Clinical Trials on Inborn Error of Bile Acid Metabolism
Total 3057 results
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Baylor College of MedicineSeattle Children's Hospital; University of California, San FranciscoCompleted
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Kaleido BiosciencesTerminatedUrea Cycle DisorderBelgium, United States, Spain, United Kingdom, Germany, Switzerland, Turkey
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HLB Cell Co., Ltd.WithdrawnUrea Cycle DisorderKorea, Republic of
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Canada
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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University of LausanneUniversity of Modena and Reggio EmiliaCompletedN-Acetylneuraminic Acid Storage DiseaseSwitzerland, Italy
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University of MilanRecruitingCOPD | Dyspnea | ArgininemiaItaly
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Manchester University NHS Foundation TrustFDNA Inc.CompletedInborn Errors of Metabolism | Dysmorphic Features | Multiple AnomaliesUnited Kingdom
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IRCCS Azienda Ospedaliero-Universitaria di BolognaUniversity of BolognaCompletedInflammation | Mortality | HyperhomocysteinemiaItaly
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde Dehydrogenase Deficiency | SSADH DeficiencyUnited States
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University of MinnesotaCompleted
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Tulane UniversityCompletedCongenital Disorders of GlycosylationUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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S.LAB (SOLOWAYS)Center of New Medical Technologies; Triangel ScientificCompletedHyperlipoproteinemias | HyperhomocysteinemiaRussian Federation
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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St George's, University of LondonNeovii BiotechWithdrawnMitochondrial Diseases | Inborn Errors of Metabolism | Metabolic Disease
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Grace Science, LLCRecruitingNGLY1 DeficiencyUnited States
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Hacettepe UniversityCompletedCaregiver Burden | Life Stress | Psychological Well-BeingTurkey
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Vitaflo International, LtdCompletedAlkaptonuria | Tyrosinemias | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type IIIUnited Kingdom
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Eva Morava-KoziczNot yet recruitingPGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of GlycosylationUnited States
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SingHealth PolyclinicsCompletedVitamin D Deficiency | Fall | Vitamin B 12 Deficiency | Folate Deficiency | Muscle Strength | HyperhomocysteinemiaSingapore
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Joanne Kurtzberg, MDAldagenWithdrawnLysosomal Storage Diseases | Inborn Errors of MetabolismUnited States
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Fondation Ophtalmologique Adolphe de RothschildNot yet recruitingAlbinism, Ocular
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University Hospital, BordeauxRecruiting
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Fondation Ophtalmologique Adolphe de RothschildCompletedAlbinism, OcularFrance
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Fondation Ophtalmologique Adolphe de RothschildCompletedAlbinism | Fovea PlanaFrance
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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Horizon Pharma Ireland, Ltd., Dublin IrelandUcyclyd Pharma, Inc.CompletedHepatic Encephalopathy | Urea Cycle DisordersUkraine
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Laboratoires GenévrierCompletedVitiligo | PiebaldismBelgium, France, Italy
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University of AarhusRecruiting
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Glycomine, Inc.Recruiting24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDGPmm2-CDGSpain, Australia, United States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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Promethera TherapeuticsCompletedUrea Cycle Disorders | Crigler Najjar SyndromeBelgium
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Eva Morava-KoziczNot yet recruitingSLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of GlycosylationUnited States
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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Netherlands Institute for Pigment DisordersAvita MedicalCompletedPiebaldism | Segmental VitiligoNetherlands
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University of West LondonCompletedVitamin B12 DeficiencyUnited Kingdom
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National Eye Institute (NEI)CompletedAlbinism | Albinism, Oculocutaneous | Albinism, OcularUnited States
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University of Wisconsin, MadisonTerminated
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National Human Genome Research Institute (NHGRI)CompletedOculocutaneous AlbinismUnited States
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde | Dehydrogenase Deficiency | DiseasesUnited States
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Academisch Medisch Centrum - Universiteit van Amsterdam...UnknownPiebaldism | Segmental VitiligoNetherlands
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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Icahn School of Medicine at Mount SinaiMayo Clinic; Baylor College of Medicine; Children's Hospital of Philadelphia; University... and other collaboratorsRecruitingCongenital Disorders of GlycosylationUnited States
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Vitaflo International, LtdUniversity College London HospitalsRecruiting