| Recruiting | Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease NCT07478172 | University of Missouri-Columbia | N/A |
| Recruiting | A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participant NCT07282847 | AskBio Inc | Phase 1 / Phase 2 |
| Not Yet Recruiting | Treatment Frequency Reduction in Pompe Disease NCT06575829 | Erasmus Medical Center | Phase 4 |
| Recruiting | Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug NCT06391736 | GeneCradle Inc | Phase 1 / Phase 2 |
| Active Not Recruiting | A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease NCT06150820 | Astellas Gene Therapies | N/A |
| Not Yet Recruiting | Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) NCT06178432 | Huashan Hospital | EARLY_Phase 1 |
| Unknown | Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease NCT05951790 | Fondazione Don Carlo Gnocchi Onlus | N/A |
| Withdrawn | Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease NCT04094948 | Duke University | Phase 2 |
| Completed | MSOT in Pompe Disease NCT05083806 | University of Erlangen-Nürnberg Medical School | N/A |
| Unknown | Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease NCT05272969 | LMU Klinikum | — |
| Active Not Recruiting | Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases NCT04929002 | Rigshospitalet, Denmark | — |
| Completed | Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders NCT04907162 | LMU Klinikum | — |
| Active Not Recruiting | Gene Transfer Study in Patients With Late Onset Pompe Disease NCT04174105 | Astellas Gene Therapies | Phase 1 / Phase 2 |
| Active Not Recruiting | A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) NCT04093349 | Spark Therapeutics, Inc. | Phase 1 / Phase 2 |
| Active Not Recruiting | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y NCT03911505 | Amicus Therapeutics | Phase 3 |
| Completed | A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe D NCT04138277 | Amicus Therapeutics | Phase 3 |
| Completed | Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pomp NCT03893240 | Spark Therapeutics, Inc. | N/A |
| Completed | Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease NCT04120168 | Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition | — |
| Active Not Recruiting | Developing a Management Approach for Patients With "Late-Onset" Pompe Disease NCT03694561 | Duke University | — |
| Completed | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Diseas NCT03729362 | Amicus Therapeutics | Phase 3 |
| Completed | Pompe Gene Therapy- Screening for Eligibility NCT03285126 | Duke University | — |
| Completed | Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidas NCT01288027 | Genzyme, a Sanofi Company | Phase 4 |
| Recruiting | Pompe Pregnancy Sub-Registry NCT00567073 | Genzyme, a Sanofi Company | — |
| Completed | Late-Onset Treatment Study Extension Protocol NCT00455195 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving NCT00731081 | Genzyme, a Sanofi Company | — |
| Completed | Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respirato NCT00268944 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-O NCT00158600 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | A Study of rhGAA in Patients With Late-Onset Pompe Disease NCT00250939 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were P NCT00765414 | Genzyme, a Sanofi Company | Phase 2 |
| Approved For Marketing | Alglucosidase Alfa Temporary Access Program NCT00520143 | Genzyme, a Sanofi Company | — |