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Clinical Trials on Rare Genetic Disease With Cutaneous Expression
Total 15460 results
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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Mayo ClinicCambridge University Hospitals NHS Foundation TrustRecruitingHeart Diseases | Cardiovascular Diseases | Myocarditis | Cardiomyopathies | Ventricular Tachycardia | Cardiovascular Abnormalities | Sarcoidosis | Sudden Cardiac Death | Cardiac Arrhythmia | Cardiac Sarcoidosis | Sudden Cardiac Arrest | Arrhythmogenic Right Ventricular Cardiomyopathy | Arrhythmogenic Right Ventricular... and other conditionsUnited States, United Kingdom
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SWOG Cancer Research NetworkNational Cancer Institute (NCI)CompletedRecurrent Adult Burkitt Lymphoma | Recurrent Adult Acute Lymphoblastic Leukemia | B-cell Adult Acute Lymphoblastic Leukemia | Acute Leukemias of Ambiguous Lineage | Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic LeukemiaUnited States
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingNiemann-Pick Disease, Type CUnited States
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University of LeicesterRecruitingMental Health Issue (E.G., Depression, Psychosis, Personality Disorder, Substance Abuse) | Cardiovascular Diseases | Stroke | Healthy | Diabetes Mellitus | Cancer | Chronic Kidney Diseases | Chronic Pain | Obstructive Sleep Apnea | Respiratory DiseaseUnited Kingdom
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedDiabetes Mellitus | Type 1 Diabetes | Diabetes Insipidus | Wolfram SyndromeUnited 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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Brigham and Women's HospitalOregon Health and Science University; Vanderbilt University; Geisinger ClinicCompletedMyocardial Infarction | Coronary Artery Disease | Smoking | Stroke | Hypertension | Chronic Obstructive Pulmonary Disease | Atrial Fibrillation | Asthma | Tuberculosis | Sleep Apnea | Sickle Cell Disease | Hyperlipidemia | Congestive Heart FailureUnited States
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Memorial Sloan Kettering Cancer CenterCompleted
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Washington University School of MedicineWithdrawnGlioma | Osteosarcoma | Ependymoma | Medulloblastoma | Rhabdomyosarcoma | Wilms Tumor | Neuroectodermal Tumor | Ewing's Sarcoma
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ConvaTec Inc.CompletedMultiple Sclerosis | Lower Urinary Tract Symptoms | Parkinson Disease | Spinal Cord Injuries | Bladder Outlet Obstruction | Cauda Equina Syndrome | Detrusor Underactivity | Enlarged Prostate With Lower Urinary Tract SymptomsUnited Kingdom, United States, France
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University of California, San FranciscoNational Institute of Neurological Disorders and Stroke (NINDS); National Center... and other collaboratorsCompletedAmyotrophic Lateral Sclerosis (ALS) | Progressive Supranuclear Palsy (PSP) | Corticobasal Degeneration (CBD) | FTLD | Frontotemporal Dementia (FTD) | PPA Syndrome | Behavioral Variant Frontotemporal Dementia (bvFTD) | Semantic Variant Primary Progressive Aphasia (svPPA) | Nonfluent Variant Primary Progressive... and other conditionsUnited States, Canada
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Casa Colina Hospital and Centers for HealthcareCompletedMultiple Sclerosis | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Graves Disease | Myasthenia Gravis | Traumatic Brain Injury | Stroke, Ischemic | Stroke, Acute | Stroke Hemorrhagic | SCI - Spinal Cord Injury | Guillain-Barre Syndrome | Lupus Erythematosus | Stroke (CVA) or TIA | Ehlers-Danlos Syndromes...United States
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Universitaire Ziekenhuizen KU LeuvenKU LeuvenUnknownObesity | Healthy Volunteers | Prader-Willi Syndrome | Muscle Hypotonia | Hypotonia-Cystinuria Syndrome | Dwarfism, Growth Hormone DeficiencyBelgium
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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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Motric BioRecruitingStroke | Multiple Sclerosis | Cerebral Palsy | Spinal Cord Injuries | Dystonia | Muscle Spasticity | Hereditary Spastic Paraplegia | Hypertonia, MuscleUnited States
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Radboud University Medical CenterRecruitingOvarian Diseases | Sex Chromosome Disorders | Infertility, Female | Turner Syndrome | Gonadal Dysgenesis | Premature Ovarian Failure | Premature MenopauseNetherlands, Germany, Poland, Sweden, Denmark
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Duke UniversitySusan G. Komen Breast Cancer FoundationCompletedLynch Syndrome | Hereditary Breast and Ovarian Cancer SyndromeUnited States
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Washington University School of MedicineNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingDiabetes Mellitus | Deafness | Ataxia | Diabetes Insipidus | Wolfram Syndrome | Optic Nerve AtrophyUnited States
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VA Office of Research and DevelopmentCompletedDepression | Insomnia | Dementia | Fibromyalgia | Anxiety | Chronic Low Back Pain | Lumbar Spinal Stenosis | Myofascial Pain Syndrome | Sacroiliac Joint Pain | Hip Ostearthritis | Maladaptive Coping | Lateral Hip and Thigh Pain | Recent Leg Length DiscrepancyUnited States
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MacroGenicsCompletedOsteosarcoma | Ewing Sarcoma | Neuroblastoma | Rhabdomyosarcoma | Wilms Tumor | Desmoplastic Small Round Cell TumorUnited 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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National Cancer Institute (NCI)CompletedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional... and other conditionsUnited States
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National Cancer Institute (NCI)WithdrawnAdvanced Adult Primary Liver Cancer | Localized Unresectable Adult Primary Liver Cancer | Recurrent Adult Primary Liver Cancer | Recurrent Extrahepatic Bile Duct Cancer | Unresectable Extrahepatic Bile Duct Cancer | Stage II Gallbladder Cancer | Stage IIIA Gallbladder Cancer | Stage IIIB Gallbladder... and other conditions
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Shirley Ryan AbilityLabRecruitingMultiple Sclerosis | Paralysis | Muscular Dystrophy | Spinal Cord Injuries | Cerebrovascular Accident | Post-polio SyndromeUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)RecruitingAcute Myeloid Leukemia | High Risk Myelodysplastic Syndrome | Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Philadelphia Chromosome Positive | Myeloproliferative Neoplasm | Myeloid Sarcoma | Blasts 10 Percent or More of Bone Marrow Nucleated CellsUnited States
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National Cancer Institute (NCI)CompletedMultiple Endocrine Neoplasia Type 2A | Multiple Endocrine Neoplasia Type 2B | Medullary Thyroid CarcinomaUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedTongue Cancer | Recurrent Metastatic Squamous Neck Cancer With Occult Primary | Recurrent Salivary Gland Cancer | Recurrent Squamous Cell Carcinoma of the Hypopharynx | Recurrent Squamous Cell Carcinoma of the Larynx | Recurrent Squamous Cell Carcinoma of the Lip and Oral Cavity | Recurrent Squamous... and other conditionsUnited States
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Aspira Women's HealthTerminatedHereditary Breast and Ovarian Cancer | Adnexal Mass | Germline BRCA1 Gene Mutation | Germline BRCA2 Gene MutationUnited States
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Umeå UniversityKarolinska Institutet; Lund University; Göteborg UniversityEnrolling by invitationLynch Syndrome | Hereditary Breast and Ovarian Cancer Syndrome | Familial Breast Cancer | Hereditary Breast Cancer | Familial Colorectal CancerSweden
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Ohio State University Comprehensive Cancer CenterNational Comprehensive Cancer NetworkCompletedTongue Cancer | Recurrent Metastatic Squamous Neck Cancer With Occult Primary | Recurrent Salivary Gland Cancer | Recurrent Squamous Cell Carcinoma of the Hypopharynx | Recurrent Squamous Cell Carcinoma of the Larynx | Recurrent Squamous Cell Carcinoma of the Lip and Oral Cavity | Recurrent Squamous... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedTongue Cancer | Recurrent Metastatic Squamous Neck Cancer With Occult Primary | Recurrent Salivary Gland Cancer | Recurrent Squamous Cell Carcinoma of the Hypopharynx | Recurrent Squamous Cell Carcinoma of the Larynx | Recurrent Squamous Cell Carcinoma of the Lip and Oral Cavity | Recurrent Squamous... and other conditionsUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedUrogenital Abnormalities | Mental Retardation | Wilm's Tumor | WAGR Syndrome | AniridiaUnited States
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Recursion Pharmaceuticals Inc.RecruitingNeurofibromatosis Type 2United States
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Jonsson Comprehensive Cancer CenterNovartis PharmaceuticalsTerminatedNeuroma, Acoustic | Neurofibromatosis Type 2United States
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Massachusetts General HospitalUnited States Department of Defense; AstraZenecaCompletedMeningioma | Neurofibromatosis 2United States
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NYU Langone HealthCompletedNeurofibromatosis Type 2 | Vestibular SchwannomasUnited States
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Li PengBetta Pharmaceuticals Co., Ltd.CompletedNeurofibromatosis Type 2 | Vestibular SchwannomaChina
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National Cancer Institute (NCI)CompletedNeurofibromatosis Type 2 | Vestibular SchwannomaUnited States
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Beijing Tiantan HospitalShandong Simcere-Medgenn Bio-pharmaceutical Co., LtdCompletedNeurofibromatosis Type 2 | Vestibular SchwannomaChina
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Medical College of WisconsinRecruitingLeukemia | Burkitt Lymphoma | Lymphoblastic Lymphoma | Myelodysplasia | Acute Myeloid Leukemia in Remission | Acute Lymphoblastic Leukemia in Remission | Chronic Myelogenous Leukemia - Chronic Phase | Chronic Myelogenous Leukemia, Accelerated Phase | Biphenotypic Acute Leukemia | Burkitt Leukemia | Chronic... and other conditionsUnited States
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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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Mayo ClinicWithdrawnB-cell Adult Acute Lymphoblastic Leukemia | Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic Leukemia | Untreated Adult Acute Lymphoblastic Leukemia | Blastic Phase Chronic Myelogenous LeukemiaUnited States
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Neuroganics LLCRamos Injury Law FirmRecruitingCOVID-19 | Vaccine Adverse Reaction | Vaccine Reaction | Vasculitis | Neuritis | Thrombocytopenia | Influenza | Corona Virus Infection | Blood Clot | Guillain-Barré | Vaccine or Biological Substance, Unspecified Causing Adverse Effects in Therapeutic Use | GWAS | Genetic SequencingUnited States
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Bellicum PharmaceuticalsActive, not recruitingStudy of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantLymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Anemia, Aplastic | Primary Immune Deficiency Disorder | Cytopenia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child | Hemoglobinopathy in ChildrenUnited States
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Joshua Mann, MPHNational Organization for Rare DisordersRecruitingVon Hippel-Lindau Disease | Hereditary Leiomyomatosis and Renal Cell Cancer | Birt-Hogg-Dube Syndrome | SDHB Gene MutationUnited 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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Massachusetts Eye and Ear InfirmaryJohns Hopkins University; Mayo Clinic; Stanford University; Ohio State University; Nationwide Children's HospitalTerminatedMeningioma | Neurofibromatosis Type 2 | Vestibular Schwannoma | Acoustic NeuromaUnited States
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National Cancer Institute (NCI)Active, not recruitingSarcoma | Metastatic Renal Cell Carcinoma | Metastatic Melanoma | Recurrent Renal Cell Carcinoma | Recurrent Melanoma | Stage IV Renal Cell Cancer AJCC v8 | Metastatic Malignant Solid Neoplasm | Metastatic Colorectal Carcinoma | Stage IV Colorectal Cancer AJCC v8 | Clinical Stage IV Cutaneous Melanoma... and other conditionsUnited States, Canada