- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Inborn Errors of Metabolism in United States
Total 3211 results
-
Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemia | Heterozygous Familial HypercholesterolemiaUnited States, Canada
-
Mirum Pharmaceuticals, Inc.CompletedInborn Errors of Bile Acid SynthesisUnited States
-
PfizerTerminated
-
University of Colorado, DenverCompletedHomocystinuriaUnited States
-
Pfizer's Upjohn has merged with Mylan to form Viatris...CompletedFamilial HypercholesterolemiaUnited States, Spain, Turkey, Canada, Belgium, Germany, Greece, Hungary, Italy, Norway, Poland, Puerto Rico, Russian Federation, Slovakia, Switzerland
-
BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States, Turkey, Canada, France, Germany, Italy, United Kingdom
-
Rigshospitalet, DenmarkUniversity College, LondonCompletedMcArdle DiseaseDenmark, United Kingdom
-
Reneo Pharma LtdCompletedMcArdle DiseaseSpain, United Kingdom
-
University Research Foundation for Lysosomal Storage...University of PittsburghCompletedGaucher DiseaseUnited States
-
SanofiRegeneron PharmaceuticalsCompletedHypercholesterolemiaUnited States, Austria, Canada, Czech Republic, Denmark, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden, United Kingdom
-
National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
-
Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
-
Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
-
JCR Pharmaceuticals Co., Ltd.CompletedMucopolysaccharidosis IUnited States, Brazil, Japan
-
Prevail TherapeuticsEli Lilly and CompanyActive, not recruitingGaucher Disease, Type 2United States, United Kingdom
-
StemCells, Inc.CompletedNeuronal Ceroid LipofuscinosisUnited States
-
PfizerCompletedGaucher DiseaseUnited States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy
-
Sangamo TherapeuticsTerminated
-
PATHFred Hutchinson Cancer CenterCompletedG6PD DeficiencyUnited States
-
PATHCompletedG6PD DeficiencyUnited States
-
Nationwide Children's HospitalSanfilippo Children's Research Foundation; The Sanfilippo Research Foundation; The Children's Medical Research FoundationCompletedMucopolysaccharidosis Type IIIA | Mucopolysaccharidosis Type IIIBUnited States
-
Birmingham Women's and Children's NHS Foundation...Active, not recruiting
-
Amicus TherapeuticsCompleted
-
University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
-
University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
-
University of FloridaBioMarin PharmaceuticalCompleted
-
University of FloridaAmicus TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type II | Pompe DiseaseUnited States
-
Diurnal LimitedCompletedCongenital Adrenal HyperplasiaUnited States
-
BioMarin PharmaceuticalCompletedStudy of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006PhenylketonuriaUnited States
-
Novartis PharmaceuticalsActive, not recruitingFamilial Hypercholesterolemia - HomozygousGreece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
-
Inozyme PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyUnited States, United Kingdom
-
Dartmouth-Hitchcock Medical CenterCelgene CorporationWithdrawn
-
CymaBay Therapeutics, Inc.CompletedHyperuricemia | GoutUnited States
-
PharmosUnknownHyperuricemia | GoutUnited States
-
Vitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustRecruiting
-
Abeona Therapeutics, IncTerminatedMucopolysaccharidosis III-BUnited States, Germany, France
-
Guy's and St Thomas' NHS Foundation TrustUnknownCardiovascular Diseases | PhenylketonuriasUnited Kingdom
-
PfizerCompletedHyperlipidemia | Hypercholesterolemia, FamilialUnited States, Canada, South Africa, Czech Republic, Italy, Finland, Netherlands, France
-
Novartis PharmaceuticalsCompletedTransfusional HemosiderosisUnited States
-
ShionogiIntegriumCompletedCombined HyperlipidemiaUnited States
-
University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
-
Boston Children's HospitalEnrolling by invitation
-
Lysosomal and Rare Disorders Research and Treatment...Team SanfilippoRecruitingSanfilippo Syndrome | MPS3United States
-
University of MinnesotaCompleted
-
Columbia UniversityNew York Blood CenterCompleted
-
Ajinomoto Co., Inc.Great Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Active, not recruiting
-
PfizerCompletedGaucher DiseaseUnited Kingdom, Canada, Israel, United States, Australia, Spain
-
Homology Medicines, IncTerminated