25 completed, 4 recruiting, 4 terminated, 2 active not recruiting. 12 Phase 4, 11 Phase 2, 6 Phase 3, 3 Phase 1, 1 Not applicable.
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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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 | |
| 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 | |
| Higher Dose of Alglucosidase Alpha for Pompe Disease Alglucosidase Alfa - Glycogen Storage Disease Type II | Not yet recruiting | Taipei Veterans General Hospital, Taiwan | 36 | 2021-09-01 | 2021-08-23 | |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) Alglucosidase alfa GZ419829 - Glycogen Storage Disease Type II | Active not recruiting | Sanofi | 16 | 2021-06-10 | 2026-06-24 | |
| Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen ALGLUCOSIDASE ALFA - Pompe's Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 41 | 2021-03-10 | 2025-02-10 |
| Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment ALGLUCOSIDASE ALFA (MYOZYME) - Glycogen Storage Disease Type II | Phase 4 | Completed | Genzyme, a Sanofi Company | 10 | 2018-12-04 | 2022-04-13 |
| A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 125 | 2018-12-04 | 2025-09-11 |
| A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829) - Glycogen Storage Disease Type II-Pompe's Disease | Phase 2 | Active not recruiting | Genzyme, a Sanofi Company | 22 | 2017-10-12 | 2026-01-30 |
| VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease VAL-1221, RhGAA - Pompe Disease | Phase 1 / Phase 2 | Terminated | Valerion Therapeutics, LLC | 12 | 2017-06-21 | 2020-06-02 |
| Search for Serum/Plasma Biomarkers in Pompe's Disease Enzyme Replacement Agent - Late Onset Pompe Disease, Pompe Disease | Unknown | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 35 | 2017-04-01 | 2017-03-20 | |
| Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829) - Glycogen Storage Disease Type II;Pompe's Disease | Phase 3 | Completed | Genzyme, a Sanofi Company | 101 | 2016-11-02 | 2024-04-04 |
| Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study) glucosidase alfa - Pompe Disease | Completed | LMU Klinikum | 112 | 2016-06-01 | 2021-02-10 | |
| Alglucosidase Alfa Pompe Safety Sub-Registry alglucosidase alfa - Pompe Disease | Completed | Genzyme, a Sanofi Company | 110 | 2015-03-20 | 2021-07-13 | |
| Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease alglucosidase alfa - Pompe Disease, Glycogen Storage Disease Type II (GSD II) | Phase 4 | Completed | Genzyme, a Sanofi Company | 21 | 2014-11-03 | 2022-03-28 |
| A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease alglucosidase alfa - Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II) | Phase 4 | Terminated | Genzyme, a Sanofi Company | 4 | 2012-08-01 | 2016-01-18 |
| Pompe Lactation Sub-Registry alglucosidase alfa - Glycogen Storage Disease, Pompe Disease | Completed | Genzyme, a Sanofi Company | 2 | 2012-03-31 | 2024-03-19 | |
| A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease Alglucosidase alfa - Pompe Disease | Phase 4 | Terminated | Genzyme, a Sanofi Company | 113 | 2012-03-01 | 2015-12-08 |
| Drug-drug Interaction Study duvoglustat, rhGAA - Pompe Disease | Phase 2 | Completed | Amicus Therapeutics | 25 | 2011-10-31 | 2025-06-26 |
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Glycogen Storage Disease Type II (35) · Glycogen Storage Disease (3) · Anaphylaxis (1) · Gaucher Disease (1) · Heartburn (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
Genzyme, a Sanofi Company (29) · Amicus Therapeutics (3) · Sanofi (2) · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau (1) · LMU Klinikum (1) · Taipei Veterans General Hospital, Taiwan (1) · University of California, San Francisco (1) · Valerion Therapeutics, LLC (1)
United States (30) · France (12) · United Kingdom (11) · Germany (9) · Netherlands (9) · Taiwan (9) · Italy (7) · Belgium (5) · Canada (5) · Australia (4) · Israel (4) · Spain (4) · Austria (3) · China (3) · Denmark (3)
Benedikt Schoser, MD (1) · Hal Landy, MD (1) · Tippi MacKenzie, MD (1)
PMID 19775921, PMID 15324318, PMID 19948615, PMID 20393176, PMID 21543987, PMID 21637107, PMID 23510062, PMID 27473031, PMID 28374935, PMID 28657663, PMID 29565424, PMID 30337566, PMID 31086307, PMID 32102934, PMID 34420548, PMID 34800399, PMID 34800400, PMID 35833019, PMID 36214004, PMID 36542086, PMID 37036722, PMID 37559825, PMID 37705424, PMID 37955580, PMID 38294575, PMID 39318718, PMID 39535661, PMID 39891377, PMID 40342075, PMID 40449831, PMID 40817977, PMID 41631150, PMID 9918480
2016 (2), 2005 (1), 2015 (1)