- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Metabolism, Inborn Errors in New Zealand
Total 7160 results
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Tulane University School of MedicineBioMarin PharmaceuticalCompletedPhenylketonuria | Behavior and Behavior Mechanisms | PAH Gene ExpressionUnited States
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La Jolla Pharmaceutical CompanyPRA Health SciencesCompletedHereditary HemochromatosisUnited States, Australia, France, United Kingdom
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Joyce FoxUltragenyx Pharmaceutical IncNo longer availableMucopolysaccharidosis Type 7United States
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Neurogene Inc.RecruitingCeroid Lipofuscinosis, Neuronal 5United States
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BioMarin PharmaceuticalCompletedStudy of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006PhenylketonuriaUnited States
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Alexion Pharmaceuticals, Inc.Enrolling by invitationHypophosphatasia (HPP)United States, Italy, Russian Federation, Spain, United Kingdom, Germany, Canada, France, Saudi Arabia, Poland, Australia
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BioMarin PharmaceuticalActive, not recruiting
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AmgenCompletedHomozygous Familial Hypercholesterolemia HoFHIndia
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Columbia UniversityWithdrawnGlycogen Storage Disease Type IIUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Cortria CorporationMontreal Heart InstituteCompletedHypertriglyceridemia | Mixed HyperlipidemiaCanada
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Exsar CorporationSuspendedType I Gaucher DiseaseUnited States
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NYU Langone HealthGenzyme, a Sanofi CompanyCompleted
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Massachusetts General HospitalNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Recruiting
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BioMarin PharmaceuticalApproved for marketing
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BioMarin PharmaceuticalCompleted
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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Novartis PharmaceuticalsCompletedIron Overload | Hereditary HemochromatosisUnited States, Germany, Italy, Australia, Canada, France
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Horizon Therapeutics Ireland DACCompleted
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VA New York Harbor Healthcare SystemMayo Clinic; New York UniversityUnknownOxalate, Primary Hyperoxaluria, MicrobiomeUnited States
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AstraZenecaCompletedFamilial HypercholesterolemiaUnited States, Spain, Netherlands, Norway, Canada
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Ardea Biosciences, Inc.CompletedGoutUnited States, Canada, Australia, New Zealand, Switzerland, Poland
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Regeneron PharmaceuticalsCompleted
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QOL Medical, LLCRecruitingCongenital Sucrase-Isomaltase Deficiency | CSID | Sucrase Isomaltase DeficiencyUnited States
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Ardea Biosciences, Inc.CompletedGoutUnited States, Canada, South Africa, New Zealand, Belgium, Australia, Germany
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Genzyme, a Sanofi CompanyWithdrawnGaucher Disease, Type 1United States, India
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Regeneron PharmaceuticalsTerminated
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaItaly, Spain, Israel, Korea, Republic of, Belgium, Germany, United States, China, France, Taiwan, Austria, Japan, India, Australia, Argentina, Poland, Turkey, Brazil, Canada, Thailand, United Kingdom, Slovakia, Czechia
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AmgenCompletedGout | Chronic Uncontrolled Gout | Uncontrolled GoutUnited States
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Australia, Austria, Canada, France, Greece, Italy, Japan, South Africa, Ukraine
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CymaBay Therapeutics, Inc.CompletedGoutUnited States, Canada, Georgia
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BioCryst PharmaceuticalsCompleted
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ShionogiIntegriumCompletedCombined HyperlipidemiaUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Ari ZimranPfizerActive, not recruitingGaucher Disease, Type 3Israel, Turkey, India
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Merck Sharp & Dohme LLCCompletedHypercholesterolemia | Familial HypercholesterolemiaUnited States, Germany, Japan, Korea, Republic of, Mexico, Norway, Turkey, United Kingdom
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Columbia UniversityNew York Blood CenterCompleted
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Dartmouth-Hitchcock Medical CenterCelgene CorporationWithdrawn
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Cytonet GmbH & Co. KGTerminatedUrea Cycle DisordersUnited States, Canada
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Aegerion Pharmaceuticals, Inc.Active, not recruitingGeneralized LipodystrophyUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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NovartisCompletedArthritis, GoutyUnited States, Switzerland, United Kingdom
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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SynlogicCompletedPhenylketonuriaUnited States
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University of RochesterBatten Disease Support and Research Assocation (BDSRA)CompletedJuvenile Neuronal Ceroid LipofuscinosisUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States