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Clinical Trials on Urea Cycle Disorders, Inborn
Total 7103 results
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedHigh-Frequency Microsatellite Instability | Mismatch Repair Gene Mutation | Mutation-Negative Lynch Syndrome | Mutation-Positive Lynch SyndromeUnited States
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Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsCompletedDiGeorge Syndrome | DiGeorge Anomaly | Complete DiGeorge Anomaly | Complete DiGeorge SyndromeUnited States
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University Hospital, Strasbourg, FranceCompletedCystic Fibrosis | Insulin-dependent Diabetes | Diabetes Related Cystic FibrosisFrance
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Novartis PharmaceuticalsCompletedFamilial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic SyndromesGermany, France, Belgium, Canada, Spain, Switzerland, United Kingdom
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Johann Wolfgang Goethe University HospitalUnknownAtaxia TelangiectasiaGermany
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University Hospital, GhentCompletedPortal Hypertension, Noncirrhotic | Cystic Fibrosis Liver Disease | Portal Hypertension Due to Cystic Fibrosis (Disorder)
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Maxima Medical CenterRecruitingChronic Pain Syndrome | Diagnosis | Nerve Entrapment Syndrome | Anterior Cutaneous Nerve Entrapment SyndromeNetherlands
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Matrix Biomed, Inc.The University of Texas Health Science Center, HoustonNot yet recruitingAtaxia Telangiectasia | Ataxia Telangiectasia Louis-Bar | Ataxia Telangiectasia in ChildrenUnited States
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Merve KurtRecruitingDuchenne Muscular Dystrophy | Virtual Reality | Spinal Muscular Atrophy | Neuromuscular Disease | BiofeedbackTurkey
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Quince Therapeutics S.p.A.CompletedGenetic Syndrome | Nervous System DiseaseIndia, United States, Australia, Germany, Italy, Norway, Poland, Spain, Tunisia, United Kingdom, Israel, Belgium
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University of ChicagoPfizer; Biogen; APDM Wearable TechnologiesActive, not recruitingSpinocerebellar Ataxia Type 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
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Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
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Columbia UniversityMuscular Dystrophy Association; Stevens Institute of TechnologyCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy Type 3United States
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Roger PackerWithdrawnNeurofibromatosis Type 1 | Tumors of the Central Nervous SystemUnited States
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Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8United States, China
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University of PittsburghCompletedPulmonary Hypertension | Friedreich Ataxia | Iron-sulfur Cluster DeficiencyUnited States
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Georgetown UniversityVirginia Commonwealth University; Cornell UniversityCompletedHereditary Breast and Ovarian CancerUnited States
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Dana-Farber Cancer InstituteNational Cancer Institute (NCI); Wyeth is now a wholly owned subsidiary of... and other collaboratorsCompletedTuberous Sclerosis | Nonmalignant Neoplasm | Lymphangioleimyomatosis | Kidney AngiomyolipomaUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsApproved for marketingDiGeorge Syndrome | DiGeorge Anomaly | Complete DiGeorge Anomaly | Complete DiGeorge SyndromeUnited States
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Laval UniversityTerminatedLung Diseases | Chronic Obstructive Pulmonary Disease | Cystic Fibrosis | Pulmonary Hypertension | Interstitial Lung Disease | DyspneaCanada
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Children's National Research InstituteActive, not recruitingNeurofibromatosis 1 | Neurofibroma | Plexiform Neurofibroma | Atypical Neurofibroma | Atypical Neurofibromatosis | Von Recklinghausen DiseaseUnited States
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Gaziosmanpasa Research and Education HospitalCompletedCOVID-19 | Cerebral Palsy | Muscular Dystrophies | Disabilities MultipleTurkey
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Novartis PharmaceuticalsCompletedFamilial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic SyndromesJapan
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Charite University, Berlin, GermanyCompletedDepression | Migraine | Asthma | Allergic Rhinitis | Headache | Atopic EczemaGermany
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University of Southern CaliforniaStanford UniversityCompletedHereditary Breast and Ovarian CancerUnited States
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Novartis PharmaceuticalsCompletedFamilial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic SyndromesGermany, France, Belgium, Canada, Spain, Switzerland, United Kingdom
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Novartis PharmaceuticalsTerminatedPlexiform Neurofibroma Associated With Neurofibromatosis Type 1Israel
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Pontificia Universidade Catolica de Sao PauloCompletedEar Malformations in the Velocardiofacial Syndrome
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University of California, San FranciscoNational Institute on Aging (NIA)CompletedDepression | Stress, Psychological | Dementia | Dementia, Vascular | Loneliness | Self Efficacy | Dementia, Mixed | Health Care Utilization | Dementia Alzheimers | Dementia of Alzheimer Type | Social Isolation | Dementia, Multi-Infarct | Dementia, Lewy Body | Dementia in Parkinsons Disease | Dementia Frontal | Dementia... and other conditionsUnited States
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National Institute of Diabetes and Digestive and...RecruitingCystic Fibrosis | Turner Syndrome | Congenital Hepatic Fibrosis | Immunologic Deficiency Syndrome | Idiopathic Non-Cirrhotic Portal HypertensionUnited States
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Diane GeorgeActive, not recruitingSickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditionsUnited States
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Boston Children's HospitalNational Eye Institute (NEI); Howard Hughes Medical InstituteRecruitingOcular Motility Disorders | Facial Palsy | Synkinesis | Sixth Nerve Palsy | Mobius Syndrome | Congenital Fibrosis of Extraocular Muscles | Duane Retraction Syndrome | Duane Radial Ray Syndrome | Brown Syndrome | Marcus Gunn Syndrome | Strabismus Congenital | Horizontal Gaze Palsy | Horizontal Gaze Palsy With Progressive... and other conditionsUnited States
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Massachusetts Eye and Ear InfirmaryNo longer availableNeurofibromatosis Type 2 | Severe Profound Sensorineural Hearing LossUnited States
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Burke Medical Research InstituteActive, not recruitingStroke | Multiple Sclerosis | Cerebral Palsy | Parkinson Disease | Spinal Cord Injuries | Neurologic Disorder | Hemiplegia | Hemiparesis | Friedreich Ataxia | Trauma, Brain | Transverse MyelitisUnited States
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RWTH Aachen UniversityDLR German Aerospace CenterRecruitingPostural Tachycardia Syndrome | Autonomic Neuropathy | Small Fiber Neuropathy | Ehlers-Danlos Syndrome Hypermobility TypeGermany
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Perouse MedicalEclevar MedtechCompletedPeripheral Arterial Disease | Marfan Syndrome | Ehlers-Danlos Syndrome | Aneurysm Arterial | Arterial DissectionsFrance
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Oslo University HospitalEnrolling by invitationScoliosis | Scoliosis; Juvenile | Scoliosis; Idiopathic, Infantile | Scoliosis Neuromuscular | Scoliosis in Neurofibromatosis | Scoliosis; Congenital, Postural | Scoliosis in Skeletal Dysplasia | Scoliosis Associated With Other Conditions | Scoliosis; Congenital, Due to Bony MalformationNorway
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Medical University of WarsawCompletedLiver Diseases | Liver Failure | Graves Ophthalmopathy | Graves Disease | Acute Liver Failure | Acute Liver Injury | Liver Dysfunction | Glucocorticoids Toxicity | Liver Injury | Liver Insufficiency | Acute Liver Injury, Drug Induced | Methylprednisolone Adverse Reaction | Dysthyroid Orbitopathy | Dysthyroid Ophthalmopathy
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Implandata Ophthalmic Products GmbHCompletedCongenital Glaucoma | Stevens-Johnson Syndrome | Graft vs Host Disease | Congenital Aniridia | Chemical BurnsGermany
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National Human Genome Research Institute (NHGRI)CompletedMarfan Syndrome | Connective Tissue Disease | Ehlers Danlos Syndrome | Dissecting Aneurysm | Nail Patella SyndromeUnited States
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National Cancer Institute (NCI)CompletedOsteosarcoma | Acute Lymphoblastic Leukemia | Ewing Sarcoma | Neurofibromatosis Type 1 | NeuroblastomaUnited States
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Paul SzabolcsRecruitingCongenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)United States
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University of ValenciaHospital Universitario La Fe; Hospital General Universitario de Valencia; Hospital...CompletedAsthma | Type 1 Diabetes | Allergic Rhinitis | Cystic Fibrosis | Pulmonary Disease | Atopic Dermatitis | Food Allergy | Allergic Asthma | Rhinoconjunctivitis | Primary Ciliary Dyskinesia | Short StatureSpain
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Rambam Health Care CampusRecruitingBronchiectasis | Cystic Fibrosis | Bronchiolitis Obliterans | Primary Ciliary Dyskinesia | Primary Immune DeficiencyIsrael
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VA Office of Research and DevelopmentNot yet recruitingAmyotrophic Lateral Sclerosis | Muscular Dystrophy | Spinal Cord Injury | Brain Stem Infarctions | Locked-in SyndromeUnited States
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University of Texas Southwestern Medical CenterRecruitingDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Dilated Cardiomyopathy | Cardiomyopathy, Hypertrophic | Heart Failure With Reduced Ejection Fraction | Cardiac Sarcoidosis | Heart Failure With Preserved Ejection FractionUnited States
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Andres SchanzerRecruitingMarfan Syndrome | Ehlers-Danlos Syndrome | Aortic Arch Aneurysm | Complex Aortic Aneurysms | Juxtarenal Aneurysms | Loeys-Dietz Syndrome | Thoracoabdominal Aneurysms | Pararenal AneurysmsUnited States
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University Health Network, TorontoSunnybrook Health Sciences Centre; University of Toronto; Hamilton Health Sciences... and other collaboratorsActive, not recruitingUniversal Screening for Lynch Syndrome in Women With Endometrial and Non-Serous Ovarian Cancer (LS2)Colorectal Neoplasms | Ovarian Neoplasms | Lynch Syndrome | Endometrial NeoplasmsCanada
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National Heart, Lung, and Blood Institute (NHLBI)CompletedHeart Diseases | Cardiovascular Diseases | Aortic Valve Stenosis | Heart Septal Defects, Atrial | Down Syndrome | Tetralogy of Fallot | Pulmonic Stenosis | Transposition of Great Vessels | Heart Septal Defects, Ventricular | Ductus Arteriosus, Patent | Coarctation of Aorta | Defect, Congenital Heart