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Clinical Trials on Inherited Metabolic Storage Diseases
Total 61973 results
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Spain, France, Taiwan, Italy
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Duke UniversityCompleted
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
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McMaster UniversityNot yet recruitingObesity | Lysosomal Storage Diseases | Muscle Loss | Glycogen Storage Disease Type II | Pompe Disease | Nutrition Poor | Glycogen Storage Disease Type II, Adult | Glycogen Storage Disease Type II Late Onset
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University of ManitobaCo-Investigator - Dr. Cheryl Rockman-GreenbergCompletedHypoglycemia | Glycogen Storage Disorder Type 1 | Cornstarch | GlycosadeCanada
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Duke UniversityGenzyme, a Sanofi CompanyCompletedNeuropathy | Myopathy | Glycogen Storage Disease Type II (Late-onset Pompe Disease)United States
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Genzyme, a Sanofi CompanyCompletedPompe DiseaseBelgium, United States, Czechia, Germany, Italy, Taiwan
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University of FloridaAmicus TherapeuticsTerminatedPompe Disease | Hypersensitivity Reaction
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Glycogenosis 2United States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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GeneCradle IncRecruitingPompe Disease (Late-onset)China
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GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
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Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
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Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknownPompe Disease | Late Onset Pompe DiseaseSpain
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Canada, Australia
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Huashan HospitalNot yet recruitingPompe Disease (Late-onset)China
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Rambam Health Care CampusRecruitingPompe Disease Infantile-OnsetIsrael
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, United Kingdom
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Amicus TherapeuticsRecruitingPompe Disease (Late-onset)United States, Japan, Australia, Taiwan, Canada
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Genzyme, a Sanofi CompanyRecruitingGlycogen Storage Disease Type II | Pompe DiseaseHungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
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Duke UniversityWithdrawnPompe Disease (Late-onset)United States
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Rambam Health Care CampusCompletedEnzyme Replacement Therapy in Pompe Disease
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe Disease Infantile-OnsetUnited States, France, South Africa
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States
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Rambam Health Care CampusUnknown
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II | Glycogenosis 2
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)United States, France, Canada, Netherlands, Australia
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University of LausanneUniversity of Modena and Reggio EmiliaCompletedN-Acetylneuraminic Acid Storage DiseaseSwitzerland, Italy
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States, Israel, France, United Kingdom
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Genzyme, a Sanofi CompanyTerminatedGlycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)United States, Taiwan, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)United States, Netherlands, United Kingdom, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)France
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Novartis PharmaceuticalsWithdrawnInherited Metabolic Disorders IMD
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University Children's Hospital, ZurichSanofiCompleted
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Central Hospital, Nancy, FranceRecruitingPompe's Disease Juvenile OnsetFrance
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University Hospital, RouenUniversity Hospital, CaenRecruitingLysosomal Storage Diseases | Neonatal ScreeningFrance
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Duke UniversityCompleted
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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Joanne Kurtzberg, MDAldagenWithdrawnLysosomal Storage Diseases | Inborn Errors of MetabolismUnited States
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Children's Hospital Medical Center, CincinnatiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedWolman Disease | Cholesterol Ester Storage Disease | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2United States
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BioMarin PharmaceuticalTerminatedLate-onset Pompe Patients Untreated or Treated With rhGAASpain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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Rigshospitalet, DenmarkCompleted
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Eunice Kennedy Shriver National Institute of Child...RecruitingLysosomal Storage Disease | Cholesterol MetabolismUnited States