Clinical Trials on Inborn Error of Bile Acid Metabolism
Total 3048 results
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Children's Hospital Medical Center, CincinnatiCompletedBile Acid Synthesis Defect | Inborn Error of Bile Acid Metabolism | Inborn Error of Bile Acid ConjugationUnited States
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Mirum Pharmaceuticals, Inc.CompletedInborn Errors of Bile Acid SynthesisUnited States
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National Human Genome Research Institute (NHGRI)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic AcidemiaUnited States
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University of MiamiHRSA/Maternal and Child Health BureauCompletedPhenylketonuria | Inborn Errors of Metabolism | GalactosemiaUnited States
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National Taiwan University HospitalCompletedAromatic L-amino Acid Decarboxylase DeficiencyTaiwan
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Vitaflo International, LtdCompleted
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Vitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustCompletedPhenylketonuria | Inborn Errors of MetabolismUnited Kingdom
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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Dr. Schär AG / SPACompletedPhenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKUUnited Kingdom
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Dr. Schär AG / SPACompletedPhenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKUUnited Kingdom
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia
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King Abdullah International Medical Research CenterCompletedPropionic Acidemia | Methylmalonic AcidemiaSaudi Arabia
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Mendel TuchmanUniversity of Colorado, Denver; Children's Hospital of Philadelphia; University... and other collaboratorsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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National Human Genome Research Institute (NHGRI)RecruitingPropionic Acidemia | Metabolic Disease | Organic AcidemiaUnited States
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Nutricia ResearchCompletedInborn Errors of Metabolism | Urea Cycle DisorderUnited States
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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited States
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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National Taiwan University HospitalUnknownPropionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Taiwan
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University of PittsburghCompleted
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.Active, not recruitingMethylmalonic AcidemiaUnited States
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Krzysztof BankiewiczNational Institute of Neurological Disorders and Stroke (NINDS); University...Recruiting
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Target PharmaSolutions, Inc.Recordati Rare DiseasesEnrolling by invitationPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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Nicola LongoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National...Completed
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Recordati Rare DiseasesTarget PharmaSolutions, Inc.RecruitingPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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Liverpool University Hospitals NHS Foundation TrustRecruitingAlkaptonuria | Homogentisic Acid | Homogentisate Dioxygenase | Alkaptonuria Severity Score IndexUnited Kingdom
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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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ModernaTX, Inc.WithdrawnMetabolism, Inborn Errors | Methylmalonic Acidemia (MMA)
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Selecta Biosciences, Inc.National Human Genome Research Institute (NHGRI)SuspendedMethylmalonic Acidemia (MMA)United States
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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University Hospital, Strasbourg, FranceUnknownThe Glutathione Synthetase DeficiencyFrance
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Children's Hospital of PhiladelphiaActive, not recruitingMaple Syrup Urine DiseaseUnited States
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National Center for Research Resources (NCRR)UNC Lineberger Comprehensive Cancer CenterCompletedMetabolism, Inborn Errors | Methionine Adenosyltransferase Deficiency
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Mayo ClinicNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationCongenital Disorders of GlycosylationUnited States
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University of LiverpoolUnknown
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University of LiverpoolLiverpool University Hospitals NHS Foundation TrustCompleted
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Liverpool John Moores UniversityLiverpool University Hospitals NHS Foundation TrustNot yet recruiting
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CENTOGENE GmbH RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
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University of California, San DiegoCompleted
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Hospices Civils de LyonUnknownLiver Post-transplant PatientsFrance