Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Pompe Disease (Late-onset)

Enzyme Replacement Therapy via intravenous infusion

ATB200

Participants received ATB200 co-administered with AT2221 (Miglustat)

AT2221

Neuromuscular Research Center

UCSF Benioff Children's Hospital

University of Florida Clinical Research Center

Infusion Associates

Mayo Clinic

St. Louis Children's Hospital

Duke University Medical Center

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

University of Calgary

National Taiwan University Hospital

ZIP Study - A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18

An Open-label Study of the Pharmacokinetics, Safety, Efficacy, and Pharmacodynamics of ATB200/AT2221 in Pediatric Subjects Aged 12 to < 18 Years With Late-onset Pompe Disease

ATB200 protein and AT2221 concentrations in plasma

incidence of treatment-emergent AEs, SAEs, infusion-associated reactions, and AEs leading to discontinuation of study drug

Change from baseline in Creatine Kinase and Urinary Hexose Tetrasaccharide

Change in anti-rhGAA antibodies from baseline over time

Amicus Therapeutics

Participants received alglucosidase alfa co-administered with placebo capsules.

alglucosidase alfa via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information

alglucosidase alfa

University of Arkansas for Medical Sciences

University of California, Irvine

UF Helath: University of Florida Clinical Research Center

University of South Florida Research Center

Emory Clinic

Indiana University Health Neuroscience Center

University of Kansas Medical Center

University of Minnesota Clinical Research Unit

Washington University School of Medicine

Billings Clinic

Hackensack University Medical Center

The Feinstein Institute for Medical Research

NYU School of Medicine

University of Cincinnati Neurology

Cincinnati Children's Hospital Medical Center

The Ohio State University Wexner Medical Center

Oregon Health & Science University

Penn State Health Milton S. Hershey Medical Center

University of Pennsylvania

University of Pittsburgh

University of Texas Health Science Center San Antonio

University of Utah, Center for Clinical and Translational Sciences

Lysosomal and Rare Disorders Research

Hospital Universitario Austral

Royal Brisbane & Women's Hospital

Royal Adelaide Hospital

Monash Medical Centre

Westmead Hospital

Medizinische Universität Innsbruck

UZ Leuven

University Clinical Centre of the Republic of Srpska

UMHAT Alexandrovska

Heritage Medical Research Clinic, University of Calgary

McMaster University Medical Centre

Aarhus Universitets Hospital

Rigshospitalet Copenhagen Neuromuscular Center

Hôpital Neurologique Pierre Wertheimer

Hôpital Raymond Poincaré

Hôpital Salengro

Hôpital de la Timone

Hôpital Pasteur

Friedrich-Baur Institut

Universitätsklinikum Bonn

Universitätsklinikum Münster

Universitätsklinikum Halle (Saale)

Eginition Hospital

Semmelweis University, Institute of Genomic Medicine and Rare Disease

University of Pécs

University of Szeged

UOC di Neurologia e Malattie Neuromuscolari

UOC Genetica Medica

Hokkaido University Hospital

Fukuoka University Hospital

Kagoshima University Hospital

Izumi City General Hospital

The Jikei University Hospital

National Center of Neurology and Psychiatry

Pusan National University

Seoul National University Hospital

Erasmus MC

University of Auckland

Szpital Uniwersytecki w Krakowie

Centrum Medyczne

University Medical Centre Ljubljana

Hospital de la Santa Creu I Sant Pau

Sahlgrenska University Hospital

Queen Elizabeth Hospital Birmingham

Cambridge University Hospitals

Royal Free Hospital NHS

Salford Royal NHS Foundation Trust

PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in FVC (sitting and supine) to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change in manual muscle strength from baseline to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change in Quantitative muscle strength from baseline to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in scores of PROMIS instruments for physical function, fatigue, dyspnea, and upper extremity questionnaire to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The PROMIS instruments for physical function (20 items), d upper extremity (7 items) measure signs and symptoms using general questions without a temporal reference. The PROMIS instruments for fatigue (8 items) and dyspnea severity (10 items) measure signs and symptoms over the past 7 days. A 5-point scale is used for each instrument (though responses may vary within or among instruments), and a total score is generated for each instrument.

Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The GSGC consists of a 10-meter walk for evaluation of gait, a 4-stair climb, Gower's maneuver, and arising from a chair. Results of the GSGC include the time required to complete the individual tests, individual scores for each of the tests (1 to 7 points for each of gait, 4-stair climb, and Gower's maneuver and 1 to 6 points for arising from a chair), and a total score. The total score ranges from a minimum of 4 points (normal performance) to a maximum of 27 points (worst score).

Change from baseline in TUG to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The TUG test measures the time it takes for the subject to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down will be recorded.

Change from baseline in scores of R-PAct scale questionnaire to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The R-PAct scale is an 18-item questionnaire to measure limitations in activities and restriction in social participation. Possible responses to questions are as follows: unable to perform, able to perform, but with difficulty, and able to perform without difficulty. A raw score ranging from 0 to 36 points is generated. The low score indicates the highest level of disability.

Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The EQ-5D-5L comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. Subjects are asked to indicate their health state by ticking the box next to the most appropriate statement in each of the 5 dimensions. The subject's self rated health is also recorded on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'.

Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo. The Subject's Global Impression of Change is designed to record the subjects' impression of their functional status since starting study drug using a 7-point scale ranging from "very much worse" to "very much improved".

Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in SVC (sitting and supine) to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in MIP to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in MEP to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Change from baseline in SNIP to assess the efficacy of ATB200/AT2221 co-administration compared with alglucosidase alfa/placebo

Evaluation of Treatment Emergent Adverse Events (TEAEs) begins after written informed consent is provided, including study related events that occur as a direct result of a study procedure to assess the safety, tolerability of ATB200/AT2221 co administration compared with alglucosidase alfa/placebo

Measurement of anti-rhGAA Abs (total, cross-reactive, and neutralizing) to assess the Immunogenicity of ATB200/AT2221 co administration compared with alglucosidase alfa/placebo

Area under the curve from time 0 extrapolated to infinite time

hexose/trötthet

ZIP Study - A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18

PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD

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