- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Inborn Errors of Metabolism in United States
Total 3211 results
-
Ardea Biosciences, Inc.Completed
-
BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
-
Revive Therapeutics, Ltd.Completed
-
NYU Langone HealthGenzyme, a Sanofi CompanyCompleted
-
Baylor Research InstituteNational Institutes of Health (NIH)CompletedHyperhomocysteinemiaUnited States
-
Inozyme PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyUnited States, United Kingdom, Canada
-
Imperial College LondonRecruitingFamilial HypercholesterolemiaUnited Kingdom
-
Regeneron PharmaceuticalsSanofiCompletedHeterozygous Familial HypercholesterolemiaUnited States, Germany
-
Duke UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedGlycogen Storage Disease Type IIUnited States
-
Dyve Biosciences, Inc.Completed
-
National Human Genome Research Institute (NHGRI)Completed
-
VA New York Harbor Healthcare SystemMayo Clinic; New York UniversityUnknownOxalate, Primary Hyperoxaluria, MicrobiomeUnited States
-
Nutricia UK LtdCompletedPKU | TyrosinemiasUnited Kingdom
-
Regeneron PharmaceuticalsActive, not recruitingGeneralized LipodystrophyUnited States, Peru, Russian Federation, Turkey
-
SanofiRegeneron PharmaceuticalsCompletedHypercholesterolaemiaNetherlands, United States, Canada, Russian Federation, South Africa
-
Massachusetts General HospitalRecruiting
-
The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited States
-
BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
-
SynlogicCompletedPhenylketonuriaUnited States
-
Genzyme, a Sanofi CompanyCompletedFamilial HypercholesterolemiaNetherlands, France, Germany, Sweden, United Kingdom
-
Travere Therapeutics, Inc.Recruiting
-
PfizerTerminatedHyperlipoproteinemia Type IIICanada, United States
-
BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
-
JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IUnited States, Brazil, Japan
-
OxTheraCompletedPrimary HyperoxaluriaFrance, United Kingdom, Spain, Belgium, United States, Germany, Tunisia
-
Aegerion Pharmaceuticals, Inc.Active, not recruitingGeneralized LipodystrophyUnited States
-
Genzyme, a Sanofi CompanyWithdrawnGaucher Disease, Type 1United States, India
-
PfizerCompletedHypertriglyceridemia | Hyperlipoproteinemia Type IVUnited States, Canada
-
BioMarin PharmaceuticalCompleted
-
BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
-
Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher Disease Type 1United States
-
National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
-
Ultragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IANetherlands, United States
-
OxTheraTerminatedHyperoxaluria, PrimaryUnited Kingdom, Spain, Belgium, United States, Germany, Tunisia
-
National Institute of Diabetes and Digestive and...Completed
-
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
-
National Center for Research Resources (NCRR)Medical University of South CarolinaCompleted
-
Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
-
BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
-
Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Australia, Austria, Netherlands, Taiwan, Ukraine
-
Vitaflo International, LtdCompleted
-
APR Applied Pharma Research s.a.RecruitingPhenylketonuriasUnited Kingdom
-
Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency SyndromeUnited States, Spain, Denmark, United Kingdom, Australia
-
Cytonet GmbH & Co. KGTerminatedUrea Cycle DisordersUnited States, Canada
-
AmgenCompletedHyperlipidemiaCanada, France, Netherlands, United Kingdom, New Zealand, South Africa, Spain, United States, Australia, Germany, Sweden, Hong Kong, Switzerland, Norway
-
University of California, San FranciscoRecruiting
-
BioMarin PharmaceuticalCompleted
-
Massachusetts General HospitalActive, not recruiting
-
National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
-
National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed