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Clinical Trials on Genetic Diseases, Inborn
Total 380033 results
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Novartis PharmaceuticalsCompletedTransfusional HemosiderosisEgypt, Spain, United Kingdom, Jordan
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University of LiverpoolLiverpool University Hospitals NHS Foundation TrustCompleted
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San Filippo Neri General HospitalCompletedHereditary HemochromatosisItaly
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ModernaTX, Inc.RecruitingGlycogen Storage DiseaseUnited States, Canada
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Orpha LabsUnknownGlycogen Storage Disease, Type 14Turkey
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Assistance Publique - Hôpitaux de ParisRecruiting
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National Eye Institute (NEI)National Human Genome Research Institute (NHGRI)Completed
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The Cleveland ClinicNational Human Genome Research Institute (NHGRI); Indiana UniversityCompletedPregnancy Complications | Genetic Disease | Genetic Syndrome | Prenatal Disorder | Prenatal Maternal AbnormalityUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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TRPHARMKlinar CRO; Damagen Genetic Diagnostic CenterEnrolling by invitationXanthomatosis, CerebrotendinousTurkey
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Orphatech Pharmaceuticals, GmbHWithdrawnMolybdenum Cofactor Deficiency Type AAustralia
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National Center for Research Resources (NCRR)UNC Lineberger Comprehensive Cancer CenterCompletedMetabolism, Inborn Errors | Methionine Adenosyltransferase Deficiency
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Completed
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Daiichi Sankyo, Inc.CompletedHypercholesterolemia | Hyperlipidemia, Familial CombinedUnited States
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Chong Kun Dang PharmaceuticalUnknownMixed HyperlipidemiaKorea, Republic of
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PfizerCompletedHypertriglyceridemia | Hyperlipoproteinemia Type IVUnited States, Canada
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National Institute of Neurological Disorders and...CompletedLysosomal Storage DiseaseUnited States, France, Israel, Netherlands
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BiogenCompletedChoroideremiaUnited States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
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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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University of TorontoUnknownCardiovascular Risk Factor | Cholesterol; LipidosisCanada
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Kuhnil Pharmaceutical Co., Ltd.UnknownHyperlipidemia, Familial CombinedKorea, Republic of
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Assistance Publique - Hôpitaux de ParisTerminatedSelenoprotein N-related MyopathyFrance
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Professor Ernest Hung-Yu NgNot yet recruitingInfertility | Aneuploidy | Genetic Disease | Chromosomal RearrangementChina
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University Children's Hospital, ZurichRecruitingUrea Cycle DisordersSwitzerland
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Dr. Schär AG / SPACompletedPhenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKUUnited Kingdom
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University of ZurichCompletedGlycogen Storage Disease Type 1 (GSD 1)Switzerland
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National Institute of Arthritis and Musculoskeletal...RecruitingSkin Diseases | Skin Diseases, Genetic | Dermatitis | Eczema | Dermatitis, AtopicUnited States
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Eli Lilly and CompanyCompletedSkin Diseases | Skin Diseases, Genetic | Dermatitis | Eczema | Dermatitis, AtopicJapan
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Organon and CoCompletedHeart Disease | Lipid Metabolism, Inborn Errors
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Organon and CoCompletedHeart Disease | Lipid Metabolism, Inborn Errors
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4D Molecular TherapeuticsActive, not recruitingChoroideremiaUnited States
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4D Molecular TherapeuticsTerminated
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University of OxfordUniversity College, London; Moorfields Eye Hospital NHS Foundation TrustCompleted
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STZ eyetrialUniversity Hospital TuebingenCompleted
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Jerry Vockley, MD, PhDAcer Therapeutics Inc.RecruitingMedium-chain Acyl-CoA Dehydrogenase DeficiencyUnited States
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University of California, San DiegoCompleted
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Cerenis Therapeutics, SATerminatedFamilial HypoalphalipoproteinemiaFrance, United States, Belgium, Canada, Israel, Italy, Netherlands
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Sanquin Research & Blood Bank DivisionsRadboud University Medical Center; Maastricht University Medical Center; Atrium... and other collaboratorsUnknownHereditary HemochromatosisNetherlands
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Stealth BioTherapeutics Inc.CompletedBarth SyndromeUnited States
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Rigshospitalet, DenmarkCompletedGlycogen Storage Disease Type IIIDenmark
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Interregionale Blutspende SRKEnrolling by invitationHereditary Hemochromatosis | HyperferritinemiaSwitzerland
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Leadiant Biosciences Ltd.CompletedCerebrotendinous Xanthomatoses
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Dr. Schär AG / SPACompletedPhenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKUUnited Kingdom
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Medical University of South CarolinaCompletedActinic PorokeratosisUnited States
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University Hospital, Clermont-FerrandUnknown
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University Hospital TuebingenRecruitingRare Diseases | Genetic PredispositionGermany
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National Institute of Arthritis and Musculoskeletal...CompletedIchthyosis | Ichthyosis, X-Linked | Ichthyosis, Lamellar | Darier Disease | Hailey-Hailey Disease | Hyperkeratosis, Epidermolytic | KeratodermaUnited States
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The University of Hong KongThe Hong Kong Polytechnic UniversityActive, not recruitingEczema | Atopic Dermatitis | Genetic Skin DiseaseHong Kong
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University of MiamiHRSA/Maternal and Child Health BureauCompletedPhenylketonuria | Inborn Errors of Metabolism | GalactosemiaUnited States