40 completed, 15 unknown, 9 recruiting, 7 terminated. 16 Phase 2, 14 Phase 1, 10 Phase 4, 9 Not applicable, 1 Early Phase 1.
Follow Pompe Disease: new trials by email Literature Review of Pompe Disease trials Research Report on Pompe Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD) S-606001, ERT - Pompe Disease | Phase 2 | Not yet recruiting | Shionogi | 60 | 2026-11-02 | 2026-10-13 |
| MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT) Antiphospholipid (aPL)-Positive, Rheumatic Arthritis | Not yet recruiting | Axiom Bioregistry | 5000 | 2026-10-18 | 2026-10-23 | |
| A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD) S-606001 - Pompe Disease | Phase 1 | Not yet recruiting | Shionogi | 15 | 2026-09-30 | 2026-08-26 |
| Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease Pompe Disease | Recruiting | IRCCS National Neurological Institute "C. Mondino" Foundation | 20 | 2026-03-30 | 2026-06-24 | |
| Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) S-606001, Placebo - Pompe Disease | Phase 2 | Recruiting | Shionogi | 45 | 2025-10-30 | 2026-06-23 |
| Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders FBIndex - Inclusion Body Myositis, Myotonic Dystrophy | Enrolling by invitation | LMU Klinikum | 108 | 2024-09-09 | 2025-09-10 | |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases Alpha-Thalassemia, Beta-Thalassemia | Unknown | xCures | 1000 | 2024-06-10 | 2024-11-14 | |
| Nutritional Therapy in Late-onset Pompe Disease Multi-ingredient supplement (PDT-MIS), Placebo (PLA) - Pompe Disease, Muscle Loss | Phase 2 | Unknown | McMaster University | 28 | 2024-04-01 | 2023-11-14 |
| A Global Prospective Observational Registry of Patients With Pompe Disease Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa - Pompe Disease | Recruiting | Amicus Therapeutics | 500 | 2024-02-16 | 2026-03-10 | |
| Pompe Disease RWE Long-term Monitoring Program Pompe Disease | Recruiting | National Taiwan University Hospital | 40 | 2023-10-01 | 2026-09-22 | |
| Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study. Siemens Magnetom 7T Plus - Pompe Disease, McArdle Disease | Active not recruiting | Medical University of Vienna | 30 | 2023-08-15 | 2025-08-26 | |
| Avalglucosidase Alfa Pregnancy Study avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666) - Pompe Disease, Pregnancy | Recruiting | Sanofi | 100 | 2022-10-26 | 2026-01-16 | |
| Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years Avalglucosidase Alfa - Glycogen Storage Disease Type II Late Onset, Pompe Disease (Late-onset) | Phase 4 | Active not recruiting | Iris Plug | 6 | 2022-10-13 | 2026-06-17 |
| Clinical Specimen Collection From Pompe Disease Patients Filgrastim - Pompe Disease | Unknown | Serhat Gumrukcu, MD PhD | 12 | 2022-09-01 | 2022-05-19 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| MSOT in Pompe Disease Multispectral Optoacoustic Tomography - Pompe Disease, Pompe Disease (Late-onset) | Not applicable | Completed | University of Erlangen-Nürnberg Medical School | 20 | 2022-05-17 | 2025-03-12 |
| Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA) Pompe Disease | Unknown | University Hospital Center of Martinique | 100 | 2021-11-01 | 2021-10-29 | |
| A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting Pompe Disease, Mucopolysaccharidosis Type I (MPS I) | Completed | Sanofi | 57 | 2021-10-14 | 2024-02-14 | |
| High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics Pompe Disease | Unknown | National Taiwan University Hospital | 1500 | 2021-08-01 | 2023-04-19 | |
| Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease Testing of reliability / validity of new questionnaire - Pompe Disease | Not applicable | Completed | University Children's Hospital, Zurich | 101 | 2021-07-01 | 2023-12-05 |
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Glycogen Storage Disease Type II (80) · Glycogen Storage Disease (8) · Lysosomal Storage Diseases (5) · Gaucher Disease (4) · Rare Diseases (4) · Anemia, Sickle Cell (3) · Amyotrophic Lateral Sclerosis (2) · Cystic Fibrosis (2) · Fabry Disease (2) · Mucopolysaccharidosis I (2) · Myasthenia Gravis (2) · Nephritis, Hereditary (2) · alpha-Thalassemia (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1)
Brain Diseases (80) · Brain Diseases, Metabolic (80) · Brain Diseases, Metabolic, Inborn (80) · Carbohydrate Metabolism, Inborn Errors (80) · Central Nervous System Diseases (80) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (80) · Genetic Diseases, Inborn (80) · Lysosomal Storage Diseases, Nervous System (80) · Metabolism, Inborn Errors (80) · Nervous System Diseases (80) · Nutritional and Metabolic Diseases (80) · Metabolic Diseases (79) · Lysosomal Storage Diseases (75) · Glycogen Storage Disease (72) · Pathological Conditions, Signs and Symptoms (10)
GAA protein, human (17) · Rituximab (4) · miglustat (4) · Albuterol (3) · 1-Deoxynojirimycin (2) · Methotrexate (2) · Watchful Waiting (2) · Acetaminophen (1) · Blood Specimen Collection (1) · Bortezomib (1) · Capsules (1) · Clenbuterol (1) · Continuous Positive Airway Pressure (1) · Diet (1) · Diphenhydramine (1)
Genzyme, a Sanofi Company (14) · National Taiwan University Hospital (9) · Duke University (8) · University of Florida (8) · Amicus Therapeutics (6) · Shionogi (3) · BioMarin Pharmaceutical (2) · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau (2) · LMU Klinikum (2) · O & O Alpan LLC (2) · Sanofi (2) · Spark Therapeutics, Inc. (2) · University of California, Irvine (2) · AskBio Inc (1) · Axiom Bioregistry (1)
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Barry J Byrne, MD, PhD (3) · Tahseen Mozaffar, MD (3) · Yin-Hsiu Chien, MD, PhD (3) · Dwight D Koeberl, MD, PhD (2) · Priya Kishnani, MD (2) · Whu-Liang Hwu, MD, PhD (2) · Yin-Hsiu Chien (2) · Barbara K Smith, PT, PhD (1) · Barbara K. Smith, PT, PhD (1) · Barry J. Byrne, MD, PhD (1) · Benedikt Schoser, MD (1)
PMID 19775921, PMID 16267188, PMID 16849828, PMID 16900450, PMID 19129538, PMID 21637107, PMID 22003934, PMID 22237443, PMID 22794786, PMID 23570273, PMID 23601496, PMID 23825616, PMID 24443373, PMID 25741864, PMID 26693141, PMID 27493997, PMID 28168524, PMID 28495044, PMID 28648664, PMID 28814660, PMID 29050825, PMID 29289479, PMID 29565424, PMID 30025991, PMID 30214072, PMID 30943998, PMID 31086307, PMID 31353854, PMID 31839530, PMID 32518148, PMID 32793419, PMID 32849613, PMID 33495531, PMID 34420548, PMID 34441913, PMID 35618441, PMID 35813979, PMID 36214004, PMID 36299500, PMID 36399131, PMID 36805083, PMID 36864705, PMID 37705424, PMID 38057636, PMID 38715621, PMID 38896264, PMID 39245687, PMID 40920287, PMID 41272769, PMID 41631150
2019 (2), 2015 (1), 2025 (1), P01HL059412 (1), R21HD090752 (1)