- ICH GCP
- US Clinical Trials Registry
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Search clinical trials for: Inborn errors of metabolism
Total 429908 results
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LMU KlinikumRecruitingPompe Disease (Late-onset) | FSHD | Spinal Muscular Atrophy Type 3 | Inclusion Body MyositisGermany
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Shaare Zedek Medical CenterRecruiting
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OrphalanErgomedRecruitingWilson's DiseaseSpain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
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Essentia HealthSt. Luke's Hospital of Duluth; WHITESIDE Institute for Clinical ResearchRecruitingFamilial Hypercholesterolemia | Genetic Disease | Cholesterol, ElevatedUnited States
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AlexionRecruiting
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Neurogene Inc.RecruitingNeuronal Ceroid Lipofuscinosis CLN5United States, United Kingdom
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Manchester University NHS Foundation TrustRecruitingAdrenal Hyperplasia, CongenitalUnited Kingdom
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SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
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University of British ColumbiaVancouver Coastal Health Research Institute; Genome British ColumbiaRecruitingAcute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditionsCanada
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Capital Medical UniversityRecruitingCerebral Amyloid AngiopathyChina
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Hospices Civils de LyonRecruitingPrimary Intestinal Hypocholesterolemia | Abetalipoproteinemia | Chylomicron Retention DiseaseFrance
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Institut de Myologie, FranceRecruitingMuscular Dystrophies | Mitochondrial Myopathies | Glycogen Storage Disease | Idiopathic Inflammatory Myopathies | Congenital MyopathyFrance
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Institut de Myologie, FranceRecruitingMuscular Dystrophies | Mitochondrial Myopathies | Glycogen Storage Disease | Idiopathic Inflammatory Myopathies | Congenital MyopathyFrance
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Poxel SANot yet recruiting
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University of Sao Paulo General HospitalRecruitingMicrobial Colonization | Amyloidosis, Hereditary, Transthyretin-RelatedBrazil
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Laval UniversityUniversité de Montréal; Centre de Recheche du Centre Hospitalier Université...RecruitingCardiovascular Diseases | Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy | Cholesterol; Metabolic DisorderCanada
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Kyowa Kirin Pharmaceutical Development LtdRecruitingX-Linked HypophosphatemiaFrance, Netherlands, United Kingdom
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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
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Lovisenberg Diakonale HospitalUniversity of BergenNot yet recruitingIrritable Bowel Syndrome | Functional Gastrointestinal Disorders | Sucrase Isomaltase Deficiency | Sucrose Intolerance Due to Sucrase-Isomaltase Deficiency | Carbohydrate; Malabsorption
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Poxel SANot yet recruiting
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Spruce BiosciencesRecruitingCongenital Adrenal Hyperplasia | 21-OHDUnited States
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University Children's Hospital, ZurichUniversity Children's Hospital Basel; UChildrenVienna; Insel Gruppe AG, University...RecruitingPhenylketonuriasSwitzerland
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Universitair Ziekenhuis BrusselRecruitingIrritable Bowel SyndromeBelgium
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University Hospital, RouenNot yet recruiting
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University Hospital, Strasbourg, FranceRecruiting
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Adrenas Therapeutics IncRecruitingCongenital Adrenal HyperplasiaUnited States
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University of PecsNot yet recruitingIrritable Bowel Syndrome | Lactose Intolerance | Lactose MalabsorptionHungary
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ModernaTX, Inc.RecruitingGlycogen Storage DiseaseUnited States, Canada
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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University Hospital Center of MartiniqueRecruiting
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University of Texas Southwestern Medical CenterRecruitingGlucose Metabolism Disorders | Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Glut1 Deficiency Syndrome 1 | Glut1 Deficiency Syndrome 1, Autosomal Recessive | Glucose Transporter Protein Type 1 Deficiency Syndrome | Glucose Transport DefectUnited States
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University of Erlangen-Nürnberg Medical SchoolRecruitingPompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-OnsetGermany
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University of AarhusRecruiting
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Assistance Publique - Hôpitaux de ParisCanon Medical Systems, USARecruiting
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Assistance Publique - Hôpitaux de ParisRecruitingAtherosclerosis | Familial HypercholesterolemiaFrance
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Children's Hospital of Orange CountyUltragenyx Pharmaceutical IncRecruitingInflammation | Mucopolysaccharidoses | Cardiac Disease | Carotid DiseaseUnited States
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Children's Mercy Hospital Kansas CityRecruiting
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TakedaRecruiting
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Chang Gung Memorial HospitalRecruitingFabry DiseaseTaiwan
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Vitaflo International, LtdRecruitingPhenylketonurias | PKU | Maple Syrup Urine Disease | Homocystinuria | Glutaric Acidemia I | Hereditary TyrosinemiaUnited Kingdom
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Inozyme PharmaNot yet recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 Deficiency
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Tang-Du HospitalAir Force Military Medical University, ChinaNot yet recruiting