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
🩺 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 ChecklistFull 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.
Explore by condition: All Hereditary Transthyretin-Mediated Amyloid Polyneuropathy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.