New drug shows promise for Long-Term control of rare nerve disease

NCT ID NCT05071300

Ongoing Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jan 04, 2026 · Last updated May 15, 2026 · Updated 23 times

Summary

This study looks at the long-term safety of a drug called eplontersen in people with a rare inherited nerve disease (hATTR-PN). About 151 participants who completed earlier studies will receive the drug for an extended period. The main goal is to check for side effects and monitor overall health, while also seeing if the drug helps control nerve damage over time.

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Contacts and locations

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Locations

Boston University School of Medicine

Boston, Massachusetts, 02118, United States
Centre Hospitalier Universitaire de Toulouse

Toulouse, Haute-Garonne, 31059, France
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria

Lisbon, 1649-035, Portugal
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio

Porto, 4099-001, Portugal
Chang Gung Memorial Hospital

Taoyuan, 333, Taiwan
China Medical University Hospital

Taichung, 40447, Taiwan
Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy
Hospital Clínico San Carlos

Madrid, 28040, Spain
Hospital El Cruce

San Juan Bautista, 1888, Argentina
Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB, Argentina
Hospital Son Llàtzer

Palma de Mallorca, 07198, Spain
Hospital Universitario Clementino Fraga Filho

Botafogo, Rio de Janeiro, 22281-100, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

São Paulo, 14051-140, Brazil
Hôpital de la Timone

Marseille, 13385, France
Indiana University Health University Hospital

Indianapolis, Indiana, 46202, United States
Instituto Fleni

Buenos Aires, C1428 AQK, Argentina
Instituto de Neurologia de Curitiba

Curitiba, 81210-310, Brazil
Istanbul Üniversitesi - Istanbul Tip Fakültesi

Istanbul, 34093, Turkey (Türkiye)
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta

Milan, 20133, Italy
Johns Hopkins University Neurology Research Office

Baltimore, Maryland, 21287, United States
Mayo Clinic

Scottsdale, Arizona, 85259, United States
National Taiwan University Hospital

Taipei, 100, Taiwan
Norrlands Universitetssjukhus

Umeå, 901 85, Sweden
Oregon Health and Science University

Portland, Oregon, 97239, United States
Perron Institute for Neurological and Translational Science

Murdoch, Western Australia, 6150, Australia
Taipei Veterans General Hospital

Taipei, 11217, Taiwan
The Cyprus Institute of Neurology and Genetics

Égkomi, 2371, Cyprus
The Neurological Institute of New York

New York, New York, 10032, United States
Toronto General Hospital

Toronto, Ontario, M5G2C4, Canada
Universidade Estadual de Campinas

Campinas, 13083-888, Brazil
University of North Carolina Hospitals - Neurology Clinic

Chapel Hill, North Carolina, 27514, United States
University of Washington Medical Center

Seattle, Washington, 98195, United States
Vancouver General Hospital

Vancouver, British Columbia, V5Z 1M9, Canada

Conditions

Explore the condition pages connected to this study.

HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOID POLYNEUROPATHY HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOID POLYNEUROPATHY