🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Contributor sign in
Latest
Clinical Trials in Canada / NCT05071300
Active, not recruiting Phase 3

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 · tracked via the Priya Life Science Canada tracker
Phase
Phase 3
Started
2022-01-04
Last updated
2025-12-04

Condition(s) studied

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Investigational drug(s) / intervention(s)

Eplontersen →

Eplontersen: Eplontersen will be administered by SC injection.

Study summary

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).

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: 1. 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. 2. 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. 3. Satisfy the following: 1. 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. 2. 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. 4. Willingness to adhere to vitamin A supplementation per protocol. Exclusion Criteria: 1\. 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.

Primary outcome measure(s)

Trial sites (33)

FacilityCityRegionStatus
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 Argentina
Instituto Fleni Buenos Aires Argentina
Hospital El Cruce San Juan Bautista Argentina
Perron Institute for Neurological and Translational Science Murdoch Western Australia
Hospital Universitario Clementino Fraga Filho Botafogo Rio de Janeiro
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
Toronto General Hospital Toronto Ontario
The Cyprus Institute of Neurology and Genetics Égkomi Cyprus
Centre Hospitalier Universitaire de Toulouse Toulouse Haute-Garonne
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
Hospital Clínico San Carlos Madrid Spain
Hospital Son Llàtzer Palma de Mallorca Spain
Norrlands Universitetssjukhus Umeå Sweden
China Medical University Hospital Taichung Taiwan
National Taiwan University Hospital Taipei Taiwan
Taipei Veterans General Hospital Taipei Taiwan
Chang Gung Memorial Hospital Taoyuan Taiwan
Istanbul Üniversitesi - Istanbul Tip Fakültesi Istanbul Turkey (Türkiye)

More Ionis Pharmaceuticals, Inc. trials in Canada

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05071300 on ClinicalTrials.gov ↗ ← All trials in Canada