NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

PHASE3CompletedINTERVENTIONAL
Enrollment

168

Participants

Timeline

Start Date

December 11, 2019

Primary Completion Date

April 11, 2023

Study Completion Date

July 12, 2023

Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
DRUG

Inotersen

Inotersen by subcutaneous injection

DRUG

Eplontersen

Eplontersen by subcutaneous injection

Trial Locations (45)

100

National Taiwan University Hospital, Taipei

333

Chang Gung Memorial Hospital - Linkou Branch, Taoyuan

1023

Auckland City Hospital, Grafton

1888

Hospital El Cruce, San Juan Bautista

2371

The Cyprus Institute of Neurology and Genetics, Égkomi

6009

Perron Institute for Neurological and Translational Science, Nedlands

10032

The Neurological Institute of New York, New York

11217

Taipei Veterans General Hospital, Taipei City

13005

Hôpital de la Timone, Marseille

19104

Penn Presbyterian Medical Center, Philadelphia

20133

Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan

21224

Johns Hopkins University Neurology Research Office, Baltimore

27100

Fondazione IRCCS Policlinico San Matteo, Pavia

27599

University of North Carolina Hospitals - Neurology Clinic, Chapel Hill

28040

Hospital Clínico San Carlos, Madrid

31059

Centre Hospitalier Universitaire de Toulouse, Toulouse

32224

Mayo Clinic - Jacksonville, Jacksonville

34093

İstanbul Üniversitesi - Istanbul Tıp Fakültesi, Istanbul

40447

China Medical University Hospital, Taichung

46202

Indiana University School of Medicine - Indianapolis, Indianapolis

55905

Mayo Clinic - Rochester, Rochester

66160

University of Kansas Medical Center, Kansas City

69120

Universitätsklinikum Heidelberg, Heidelberg

85259

Mayo Clinic - Arizona, Scottsdale

94270

Hôpital Bicêtre, Le Kremlin-Bicêtre

97080

Universitätsklinikum Würzburg, Würzburg

97239

Oregon Health and Science University, Portland

98124

Azienda Ospedaliera Universitaria Policlinico Gaetano Martino, Messina

98195

University of Washington Medical Center, Seattle

02118

Boston University School of Medicine, Boston

C1199ABB

Hospital Italiano de Buenos Aires, Ciudad Autónoma de Buenos Aires

C1023AAB

STAT Research, Buenos Aires

C1428 AQK

Instituto Fleni, Buenos Aires

81210-310

Instituto de Neurologia de Curitiba, Curitiba

13083-970

Universidade Estadual de Campinas, Campinas

14049-900

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto, Ribeirão Preto

21941-617

Hospital Universitário Clementino Fraga Filho, Rio de Janeiro

04032-060

Associação de Assistência à Criança Deficiente - Unidade Lar Escola, São Paulo

M4C 3E7

Toronto General Hospital, Toronto

711 10

University General Hospital of Heraklion (PAGNI), Heraklion

00168

Fondazione Policlinico Universitario Agostino Gemelli, Roma

1649-028

Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria, Lisbon

4099-001

Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio, Porto

07198

Hospital Son Llàtzer, Palma de Mallorca

907 37

Norrlands Universitetssjukhus, Umeå

Sponsors
All Listed Sponsors
lead

Ionis Pharmaceuticals, Inc.

INDUSTRY