A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

PHASE3CompletedINTERVENTIONAL
Enrollment

119

Participants

Timeline

Start Date

December 18, 2019

Primary Completion Date

December 31, 2024

Study Completion Date

December 31, 2024

Conditions
Pompe Disease (Late-onset)
Interventions
DRUG

AT2221

Participants received ATB200 co-administered with AT2221 (Miglustat)

BIOLOGICAL

ATB200

Enzyme Replacement Therapy via intravenous infusion

Trial Locations (60)

100

National Taiwan University Hospital, Taipei

1000

University Medical Centre Ljubljana, Ljubljana

1085

Semmelweis University, Budapest

1142

University of Auckland, Auckland

3000

UZ Leuven, Leuven

3168

Monash Medical Centre, Clayton

4029

Royal Brisbane & Women's Hospital, Brisbane

5000

Royal Adelaide Hospital, Adelaide

6725

University of Szeged, Szeged

7623

University of Pécs, Pécs

10017

NYU School of Medicine, New York

11021

Northwell Health, Great Neck

11528

Eginition Hospital, Athens

13385

Hôpital de la Timone, Marseille

15213

UPMC Montefiore Clinical and Translational Research Center, Pittsburgh

19104

University of Pennsylvania, Philadelphia

22030

Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax

27710

Duke University Medical Center, Durham

30322

Emory Clinic, Atlanta

32610

University of Florida Clinical Research Center, Gainesville

41345

Sahlgrenska University Hospital, Gothenburg

43210

The Ohio State University Wexner Medical Center, Columbus

45219

University of Cincinnati Gardner Neuroscience Institute, Cincinnati

45229

Cincinnati Children's Hospital, Cincinnati

46202

IU Health Neuroscience Center, Indianapolis

48149

Universitätsklinikum Münster, Münster

50612

Pusan National University, Yangsan

53127

Universitätsklinikum Bonn, Bonn

59037

Hôpital Salengro, Lille

59101

Billings Clinic, Billings

63110

Washington University School of Medicine, St Louis

69577

Hôpital Pierre Wertheimer, Bron

72205

University of Arkansas for Medical Sciences, Little Rock

78000

University Clinical Centre of the Republic of Srpska, Banja Luka

80131

Universitaria Federico II, Napoli

80336

Friedrich-Baur Institut, München

84108

University of Utah, Salt Lake City

92380

Hôpital Raymond Poincaré, Garches

92868

University of California, Irvine, Irvine

98124

UOC di Neurologia e Malattie Neuromuscolari, Messina

07061

Hackensack University Medical Center, Hackensack

B1629ODT

Hospital Universitario Austral, Buenos Aires

Unknown

Westmead Hospital, Westmead

Aarhus Universitetshospital, Aarhus

Hospital de la Santa Creu I Sant Pau, Barcelona

A-6020

Medizinische Universität Innsbruck, Innsbruck

T3B 6A8

Alberta Children's Hospital, Heritage Medical Research Clinic, Calgary

L8N 3Z5

McMaster University Medical Centre, Hamilton

06001

Hôpital Pasteur 2, Nice

814-0180

Fukuoka University Hospital, Fukuoka

890-8520

Kagoshima University Hospital, Kagashima

594 0073

Izumi City General Hospital, Osaka

060 8648

Hokkaido University Hospital, Sapporo

105-8471

The Jikei University Hospital, Tokyo

3015 GD

Erasmus MC, Rotterdam

35-326

Centrum Medyczne Medyk, Rzeszów

B15 2TH

Queen Elizabeth Hospital Birmingham, Birmingham

CB2 0QQ

Cambridge University Hospitals NHS Foundation Trust, Cambridge

NW3 2QG

Royal Free Hospital NHS Foundation Trust, London

M6 8HD

Salford Royal NHS Foundation Trust, Salford

Sponsors
All Listed Sponsors
lead

Amicus Therapeutics

INDUSTRY

NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD | Biotech Hunter | Biotech Hunter