🏛️ Verified NIH Record • Updated 2025-12-02

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 (NCT05071300)

An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Ionis Pharmaceuticals, Inc.

🩺 Plain-English Summary (8th-Grade Level)

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).

Who can join: Review the eligibility criteria below.

Location: Mayo Clinic — Scottsdale, Arizona

Age range: 18 Years

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (33 Sites)

Showing the first 25 of 33 sites.

  • Mayo Clinic — Scottsdale, Arizona
  • Indiana University Health University Hospital — Indianapolis, Indiana
  • Johns Hopkins University Neurology Research Office — Baltimore, Maryland
  • Boston University School of Medicine — Boston, Massachusetts
  • The Neurological Institute of New York — New York, New York
  • University of North Carolina Hospitals - Neurology Clinic — Chapel Hill, North Carolina
  • Oregon Health and Science University — Portland, Oregon
  • University of Washington Medical Center — Seattle, Washington
  • Hospital Italiano de Buenos Aires — Buenos Aires,
  • Instituto Fleni — Buenos Aires,
  • Hospital El Cruce — San Juan Bautista,
  • Perron Institute for Neurological and Translational Science — Murdoch, Western Australia
  • Hospital Universitario Clementino Fraga Filho — Botafogo, Rio de Janeiro
  • Universidade Estadual de Campinas — Campinas,
  • Instituto de Neurologia de Curitiba — Curitiba,
  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto — São Paulo,
  • Vancouver General Hospital — Vancouver, British Columbia
  • Toronto General Hospital — Toronto, Ontario
  • The Cyprus Institute of Neurology and Genetics — Égkomi,
  • Centre Hospitalier Universitaire de Toulouse — Toulouse, Haute-Garonne
  • Hôpital de la Timone — Marseille,
  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta — Milan,
  • Fondazione IRCCS Policlinico San Matteo — Pavia,
  • Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria — Lisbon,
  • Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio — Porto,

📞 Contact the Study Coordinator

Phone: (800) 664-4580

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗