69 completed, 24 unknown, 19 recruiting, 12 active not recruiting. 27 Phase 2, 20 Not applicable, 20 Phase 1, 19 Phase 4, 8 Phase 3.
Follow Glycogen Storage Disease Type II: new trials by email Literature Review of Glycogen Storage Disease Type II trials Research Report on Glycogen Storage Disease Type II
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 |
| 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 |
| A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease DNL952 - Late-onset Pompe Disease | Phase 1 | Recruiting | Denali Therapeutics Inc. | 32 | 2026-05-12 | 2026-08-17 |
| A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) AB-1009 (GAA Gene) - Pompe Disease (Late-onset), Pompe Disease Late-Onset | Phase 1 / Phase 2 | Recruiting | AskBio Inc | 12 | 2026-04-15 | 2026-10-13 |
| 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 | |
| Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease Whole-body Electrical Muscle Stimulation Exercise - Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis | Not applicable | Recruiting | University of Missouri-Columbia | 50 | 2026-03-10 | 2026-03-17 |
| 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 |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD Avalglucosidase alfa - Glycogen Storage Disease Type II, Pompe's Disease | Phase 4 | Recruiting | Genzyme, a Sanofi Company | 13 | 2025-05-07 | 2026-03-23 |
| Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases ARN extraction from muscle biopsies - Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne | Not applicable | Recruiting | Assistance Publique Hopitaux De Marseille | 50 | 2025-03-01 | 2025-02-18 |
| A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD reSEES - Intermediate AMD | Not applicable | Recruiting | Oculox Technologies SA | 30 | 2024-11-01 | 2024-11-15 |
| Treatment Frequency Reduction in Pompe Disease Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks - Pompe Disease (Late-onset), GAA Deficiency | Phase 4 | Not yet recruiting | Erasmus Medical Center | 10 | 2024-10-01 | 2024-08-28 |
| 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 | |
| Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug GC301 - Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Recruiting | GeneCradle Inc | 33 | 2024-04-19 | 2025-07-03 |
| 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 | |
| A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease No Intervention - Pompe Disease (Late-onset) | Not applicable | Active not recruiting | Astellas Gene Therapies | 119 | 2024-02-01 | 2026-10-08 |
| Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) CRG003 injection - Pompe Disease (Late-onset) | Early Phase 1 | Not yet recruiting | Huashan Hospital | 6 | 2023-12-01 | 2023-12-21 |
| Pompe Disease RWE Long-term Monitoring Program Pompe Disease | Recruiting | National Taiwan University Hospital | 40 | 2023-10-01 | 2026-09-22 |
Showing 20 of 146. Sign in to see all of them.
Glycogen Storage Disease (11) · Lysosomal Storage Diseases (9) · Gaucher Disease (6) · Myositis, Inclusion Body (5) · Neuromuscular Diseases (5) · Fabry Disease (4) · Glycogen Storage Disease Type V (4) · Muscular Atrophy, Spinal (4) · Muscular Diseases (4) · Muscular Dystrophy, Duchenne (4) · Myotonic Dystrophy (4) · Rare Diseases (4) · Amyotrophic Lateral Sclerosis (3) · Anemia, Sickle Cell (3)
Brain Diseases (146) · Brain Diseases, Metabolic (146) · Brain Diseases, Metabolic, Inborn (146) · Central Nervous System Diseases (146) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (146) · Genetic Diseases, Inborn (146) · Lysosomal Storage Diseases, Nervous System (146) · Nervous System Diseases (146) · Nutritional and Metabolic Diseases (146) · Carbohydrate Metabolism, Inborn Errors (145) · Metabolism, Inborn Errors (145) · Metabolic Diseases (144) · Lysosomal Storage Diseases (137) · Glycogen Storage Disease (135) · Musculoskeletal Diseases (20)
GAA protein, human (35) · miglustat (7) · Rituximab (4) · Albuterol (3) · Watchful Waiting (3) · 1-Deoxynojirimycin (2) · Blood Specimen Collection (2) · Clenbuterol (2) · Methotrexate (2) · Observation (2) · Ranibizumab (2) · Vital Signs (2) · Walk Test (2) · Acetaminophen (1) · Bortezomib (1)
Genzyme, a Sanofi Company (33) · Duke University (12) · Amicus Therapeutics (9) · National Taiwan University Hospital (9) · University of Florida (8) · LMU Klinikum (5) · Sanofi (4) · O & O Alpan LLC (3) · Shionogi (3) · AskBio Inc (2) · Astellas Gene Therapies (2) · BioMarin Pharmaceutical (2) · CENTOGENE GmbH Rostock (2) · Centre Hospitalier Universitaire de Nice (2) · Centre Hospitalier de Cayenne (2)
United States (82) · Germany (29) · France (26) · United Kingdom (22) · Taiwan (21) · Italy (19) · Netherlands (18) · Australia (11) · Belgium (11) · Canada (10) · Denmark (10) · Spain (10) · China (7) · Israel (6) · Japan (5)
Barry J Byrne, MD, PhD (3) · Harrison Jones, PhD (3) · Ozlem Goker-Alpan, MD (3) · Tahseen Mozaffar, MD (3) · Yin-Hsiu Chien, MD, PhD (3) · Dwight D Koeberl, MD, PhD (2) · Peter Bauer, Prof. (2) · Priya Kishnani, MD (2) · Renuka Limgala, PhD (2) · Stephan Wenninger, PD Dr. med. (2) · Whu-Liang Hwu, MD, PhD (2)
PMID 19775921, PMID 41631150, PMID 31303277, PMID 16267188, PMID 16849828, PMID 16900450, PMID 19129538, PMID 20393176, PMID 21543987, PMID 21637107, PMID 22003934, PMID 22237443, PMID 22794786, PMID 23570273, PMID 23587901, PMID 23601496, PMID 23825616, PMID 24443373, PMID 25741864, PMID 26381077, PMID 26693141, PMID 27106274, PMID 27473031, PMID 27493997, PMID 28168524, PMID 28495044, PMID 28648664, PMID 28649546, PMID 28814660, PMID 29050825, PMID 29289479, PMID 29565424, PMID 30025991, PMID 30214072, PMID 30430231, PMID 30943998, PMID 30944902, PMID 30990523, PMID 31086307, PMID 31315600, PMID 31353854, PMID 31492716, PMID 31839530, PMID 32518148, PMID 32793419, PMID 32849613, PMID 33127291, PMID 33495531, PMID 33711277, PMID 33881155
2019 (3), 2025 (2), 2005 (1), 2015 (1), P01HL059412 (1), R21AR069880 (1), R21HD090752 (1)