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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 Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug remains safe over time and helps control nerve damage.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Eplontersen (a drug given as a shot under the skin)

What this could lead to

If successful, this could confirm eplontersen as a safe long-term treatment option to slow nerve damage in people with hATTR-PN.

What could go wrong

This is an extension study, so participants already know the drug is generally safe, but long-term risks (like effects on platelets, kidneys, or liver) are still being checked. The trial is not designed to prove a cure, only to manage the disease.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

familial amyloid neuropathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOID POLYNEUROPATHY

Contacts and locations

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