Clinical Trials on Inborn Errors of Metabolism in United States
Total 3228 results
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Peloton Therapeutics, Inc., a subsidiary of Merck...National Institutes of Health (NIH)CompletedClear Cell Renal Cell Carcinoma | Von Hippel-Lindau Disease | Clear Cell RCC | ccRCC | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL | Von Hippel | Von Hippel's Disease | Von Hippel-Lindau Syndrome, Modifiers ofUnited States
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DIApedia, LLCNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedDiabetes | Plantar Ulcer | Neuropathy Causing Loss of Protective SensationUnited States
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Institute for Neurodegenerative DisordersTerminatedMultiple Sclerosis | Parkinson Disease | Alzheimer Disease | Healthy ControlsUnited States
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Institute for Neurodegenerative DisordersCompletedMultiple Sclerosis | Parkinson Disease | Alzheimer DiseaseUnited States
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Washington University School of MedicineAssociation of Frontotemporal Degeneration; Tau ConsortiumCompletedProgressive Supranuclear Palsy (PSP) | Corticobasal Degeneration (CBD) | Frontotemporal Dementia (FTD MAPT Mutation)United States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingTobacco Use Disorder | Fanconi Anemia | Lichen Planus | Oral Neoplasm | Erythroplakia | Premalignant Lesion | Oral Cavity and Lip Precancerous Condition | Oral Cavity LeukoplakiaUnited States
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Ionis Pharmaceuticals, Inc.CompletedHereditary AngioedemaUnited States, Netherlands
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ShireCompletedHereditary AngioedemaUnited States
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University of Texas at AustinRecruitingStroke | Multiple Sclerosis | Healthy | Muscular Diseases | Movement Disorders | Motor Neuron Disease | Traumatic Brain Injury | Spinal Cord Injuries | Motor DisordersUnited States
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National Institute of Neurological Disorders and...RecruitingAmyotrophic Lateral Sclerosis | Frontotemporal Dementia | Progressive Supranuclear PalsyUnited States
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FORUM Pharmaceuticals IncUnknownGranulin MutationBelgium, United States, France, Italy, Netherlands, United Kingdom
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The Bluefield Project to Cure Frontotemporal DementiaMayo Clinic; University of California, San FranciscoActive, not recruitingFrontotemporal Dementia | Frontotemporal Lobar Degeneration | FTD-GRNUnited States
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Tufts Medical CenterNational Institute on Aging (NIA)CompletedMonoclonal Gammopathy of Undetermined Significance | Smoldering Multiple MyelomaUnited States
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Ohio State University Comprehensive Cancer CenterCompletedColorectal Cancer | Lynch SyndromeUnited States
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Rhythm Pharmaceuticals, Inc.CompletedObesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor DeficiencyUnited States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
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SunovionCompletedParkinson's Disease | Off Episodes of Parkinson DiseaseUnited States, Italy
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Santhera PharmaceuticalsCompletedFriedreich's AtaxiaNetherlands, Austria, United Kingdom, Belgium, France, Germany
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Grand Valley State UniversityActive, not recruitingCerebral Palsy | Spinal Cord Injuries | Spinal Muscular Atrophy | Genetic Disease | Spina Bifida | Development DelayUnited States
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Yale UniversityRecruitingMultiple Sclerosis | Parkinson Disease | REM Sleep Behavior DisorderUnited States
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Sequenom, Inc.CompletedTurner Syndrome | Down Syndrome (Trisomy 21) | Patau Syndrome (Trisomy 13) | Edwards Syndrome (Trisomy 18)United States
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DanceStream ProjectsAlzheimer's AssociationRecruitingDementia | Alzheimer Disease | Mild Cognitive Impairment | Lewy Body Disease | Frontotemporal Dementia | Vascular DementiaUnited States
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KintoAlzheimer's AssociationCompletedDementia | Alzheimer Disease | Lewy Body Disease | Frontotemporal Dementia | Vascular Dementia | Mixed DementiaUnited States
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Mayo ClinicNational Institute on Aging (NIA)Enrolling by invitationFrontotemporal Dementia | Vascular Dementia | Alzheimer's Disease | Dementia With Lewy BodiesUnited States
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University of PennsylvaniaCompletedFrontotemporal Degeneration | Multiple System Atrophy | Progressive Supranuclear PalsyUnited States
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Harmony Biosciences, LLCActive, not recruitingPrader-Willi SyndromeUnited States
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Children's Hospital of PhiladelphiaIlera HealthcareTerminatedEpilepsy | Multiple Sclerosis | Glaucoma | Cancer | Parkinson Disease | HIV/AIDS | ALS | Autism Spectrum Disorder | Sickle Cell Disease | Spasticity, Muscle | Huntington Disease | Opioid Use | Neuropathy | Terminal Illness | Inflammatory Bowel Disease (IBD) | Intractable Pain | Ptsd | Dyskinetic SyndromeUnited States
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University of EdinburghNHS LothianRecruitingOsteogenesis ImperfectaUnited Kingdom, Ireland
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Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...RecruitingSolid Tumor | Liposarcoma | Rhabdomyosarcoma | Wilms Tumor | Malignant Rhabdoid Tumor | Yolk Sac Tumor | Liver Cell Carcinoma | Embryonal Sarcoma of the LiverUnited States
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Boston UniversityNational Institute on Minority Health and Health Disparities (NIMHD); University... and other collaboratorsRecruitingLynch Syndrome | Hereditary Breast and Ovarian CancerUnited States
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University of BirminghamThe Leeds Teaching Hospitals NHS Trust; Barts & The London NHS Trust; University... and other collaboratorsRecruitingPolycystic Ovary SyndromeUnited Kingdom
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Forest LaboratoriesCompletedCystic Fibrosis | Pancreatic InsufficiencyUnited States
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Wayne State UniversityChildren's Hospital of Philadelphia; Washington University School of Medicine; Columbia University and other collaboratorsUnknownDilated Cardiomyopathy | Hypertrophic CardiomyopathyUnited States, Canada
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Oregon Health and Science UniversityNorthWest Rehabilitations Associates; APDMUnknownStroke | Multiple Sclerosis | Parkinson Disease | Gait Disorders, Neurologic | Gait Disorder, Sensorimotor | Gait, Unsteady | Gait Ataxia | Gait, Stumbling | Gait, ShufflingUnited States
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Mayo ClinicNational Institute of Neurological Disorders and Stroke (NINDS); National Institute...CompletedFamilial Frontotemporal DementiaUnited States, Canada
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Novartis PharmaceuticalsCompletedParkinson's Disease With End of Dose Wearing OffUnited States, Puerto Rico
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Imperial College LondonUniversity College, LondonRecruitingInflammation | Obesity | Arrhythmia | Cardiomyopathy, Dilated | Sudden Cardiac DeathUnited Kingdom
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AbiliTech Medical Inc.Richard M. Schulze Family FoundationRecruitingCerebral Palsy | Muscular Dystrophies | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Spinal Muscular Atrophy | Limb Girdle Muscular Dystrophy | FSHDUnited States
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University of NottinghamNottingham University Hospitals NHS Trust; Action for A-TCompleted
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Ocera Therapeutics, Inc.CompletedHepatic Encephalopathy | Acute Episode of Overt Hepatic EncephalopathyBelgium, Germany, Italy, Spain, United States, France, Austria, Russian Federation, Czechia, Australia, Hungary, Israel, Netherlands, Bulgaria, Estonia, Denmark, New Zealand
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Novartis PharmaceuticalsCompletedSpinal and Bulbar Muscular AtrophyUnited States, Germany, Italy, Denmark
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Pure Biologics S.A.Presage BiosciencesRecruitingTriple Negative Breast Cancer | Head and Neck Squamous Cell Carcinoma | Soft Tissue Sarcoma AdultUnited States
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Towson UniversityUnknownQuality of Life | Covid19 | Parkinson Disease | Satisfaction, PatientUnited States
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Novartis PharmaceuticalsCompletedHereditary Periodic FeversBelgium, Italy, Japan, Russian Federation, Turkey, Spain, Germany, United Kingdom, France, United States, Ireland, Israel, Canada, Hungary, Netherlands, Switzerland
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National Cancer Institute (NCI)CompletedPsoriasis | Basal Cell Carcinoma | Keratosis Palmaris et PlantarisUnited States
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NovartisCompletedPain | Lung Cancer | Unspecified Adult Solid Tumor, Protocol Specific | Metastatic Cancer | Hypercalcemia of MalignancyUnited States
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Otsuka Pharmaceutical Europe LtdUnknownAutosomal Dominant Polycystic Kidney DiseaseSpain, United Kingdom, Germany, France, Belgium, Switzerland, Austria
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University of GreenwichIngesport Health Spa ConsultingNot yet recruitingQuality of Life | Obesity | Stress, Psychological | Self-Perception | Weight Loss | Happiness | Obesity, Abdominal | Muscle LossUnited Kingdom
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University of MiamiNational Parkinson FoundationCompletedParkinson's Disease | Sleep Disordered Breathing | Sleep Obstructive ApneaUnited States
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Soleno Therapeutics, Inc.Active, not recruitingPrader-Willi SyndromeUnited States, United Kingdom
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European Friedreich's Ataxia Consortium for Translational...RecruitingFriedreich's AtaxiaAustria, Germany, Italy, Spain, Belgium, Czechia, France, Greece, Ireland, United Kingdom