13 completed, 7 active not recruiting, 4 recruiting, 2 not yet recruiting. 6 Phase 2, 5 Not applicable, 5 Phase 3, 4 Phase 1, 4 Phase 4.
Follow Pompe Disease (Late-onset): new trials by email Literature Review of Pompe Disease (Late-onset) trials Research Report on Pompe Disease (Late-onset)
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease Inspiratory Muscle Training (IMT), Inspiratory Muscle Training (IMT) + Air Stacking - Glycogen Storage Disease Type II, Pompe Disease (Late-onset) | Not applicable | Unknown | Fondazione Don Carlo Gnocchi ETS | 20 | 2023-03-01 | 2023-07-19 |
| Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Clenbuterol, Placebos - Pompe Disease (Late-onset) | Phase 2 | Withdrawn | Duke University | 2023-01-01 | 2022-08-17 | |
| 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 |
| 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 |
| Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease Beck depression inventory fast screen (Questionnaire), Brief Pain Inventory (BPI) (Questionnaire), German Pain Inventory (Questionnaire) - Pompe Disease (Late-onset), Inclusion Body Myositis | Unknown | LMU Klinikum | 95 | 2022-03-31 | 2023-10-10 | |
| Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases No intervention - Glycogen Storage Disease, McArdle Disease | Active not recruiting | Copenhagen University Hospital, Rigshospitalet | 50 | 2021-12-10 | 2025-08-11 | |
| Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory - Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1) | Completed | LMU Klinikum | 82 | 2021-04-15 | 2022-08-24 | |
| Gene Transfer Study in Patients With Late Onset Pompe Disease zocaglusagene nuzaparvovec - Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Active not recruiting | Astellas Gene Therapies | 11 | 2020-10-27 | 2026-09-02 |
| A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) SPK-3006 - Pompe Disease, Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Active not recruiting | Spark Therapeutics, Inc. | 4 | 2020-10-01 | 2024-11-27 |
| ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Cipaglucosidase Alfa, Miglustat - Pompe Disease (Late-onset) | Phase 3 | Active not recruiting | Amicus Therapeutics | 21 | 2020-02-13 | 2026-09-17 |
| A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) AT2221, ATB200 - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 119 | 2019-12-18 | 2026-03-05 |
| Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease Neutralizing Antibody to SPK-3006 capsid - Pompe Disease, Pompe Disease (Late-onset) | Not applicable | Completed | Spark Therapeutics, Inc. | 61 | 2019-06-12 | 2023-03-24 |
| Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease Laboratory Tests - Duchenne Muscular Dystrophy, Pompe Disease (Late-onset) | Completed | Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition | 590 | 2019-04-01 | 2022-10-24 | |
| Developing a Management Approach for Patients With "Late-Onset" Pompe Disease Observational - Pompe Disease, Pompe Disease (Late-onset) | Active not recruiting | Duke University | 20 | 2019-03-25 | 2026-09-03 |
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Glycogen Storage Disease Type II (30) · Lysosomal Storage Diseases (3) · Myositis, Inclusion Body (3) · Neuromuscular Diseases (3) · Glycogen Storage Disease (2) · Spinal Muscular Atrophies of Childhood (2) · Amyotrophic Lateral Sclerosis (1) · Charcot-Marie-Tooth Disease (1) · Glycogen Storage Disease Type V (1) · Lambert-Eaton Myasthenic Syndrome (1) · Mitochondrial Myopathies (1) · Motor Neuron Disease (1) · Muscular Atrophy, Spinal (1) · Muscular Dystrophy, Duchenne (1) · Muscular Dystrophy, Facioscapulohumeral (1)
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GAA protein, human (7) · miglustat (3) · Vital Signs (2) · Walk Test (2) · Blood Specimen Collection (1) · Clenbuterol (1) · Clinical Laboratory Techniques (1) · Watchful Waiting (1) · alpha-Glucosidases (1)
Genzyme, a Sanofi Company (8) · Amicus Therapeutics (3) · Duke University (3) · Astellas Gene Therapies (2) · LMU Klinikum (2) · Spark Therapeutics, Inc. (2) · AskBio Inc (1) · Copenhagen University Hospital, Rigshospitalet (1) · Erasmus Medical Center (1) · Fondazione Don Carlo Gnocchi ETS (1) · GeneCradle Inc (1) · Huashan Hospital (1) · Iris Plug (1) · Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition (1) · University of Erlangen-Nürnberg Medical School (1)
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Stephan Wenninger, PD Dr. med. (2) · Tahseen Mozaffar, MD (2) · Chongbo Zhao, MD,PhD (1) · Dwight Koeberl (1) · Dwight Koeberl, MD, PhD (1) · Ferdinand Knieling, MD (1) · Guang Yang (1) · Nadine van der Beek, MD PHD (1) · Paolo Banfi, MD (1) · Pieter A. van Doorn, Prof. dr. (1) · Priya Kishnani, MD (1) · Regina Trollmann, MD (1)
PMID 41631150, PMID 20393176, PMID 21543987, PMID 23587901, PMID 26381077, PMID 27106274, PMID 27473031, PMID 28649546, PMID 30430231, PMID 30944902, PMID 31303277, PMID 33127291, PMID 33711277, PMID 33881155, PMID 34522820, PMID 34800400, PMID 37818166, PMID 38294575, PMID 38418563, PMID 39245687, PMID 39318718, PMID 39535661, PMID 40342075, PMID 41769220, PMID 7480122
2019 (3), 2005 (1)