- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Brain Diseases, Metabolic
Total 380587 results
-
BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
-
Duke UniversityWithdrawnPompe Disease (Late-onset)United States
-
Rambam Health Care CampusCompletedEnzyme Replacement Therapy in Pompe Disease
-
Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe Disease Infantile-OnsetUnited States, France, South Africa
-
Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
-
Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Spain, France, Taiwan, Italy
-
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
-
Duke UniversityCompleted
-
Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
-
BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
-
University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States
-
Rambam Health Care CampusUnknown
-
Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II | Glycogenosis 2
-
Genzyme, a Sanofi CompanyCompletedPompe DiseaseBelgium, United States, Czechia, Germany, Italy, Taiwan
-
University of FloridaAmicus TherapeuticsTerminatedPompe Disease | Hypersensitivity Reaction
-
Amicus TherapeuticsRecruitingGlycogen Storage Disease Type II Infantile OnsetUnited States, Taiwan, France, Germany, Italy, United Kingdom
-
Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Glycogenosis 2United States
-
Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Diseases | Metachromatic LeukodystrophyItaly
-
Assistance Publique - Hôpitaux de ParisShire; Zymenex A/S; European Leukodystrophy AssociationCompletedMetachromatic LeukodystrophyFrance
-
metaX Institut fuer Diatetik GmbHGreat Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Not yet recruitingAlkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
-
Hacettepe UniversityCompletedCaregiver Burden | Life Stress | Psychological Well-BeingTurkey
-
National Institute of Neurological Disorders and...Completed
-
National Taiwan University HospitalRecruiting
-
Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
-
Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
-
Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
-
Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II;Pompe's DiseaseCzechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
-
Central Hospital, Nancy, FranceRecruitingPompe's Disease Juvenile OnsetFrance
-
ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
-
Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase DeficiencyUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
-
Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II)United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
-
Roberta BrintonNational Institute on Aging (NIA); Cornell University; ADM DiagnosticsRecruitingCognitive Change | Menopause | Brain Disorder, MetabolicUnited States
-
ShirePharmaNet; Nextrials, Inc.WithdrawnLeukodystrophy, Globoid Cell
-
Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
-
National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited States
-
Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
-
National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
-
The Cooper Health SystemCompletedMetachromatic LeukodystrophyUnited States
-
Emory UniversityRecruiting
-
Vitaflo International, LtdCompletedAlkaptonuria | Tyrosinemias | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type IIIUnited Kingdom
-
Duke UniversityCompleted
-
Medical University of ViennaRecruitingPompe Disease | McArdle DiseaseAustria
-
University of LausanneUniversity of Modena and Reggio EmiliaCompletedN-Acetylneuraminic Acid Storage DiseaseSwitzerland, Italy
-
Ionis Pharmaceuticals, Inc.RecruitingPelizaeus-Merzbacher DiseaseFrance, Netherlands
-
StemCells, Inc.CompletedPelizaeus-Merzbacher DiseaseUnited States
-
Massachusetts General HospitalCompleted
-
University Hospital, RouenUnknownAnderson-Fabry DiseaseFrance
-
Passage Bio, Inc.SuspendedLeukodystrophy, Globoid CellUnited States, Brazil, Netherlands, United Kingdom, Canada, Israel
-
CENTOGENE GmbH RostockWithdrawnSplenomegaly | HepatomegalyGermany, India, Sri Lanka
-
Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II Pompe DiseaseUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom