Phase
Condition
Amyloidosis
Treatment
Eplontersen
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged bythe Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completionof either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both areInvestigator-Sponsored studies with inotersen - the unconjugated version ofeplontersen) as judged by the Investigator and Sponsor.
Must have given written informed consent (signed and dated) and any authorizationsrequired 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, bilateralsalpingectomy, 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 spontaneousamenorrhea without an alternative medical cause and follicle-stimulatinghormone (FSH) levels in the postmenopausal range for the laboratoryinvolved;
- Abstinent*;
- If engaged in sexual relations of child-bearing potential, agree to usehighly effective contraceptive methods from the time of signing theinformed consent form until at least 24 weeks after the last dose ofeplontersen and agree to receive pregnancy tests per protocol.
Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent*, ifengaged in sexual relations with a woman of child-bearing potential (WOCBP),the participant or the participant's non-pregnant female partner must use ahighly effective contraceptive method from the time of signing the informedconsent form until at least 24 weeks after the last dose of eplontersen. *Abstinence (i.e., refraining from heterosexual intercourse throughout theduration 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-ovulationmethods), declaration of abstinence for the duration of a trial and withdrawalare not acceptable methods of contraception.
Willingness to adhere to vitamin A supplementation per protocol.
Exclusion
Exclusion Criteria:
- Have any new condition or worsening of existing condition that in the opinion of theInvestigator or Sponsor would make the participant unsuitable for enrollment orcould interfere with the participant taking part in or completing the study.
Study Design
Study Description
Connect with a study center
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
ArgentinaSite Not Available
Instituto Fleni
Buenos Aires, C1428 AQK
ArgentinaSite Not Available
Hospital El Cruce
Florencio Varela, 1888
ArgentinaSite Not Available
Perron Institute for Neurological and Translational Science
Murdoch, Western Australia 6150
AustraliaSite Not Available
Hospital Universitario Clementino Fraga Filho
Botafogo, Rio De Janeiro 22281-100
BrazilSite Not Available
Universidade Estadual de Campinas
Campinas, 13083-888
BrazilSite Not Available
Instituto de Neurologia de Curitiba
Curitiba, 81210-310
BrazilSite Not Available
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
São Paulo, 14051-140
BrazilSite Not Available
Vancouver General Hospital
Vancouver, British Columbia V5Z 1M9
CanadaSite Not Available
Toronto General Hospital
Toronto, Ontario M5G2C4
CanadaSite Not Available
The Cyprus Institute of Neurology and Genetics
Égkomi, 2371
CyprusSite Not Available
Centre Hospitalier Universitaire de Toulouse
Toulouse, Haute-Garonne 31059
FranceSite Not Available
Hôpital de la Timone
Marseille, 13385
FranceSite Not Available
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta
Milano, 20133
ItalySite Not Available
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100
ItalySite Not Available
Auckland City Hospital
Grafton, 1023
New ZealandSite Not Available
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
Lisboa, 1649-035
PortugalSite Not Available
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
Porto, 4099-001
PortugalSite Not Available
Hospital Clínico San Carlos
Madrid, 28040
SpainSite Not Available
Hospital Son Llàtzer
Palma De Mallorca, 07198
SpainSite Not Available
Norrlands Universitetssjukhus
Umeå, 901 85
SwedenSite Not Available
China Medical University Hospital
Taichung, 40447
TaiwanSite Not Available
Taipei Veterans General Hospital
Taipei, 11217
TaiwanSite Not Available
National Taiwan University Hospital
Taipei City, 100
TaiwanSite Not Available
Chang Gung Memorial Hospital
Taoyuan, 333
TaiwanSite Not Available
Istanbul Üniversitesi - Istanbul Tip Fakültesi
Istanbul, 34093
TurkeySite Not Available
Mayo Clinic
Scottsdale, Arizona 85259
United StatesSite Not Available
Indiana University Health University Hospital
Indianapolis, Indiana 46202
United StatesSite Not Available
Johns Hopkins University Neurology Research Office
Baltimore, Maryland 21287
United StatesSite Not Available
Boston University School of Medicine
Boston, Massachusetts 02118
United StatesSite Not Available
The Neurological Institute of New York
New York, New York 10032
United StatesSite Not Available
University of North Carolina Hospitals - Neurology Clinic
Chapel Hill, North Carolina 27514
United StatesSite Not Available
Oregon Health and Science University
Portland, Oregon 97239
United StatesSite Not Available
University of Washington Medical Center
Seattle, Washington 98195
United StatesSite Not Available
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