of New York
New York, New York, United States
University of North Carolina Hospitals - Neurology Clinic
Chapel Hill, North Carolina, United States
Oregon Health and Science University
Portland, Oregon, United States
University of Washington Medical Center
Seattle, Washington, United States
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
Instituto Fleni
Buenos Aires, Argentina
Hospital El Cruce
San Juan Bautista, Argentina
Perron Institute for Neurological and Translational Science
Murdoch, Western Australia, Australia
Hospital Universitario Clementino Fraga Filho
Botafogo, Rio de Janeiro, Brazil
Universidade Estadual de Campinas
Campinas, Brazil
Instituto de Neurologia de Curitiba
Curitiba, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
São Paulo, Brazil
Vancouver General Hospital
Vancouver, British Columbia, Canada
Toronto General Hospital
Toronto, Ontario, Canada
The Cyprus Institute of Neurology and Genetics
Égkomi, Cyprus
Centre Hospitalier Universitaire de Toulouse
Toulouse, Haute-Garonne, France
Hôpital de la Timone
Marseille, France
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta
Milan, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
Lisbon, Portugal
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
Porto, Portugal
Hospital Clínico San Carlos
Madrid, Spain
Hospital Son Llàtzer
Palma de Mallorca, Spain
Norrlands Universitetssjukhus
Umeå, Sweden
China Medical University Hospital
Taichung, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan
Chang Gung Memorial Hospital
Taoyuan District, Taiwan
Istanbul Üniversitesi - Istanbul Tip Fakültesi
Istanbul, Turkey (Türkiye)