125
Participants
Start Date
December 4, 2018
Primary Completion Date
December 15, 2020
Study Completion Date
January 15, 2021
Cipaglucosidase Alfa
Participants received an intravenous (IV) infusion dose over a 4-hour duration every 2 weeks (Q2W).
Miglustat
Participants received weight-based doses 1 hour prior to cipaglucosidase alfa infusion Q2W.
Alglucosidase Alfa
Participants received an IV infusion dose over a 4-hour duration Q2W.
Placebo
Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W.
National Taiwan University Hospital, Taipei
University Medical Centre Ljubljana, Ljubljana
Semmelweis University, Institute of Genomic Medicine and Rare Disease, Budapest
Rigshospitalet Copenhagen Neuromuscular Center, Copenhagen
Westmead Hospital, Westmead
UZ Leuven, Leuven
Monash Medical Centre, Melbourne
Royal Brisbane & Women's Hospital, Herston
Royal Adelaide Hospital, Adelaide
University of Szeged, Szeged
University of Pécs, Pécs
Aarhus Universitets Hospital, Aarhus N
NYU School of Medicine, New York
The Feinstein Institute for Medical Research, Manhasset
Eginition Hospital, Athens
Hôpital de la Timone, Marseille
University of Pittsburgh, Pittsburgh
Penn State Health Milton S. Hershey Medical Center, Hershey
University of Pennsylvania, Philadelphia
Lysosomal and Rare Disorders Research, Fairfax
Duke University Medical Center, Durham
Emory Clinic, Atlanta
UF Helath: University of Florida Clinical Research Center, Gainesville
University of South Florida Research Center, Tampa
Sahlgrenska University Hospital, Gothenburg
The Ohio State University Wexner Medical Center, Columbus
University of Cincinnati Neurology, Cincinnati
Cincinnati Children's Hospital Medical Center, Cincinnati
Indiana University Health Neuroscience Center, Indianapolis
Universitätsklinikum Münster, Münster
Pusan National University, Yangsan
Universitätsklinikum Bonn, Bonn
University of Minnesota Clinical Research Unit, Minneapolis
Hôpital Salengro, Lille
Billings Clinic, Billings
Washington University School of Medicine, St Louis
University of Kansas Medical Center, Kansas City
Hôpital Neurologique Pierre Wertheimer, Bron
University of Arkansas for Medical Sciences, Little Rock
University Clinical Centre of the Republic of Srpska, Banja Luka
University of Texas Health Science Center San Antonio, San Antonio
UOC Genetica Medica, Napoli
Friedrich-Baur Institut, Munich
University of Utah, Center for Clinical and Translational Sciences, Salt Lake City
Neuromuscular Research Center, Phoenix
Hôpital Raymond Poincaré, Garches
University of California, Irvine, Irvine
Oregon Health & Science University, Portland
UOC di Neurologia e Malattie Neuromuscolari, Messina
Hackensack University Medical Center, Hackensack
Hospital Universitario Austral, Buenos Aires
Medizinische Universität Innsbruck, Innsbruck
UMHAT Alexandrovska, Sofia
Kagoshima University Hospital, Kagoshima
Izumi City General Hospital, Osaka
National Center of Neurology and Psychiatry, Tokyo
University of Auckland, Auckland
Queen Elizabeth Hospital Birmingham, Birmingham
Cambridge University Hospitals, Cambridge
Heritage Medical Research Clinic, University of Calgary, Calgary
McMaster University Medical Centre, Hamilton
Hôpital Pasteur, Nice
Universitätsklinikum Halle (Saale), Halle
Hokkaido University Hospital, Sapporo
Fukuoka University Hospital, Fukuoka
The Jikei University Hospital, Tokyo
Erasmus MC, Rotterdam
Szpital Uniwersytecki w Krakowie, Małogoskie
Centrum Medyczne, Rzeszów
Seoul National University Hospital, Seoul
Hospital de la Santa Creu I Sant Pau, Barcelona
Royal Free Hospital NHS, London
Salford Royal NHS Foundation Trust, Salford
Lead Sponsor
Amicus Therapeutics
INDUSTRY