- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, Inborn in United States
Total 147152 results
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Baylor College of MedicineSeattle Children's Hospital; University of California, San FranciscoCompleted
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Johns Hopkins UniversityClark Charitable Foundation Inc.CompletedOcular Albinism (OA) | Oculocutaneous Albinism (OCA)United States
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Mayo ClinicCompleted
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oxalosis...Completed
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Lysosomal and Rare Disorders Research and Treatment...UnknownLysosomal Storage DiseasesUnited States
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Northwestern UniversityAnn & Robert H Lurie Children's Hospital of ChicagoCompleted
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Ulthera, IncCompletedSkin Laxity | Skin CrepinessUnited States
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Oregon Health and Science UniversityUniversity of Oregon; Oregon State UniversityRecruitingCarbohydrate IntoleranceUnited States
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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UniQure Biopharma B.V.Chiesi Farmaceutici S.p.A.Withdrawn
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Emory UniversityPatient-Centered Outcomes Research InstituteCompleted
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Massachusetts General HospitalAlexion PharmaceuticalsUnknownCholesterol Ester Storage Disease | Lysosomal Acid Lipase DeficiencyUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Netherlands
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OxTheraCompletedPrimary HyperoxaluriaUnited States
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National Heart, Lung, and Blood Institute (NHLBI)Completed
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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Shasa HuBerg, LLCCompletedEpidermolysis BullosaUnited States
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Masonic Cancer Center, University of MinnesotaTerminatedEpidermolysis BullosaUnited States
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The Rogosin InstituteRecruitingHomozygous Familial HypercholesterolemiaUnited States
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National Heart, Lung, and Blood Institute (NHLBI)Completed
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Children's National Research InstitutePatient-Centered Outcomes Research Institute; Eunice Kennedy Shriver National... and other collaboratorsCompleted
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National Institute of Neurological Disorders and...National Institute of Neurological Disorders and Stroke@@@Shire Human Genetic...Completed
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Children's Hospital of PhiladelphiaActive, not recruitingMaple Syrup Urine DiseaseUnited States
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Rutgers, The State University of New JerseyCompleted
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RHEACELL GmbH & Co. KGRecruitingEpidermolysis BullosaUnited States
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Masonic Cancer Center, University of MinnesotaCompletedEpidermolysis BullosaUnited States
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Masonic Cancer Center, University of MinnesotaCompletedEpidermolysis BullosaUnited States
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Masonic Cancer Center, University of MinnesotaCompletedEpidermolysis BullosaUnited States
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BTL Industries Ltd.Completed
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Eva Morava-KoziczNot yet recruitingPGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of GlycosylationUnited States
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Dermelix Biotherapeutics, LLC.Withdrawn
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Monica E. KleinmanSchering-PloughCompletedProgeria | Hutchinson-Gilford SyndromeUnited States
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SanofiRecruiting
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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Columbia UniversityWithdrawnGlycogen Storage Disease Type IIUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Spain, Italy
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Amicus TherapeuticsActive, not recruitingBatten Disease | CLN6United States
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Kedrion S.p.A.CompletedLigneous ConjunctivitisUnited States, Italy
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University of California, Los AngelesEnrolling by invitationTophaceous GoutUnited States
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National Eye Institute (NEI)Completed
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Boston Children's HospitalDana-Farber Cancer Institute; Brigham and Women's Hospital; Schering-PloughCompletedProgeria | Hutchinson-Gilford SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...UnknownChromosome DisordersUnited States
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Murdoch Childrens Research InstituteNorthwestern University; United Christian Hospital; Australian Wool Innovation...RecruitingDermatitis, AtopicUnited States
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Fresh Tracks Therapeutics, Inc.CompletedDermatitis, AtopicUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom