500
Participants
Start Date
February 16, 2024
Primary Completion Date
December 20, 2034
Study Completion Date
December 20, 2034
Cipaglucosidase alfa
Enzyme Replacement Therapy (ERT) via intravenous infusion
Miglustat
Participants received ATB200 co-administered with AT2221 (Miglustat)
Alglucosidase alfa or Avalglucosidase alfa
Patients prescribed other commercially available ERT after local regulatory approval
Untreated
Patients who are not currently receiving any medical therapy for Pompe disease.
NOT_YET_RECRUITING
University Medical Centre Ljubljana, Institute of Clinical Neurophysiology, Ljubljana
NOT_YET_RECRUITING
Medizinische Universitaet Wien, Vienna
RECRUITING
Laboratory for Muscle Diseases and Neuropathies, Leuven
NOT_YET_RECRUITING
University of Szeged, Szent-Györgyi Albert Clinical Center, Szeged
NOT_YET_RECRUITING
University of Pécs, Pécs
RECRUITING
Aarhus Universitets hospital, Aarhus C
RECRUITING
NYU Neurogenetics, NYU Langone Medical Center, New York
NOT_YET_RECRUITING
Department of Neurosciences Rita Levi Montalcini, University of Torino, Torino
RECRUITING
University of Pittsburgh, Pittsburgh
RECRUITING
UPMC Children's Hospital of Pittsburgh, Pittsburgh
RECRUITING
University of Pennsylvania Perelman Center for Advanced Medicine, Philadelphia
RECRUITING
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax
RECRUITING
Duke University Medical Center, Durham
RECRUITING
Emory University, Atlanta
RECRUITING
Wolfson Children's Hospital, Jacksonville
NOT_YET_RECRUITING
Regional Coordinating Centre for Rare Diseases, university Hospital of Udine, Udine, Italy, Udine
RECRUITING
Universitätsklinikum Gießen und Marburg GmhH, Marburg
RECRUITING
Ruhr-Universität Bochum im St. Josef-Hospital, Bochum
RECRUITING
University of Cincinnati Medical Center, Cincinnati
RECRUITING
Cincinnati Children's Hospital, Cincinnati
RECRUITING
Indiana University, IU Health Physicians Neurology, Indianapolis
RECRUITING
Washington University School of Medicine, St Louis
NOT_YET_RECRUITING
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari, Bari
RECRUITING
University of Arkansas Medical Science, Little Rock
NOT_YET_RECRUITING
Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular Diseases, Napoli
NOT_YET_RECRUITING
Universitat Munchen - Friedrich Baur Institut, München
NOT_YET_RECRUITING
University of Utah, Salt Lake City
NOT_YET_RECRUITING
Universitaetsklinikum Ulm, Ulm
RECRUITING
University of California Irvine, Irvine
NOT_YET_RECRUITING
Eginition Hospital, Athens
RECRUITING
SphinCS, Institute of Clinical Science in LSD, Höchheim
NOT_YET_RECRUITING
Dept of Pediatrics Erasmus MC - Sophia Children's Hospital, Rotterdam
NOT_YET_RECRUITING
entrum Medyczne Medyk, Rzeszów
RECRUITING
Queen Elizabeth Hospital Birmingham, Birmingham
RECRUITING
Cambridge University - Addenbrooke's Hospital, Cambridge
NOT_YET_RECRUITING
University Hospital of Wale, Cardiff, Cardiff
RECRUITING
National Hospital for Neurology and Neurosurgery, London
RECRUITING
Great Ormond Street Hospital NHS Foundation Trust, London
RECRUITING
Royal Free Hospital NHS Foundation Trust, Manchester
RECRUITING
Salford Royal NHS Foundation Trust, Salford
Lead Sponsor
Amicus Therapeutics
INDUSTRY