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Clinical Trials on Anticipation, Genetic
Total 14793 results
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Children's Hospital of PhiladelphiaCompletedRheumatoid Arthritis | Lupus Erythematosus, Systemic | Idiopathic Thrombocytopenic Purpura | Inflammatory Bowel Disease | Anemia, Hemolytic, Autoimmune | Evans Syndrome | Autoimmune Pancytopenia | Autoimmune Lymphoproliferative Syndrome (ALPS) | Autoimmune NeutropeniaUnited States
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University of California, San FranciscoNational Cancer Institute (NCI)CompletedLeukemia | Thrombocytopenia | Fanconi Anemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan AnemiaUnited States
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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Ospedale San RaffaeleRecruiting
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Massachusetts General HospitalAgios Pharmaceuticals, Inc.RecruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditionsUnited States
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University of MiamiNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes...RecruitingAmyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Frontotemporal Dementia | Progressive Muscular Atrophy | Hereditary Spastic ParaplegiaUnited States, South Africa
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Changi General HospitalSingapore General HospitalRecruitingPrimary Aldosteronism | Hypokalemia | Chronic Renal Disease | Cardiovascular Morbidity | Mineralocorticoid Excess | Adrenalectomy; Status | Primary Aldosteronism Due to Aldosterone Producing Adenoma | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral) | Mineralocorticoid Antagonists [Aldosterone...Singapore
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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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Washington University School of MedicineEnrolling by invitation
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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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Reata, a wholly owned subsidiary of BiogenCompletedFocal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 DiabetesUnited States
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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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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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Alcon ResearchCompletedKeratoconjunctivitis | Corneal Edema | Corneal Ulcer | Bullous Keratopathy | Bell Palsy | Corneal Dystrophy | Entropion | Filamentary Keratitis | Corneal Erosion | Foreign Body in Cornea | Sicca Syndrome; Keratoconjunctivitis (Etiology)United States
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CENTOGENE GmbH RostockWithdrawnNiemann-Pick Disease, Type C | Niemann-Pick DiseaseGermany, India, Sri Lanka
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OHSU Knight Cancer InstituteNational Heart, Lung, and Blood Institute (NHLBI)CompletedChronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative NeoplasmsUnited States
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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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National Cancer Institute (NCI)CompletedB-cell Adult Acute Lymphoblastic Leukemia | Acute Undifferentiated Leukemia | Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic Leukemia | B-cell Childhood Acute Lymphoblastic Leukemia | L1 Childhood Acute Lymphoblastic Leukemia | L2 Childhood Acute Lymphoblastic Leukemia | T-cell... and other conditionsUnited States
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Dana-Farber Cancer InstituteTerminatedSarcoma | Soft Tissue Sarcoma | Clear Cell Renal Cell Carcinoma | Osteosarcoma | Sarcoma, Ewing | Ewing Sarcoma | Rhabdoid Tumor | Renal Tumor | Rhabdomyosarcoma | Wilms Tumor | Non-Rhabdomyosarcoma Soft Tissue Sarcoma, NosUnited States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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University of Colorado, DenverCompletedMarfan Syndrome | Turner Syndrome | Loeys-Dietz Syndrome | Thoracic Aortic Aneurysm and Dissection Syndromes | Ehlers-Danlos Type IV SyndromeUnited States
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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
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; University of Oxford; National Human...CompletedNiemann-Pick Disease, Type CUnited States
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Lenetix Medical Screening LaboratoryUnknownDown Syndrome (Trisomy 21) | Edward's Syndrome (Trisomy 18) | Patau Syndrome (Trisomy 13) | Klinefelter Syndrome (47, XXY) | and Other Chromosome | Abnormalities.United States
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National Institute of Neurological Disorders and...CompletedBrain Neoplasm | Alzheimer's Disease | Niemann Pick DiseaseUnited States
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National Institute of Neurological Disorders and...Baylor College of MedicineCompletedPrader-Willi Syndrome | DiGeorge Syndrome | Williams Syndrome | Angelman Syndrome | Smith-Magenis Syndrome | Chromosome Abnormalities | Shprintzen SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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LMU KlinikumSeventh Framework Programme; NBIA AllianceRecruitingNeurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditionsCanada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
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Centre d'Etude des Cellules SouchesGenethonRecruitingWolfram SyndromeFrance, Spain
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Merck Sharp & Dohme LLCRecruitingVon Hippel-Lindau Disease | Pancreatic Neuroendocrine Tumor | Pheochromocytoma/Paraganglioma | Advanced Gastrointestinal Stromal Tumor | HIF-2α Mutated CancersCanada, United States, Denmark, France, Germany, Hungary, Italy, Russian Federation, Spain, Sweden, Turkey, Israel, United Kingdom, Netherlands, Australia, China, Singapore, Japan
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LLS PedAL Initiative, LLCNational Cancer Institute (NCI); Children's Oncology GroupRecruitingAcute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Myeloid Leukemia Associated With Down Syndrome | Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic Syndrome Post Cytotoxic TherapyUnited States, Canada, Australia, Puerto Rico, New Zealand
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Scott R. Plotkin, MD, PhDTakeda; National Comprehensive Cancer Network; The Children's Tumor FoundationRecruitingEpendymoma | Meningioma | Neurofibromatosis Type 2 | Vestibular Schwannoma | Non-vestibular SchwannomaUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingBlast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Philadelphia Chromosome Positive | Recurrent Acute Lymphoblastic Leukemia | Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Refractory Acute Lymphoblastic Leukemia | Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive and other conditionsUnited States
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Amylyx Pharmaceuticals Inc.Active, not recruiting
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Washington University School of MedicineCompletedGlioma | Neuroectodermal Tumors, Primitive | Osteosarcoma | Sarcoma, Ewing | Retinoblastoma | Rhabdomyosarcoma | Wilms TumorUnited States
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Children's Hospital Medical Center, CincinnatiAstraZenecaActive, not recruitingGlioma | Ependymoma | Meningioma | Neurofibromatosis 2 | Vestibular SchwannomaUnited 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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Wen-Shiou PanCompletedHeart Failure | Stroke | Pulmonary Disease, Chronic Obstructive | Kidney Failure, Chronic | Alzheimer Disease, Late Onset | Kidney Failure, Acute | Cystic Fibrosis Pulmonary Exacerbation | Chronic Liver Disease and CirrhosisTaiwan
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National Cancer Institute (NCI)CompletedNeurofibromatosis Type 1 | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary Central Nervous System Neoplasm | Recurrent Childhood Brain Stem Glioma | Recurrent Childhood Visual Pathway GliomaUnited States
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Azienda Socio Sanitaria Territoriale degli Spedali...CompletedAtaxia | Multiple System Atrophy | Spinocerebellar Ataxias | Spinocerebellar Ataxia 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Ataxia With Oculomotor Apraxia | CANVASItaly
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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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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; Weill Medical College of Cornell...CompletedNeimann-Pick DiseaseUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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University of South FloridaCompletedAlcohol Dependence | Anxiety Disorders | ADHD | Mania | Major Depression | Conduct Disorder | Oppositional Defiant Disorder | Eating Disorders | Dysthymia | Pervasive Developmental Disorder | Drug Dependence | Suicidality | Psychotic Disorder | Tourettes DisorderUnited States
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Hospital Universitari Vall d'Hebron Research InstituteErasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...RecruitingIron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary AnemiaSpain
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Third Military Medical UniversityNot yet recruitingPrimary Aldosteronism Due to Aldosterone Producing Adenoma | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)China
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Leigh R. Hochberg, MD, PhD.National Institute on Deafness and Other Communication Disorders (NIDCD)RecruitingAmyotrophic Lateral Sclerosis | Muscular Dystrophies | Spinal Cord Injuries | Tetraplegia | Dysarthria | Brain Stem Infarctions | Locked in Syndrome | AnarthriaUnited States
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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