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

Running, not enrolling · Phase 3

Conditions studied: Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

In brief

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).

Key facts

Study ID

NCT05071300

Run by

Ionis Pharmaceuticals, Inc.

People needed

151

Starts

2022-01-04

Expected to finish

2029-08-01

Last updated by the study team

2025-12-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged by the Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both are Investigator-Sponsored studies with inotersen - the unconjugated version of eplontersen) as judged by the Investigator and Sponsor.
• Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
• Satisfy the following:
• Females: must be non-pregnant and non-lactating and either:
• Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy);
• Post-menopausal (defined as 12 months of spontaneous amenorrhea in females > 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and follicle-stimulating hormone (FSH) levels in the postmenopausal range for the laboratory involved;
• Abstinent*;
• If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of eplontersen and agree to receive pregnancy tests per protocol.
• Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent*, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the participant or the participant's non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of eplontersen. *Abstinence (i.e., refraining from heterosexual intercourse throughout the duration of study participation) is only acceptable as true abstinence, i.e., when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial and withdrawal are not acceptable methods of contraception.
• Willingness to adhere to vitamin A supplementation per protocol.

You may not qualify if…

• Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrollment or could interfere with the participant taking part in or completing the study.

Where it is running

• 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.