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Clinical Trials on Rare Genetic Disease With Cutaneous Expression
Total 15400 results
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Luminex CorporationCompletedAdrenal Hyperplasia, Congenital | Cystic Fibrosis | Congenital HypothyroidismUnited States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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Weill Medical College of Cornell UniversityUniversity of California, Los Angeles; University of Wisconsin, MilwaukeeCompletedTourette's Syndrome | Tourette's Disorder | Tourette Disorder | Gilles de la Tourette Syndrome | Tourette's Disease | Tourette Disease | Tic Disorder, Combined Vocal and Multiple Motor | Multiple Motor and Vocal Tic Disorder, Combined | Gilles de La Tourette's Disease | Gilles De La Tourette's Syndrome | Combined... and other conditions
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Gynecologic Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Uterine Corpus Carcinoma | Endometrial Clear Cell Adenocarcinoma | Endometrial Serous Adenocarcinoma | Endometrial Adenocarcinoma | Endometrial Adenosquamous Carcinoma | Endometrial Endometrioid Adenocarcinoma, Variant With Squamous Differentiation | Endometrioid Stromal SarcomaUnited States
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University of Southern CaliforniaNational Cancer Institute (NCI)WithdrawnRecurrent Bladder Cancer | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Transitional Cell Carcinoma of the Bladder | Ureter Cancer | Recurrent Urethral Cancer | Urethral Cancer Associated With Invasive Bladder Cancer | Localized Transitional Cell Cancer... and other conditions
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University of VermontUniversity of Massachusetts, Worcester; Arizona State University; Case Western... and other collaboratorsActive, not recruitingDepression | Heart Failure | Hypertension | Diabetes | Arthritis | Chronic Pain | Irritable Bowel Syndrome | Insomnia | Asthma | Fibromyalgia | Anxiety | Substance Use Disorder | Chronic Obstructive Lung Disease | Problem DrinkingUnited States
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)CompletedChronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell... and other conditionsUnited States, Italy
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1Israel, Sweden, United Kingdom
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States
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Johns Hopkins UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...RecruitingLynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation CarrierUnited States
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University Hospital, AngersUniversity of Angers, AngersUnknownIsolated Growth Hormone Deficiency | Turner Syndrome | Pediatrics | Social Cognition | Cognitive Functions
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Fusion Pharmaceuticals Inc.RecruitingGastric Cancer | Colorectal Cancer | Ewing Sarcoma | Squamous Cell Carcinoma of Head and Neck | Pancreatic Ductal Adenocarcinoma (PDAC) | NTSR1 Expressing Solid Tumours | Neuroendocrine Differentiated (NED) Prostate CancerUnited States, Australia
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CENTOGENE GmbH RostockTerminatedDeafness | Immunodeficiency | Mental Retardation | Gingival Hypertrophy | Skeletal Abnormalities | Facial DysmorphismGermany, Switzerland
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingStage III Renal Cell Cancer AJCC v8 | Stage IV Renal Cell Cancer AJCC v8 | Kidney Medullary Carcinoma | Loss of INI 1 Protein ExpressionUnited States
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Randy KardonRecruitingLeber Hereditary Optic Neuropathy | Ischemic Optic Neuropathy | Hemianopia | Branch Retinal Artery Occlusion | Acute Zonal Occult Outer RetinopathyUnited States
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Leigh R. Hochberg, MD, PhD.National Institute of Neurological Disorders and Stroke (NINDS); US Department... and other collaboratorsRecruitingAmyotrophic Lateral Sclerosis | Muscular Dystrophy | Spinal Cord Injuries | Tetraplegia | Brain Stem Infarctions | Locked in SyndromeUnited States
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Inozyme PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyUnited States, United Kingdom
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Universitaire Ziekenhuizen KU LeuvenCompletedRegistry With Information About Colitis Ulcerosa and Familial Adenomatous Polyposis Patients (KOPOF)Adenomatous Polyposis Coli | Colitis,Ulcerative | Reservoir, IleoanalBelgium
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University College, LondonUnknownHypopituitarism | Primary Ovarian Insufficiency | Turner's Syndrome | Hypogonadotrophic Hypogonadism | Primary Amenorrhoea | Hypothalamic AmenorrhoeaUnited Kingdom
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AstraZenecaRecruitingAdv Solid Malig - H&N SCC, ATM Pro / Def NSCLC, Gastric, Breast and Ovarian CancerSpain, United States, Belgium, United Kingdom, France, Hungary, Canada, Korea, Republic of, Australia
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Children's National Research InstituteNational Center for Advancing Translational Sciences (NCATS)RecruitingHypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia IUnited States
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National Cancer Institute (NCI)RecruitingKidney Cancer | Renal Cell Carcinoma | Urologic Malignant Disorders | Familial Renal Cancer (FRC) | Clear Cell Renal CancerUnited States
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Leiden University Medical CenterRecruitingCognitive Decline | Cerebral Amyloid Angiopathy | Intracerebral Hemorrhage | Hypertensive Hemorrhage, IntracranialNetherlands
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Bellicum PharmaceuticalsNo longer availableHurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage DisorderUnited States
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Centre Hospitalier Universitaire VaudoisSwiss National Science FoundationRecruitingKallmann Syndrome | Hypogonadotropic Hypogonadism | Hypothalamic Amenorrhea | Polycystic Ovarian Syndrome | Precocious Puberty | Cleft Lip and Palate | Cleft Palate | Cleft LipSwitzerland
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Cardiff UniversityUniversity of CanterburyRecruitingCleft Lip and Palate | Parenting | Neurofibromatoses | Skin Condition | Alopecia | Craniofacial | Limb DifferenceUnited Kingdom
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Massachusetts Eye and Ear InfirmaryRecruiting
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Massachusetts General HospitalRecruitingNeurofibromatoses | Neurofibromatosis 1 | Neurofibromatosis 2 | SchwannomatosisUnited States
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The Children's Tumor FoundationRecruitingNeurofibromatosis 1 | Neurofibromatosis 2 | SchwannomatosisUnited States
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National Institute of Neurological Disorders and...Recruiting
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FDA Office of Orphan Products DevelopmentCochlearCompleted
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Saccadous, Inc.Barrow Neurological Institute; Dignity HealthCompletedParkinson Disease | Parkinsonian Disorders | Corticobasal Degeneration | Progressive Supranuclear Palsy | Huntington Disease | Essential Tremor | Parkinson's Disease and Parkinsonism | Vascular Parkinsonism | Parkinsonian Syndrome | Multiple System Atrophy, Parkinson VariantUnited States
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Altoosi University CollegeBabylon UniversityUnknownCerebral Palsy | Cancer | HIV/AIDS | Muscular Dystrophy | Cystic Fibrosis | Cardiac Anomaly | Batten's DiseaseIraq
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Fairview University Medical CenterUnknownMucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell DiseaseUnited States
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Johns Hopkins UniversityNational Institutes of Health (NIH); The Marfan FoundationCompletedSnoring | Sleep-disordered BreathingUnited States
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NYU Langone HealthNovartis Pharmaceuticals; The Children's Tumor FoundationCompletedNeurofibromatosis Type IIUnited States
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Inozyme PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyUnited States, United Kingdom, Canada
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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The University of Texas Health Science Center at...National Institute of General Medical Sciences (NIGMS)RecruitingKidney Neoplasms | Bone Cancer | Thyroid Neoplasms | Pheochromocytoma | Paraganglioma | Other Cancer | Inherited Cancer Syndrome | Associated ConditionsUnited States
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ZevraDenmarkActive, not recruitingNiemann-Pick Disease, Type CUnited States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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CENTOGENE GmbH RostockWithdrawnNiemann-Pick Disease, Type C | Niemann-Pick DiseaseGermany, India, Sri Lanka
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University Hospital, GrenobleTerminatedPsychiatric Adults PatientsFrance
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedNiemann-Pick Disease, Type CUnited States