9 completed. 9 Phase 2, 6 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 9 | Completed 9 |
| Phase 1 | 6 | Completed 6 |
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 |
|---|---|---|---|---|---|---|
| First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 ATB200, AT2221 - Pompe Disease | Phase 1 / Phase 2 | Completed | Amicus Therapeutics | 29 | 2016-04-01 | 2025-10-23 |
| Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy Clenbuterol, Placebo - Pompe Disease | Phase 1 / Phase 2 | Completed | Dwight Koeberl, M.D., Ph.D. | 17 | 2013-09-01 | 2019-07-02 |
| Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease Albuterol, Placebo - Pompe Disease | Phase 1 / Phase 2 | Completed | Duke University | 16 | 2013-06-01 | 2019-07-15 |
| Drug-drug Interaction Study duvoglustat, rhGAA - Pompe Disease | Phase 2 | Completed | Amicus Therapeutics | 25 | 2011-10-31 | 2025-06-26 |
| Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease BMN 701 - Pompe Disease | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 22 | 2011-01-17 | 2018-06-11 |
| Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease rAAV1-CMV-GAA (study agent) Administration, RMST - Pompe Disease | Phase 1 / Phase 2 | Completed | University of Florida | 9 | 2010-09-01 | 2018-09-14 |
| rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) Myozyme - Glycogen Storage Disease Type II, Pompe Disease | Phase 1 / Phase 2 | Completed | Genzyme, a Sanofi Company | 20 | 2003-02-01 | 2014-02-05 |
| A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Alglucosidase alfa - Glycogen Storage Disease Type II, Pompe Disease | Phase 2 | Completed | Genzyme, a Sanofi Company | 2 | 2003-01-01 | 2014-02-05 |
| Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease recombinant human acid alpha-glucosidase (rhGAA) - Pompe Disease, Glycogen Storage Disease Type II | Phase 2 | Completed | Genzyme, a Sanofi Company | 8 | 2001-05-01 | 2014-11-13 |
Glycogen Storage Disease Type II (9) · Glycogen Storage Disease (1)
Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Carbohydrate Metabolism, Inborn Errors (9) · Central Nervous System Diseases (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Lysosomal Storage Diseases (9) · Lysosomal Storage Diseases, Nervous System (9) · Metabolic Diseases (9) · Metabolism, Inborn Errors (9) · Nervous System Diseases (9) · Nutritional and Metabolic Diseases (9) · Glycogen Storage Disease (8)
GAA protein, human (4) · 1-Deoxynojirimycin (1) · Albuterol (1) · Capsules (1) · Clenbuterol (1) · Genetic Engineering (1) · Organization and Administration (1) · Pharmaceutical Preparations (1) · miglustat (1)
Genzyme, a Sanofi Company (3) · Amicus Therapeutics (2) · BioMarin Pharmaceutical (1) · Duke University (1) · Dwight Koeberl, M.D., Ph.D. (1) · University of Florida (1)
United States (9) · United Kingdom (4) · France (3) · Australia (2) · Germany (2) · Canada (1) · Israel (1) · Netherlands (1) · New Zealand (1)
Barry J Byrne, MD, PhD (1) · Dwight D Koeberl, MD, PhD (1) · Shelley Collins, MD (1)
PMID 19775921, PMID 22794786, PMID 23570273, PMID 30025991, PMID 31839530, PMID 38057636, PMID 40920287, PMID 41631150
P01HL059412 (1)