Clinical Trials on Genetic Diseases, X-Linked
Total 15161 results
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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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IpsenCompletedCerebral Palsy | Cervical Dystonia | Muscle Spasticity | Cerebrovascular Accident | Equinus Deformity | Hemifacial Spasm | Blepharospasm | Spasmodic Torticollis | Facial Hyperdynamic LinesFrance, Germany, Russian Federation
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Memorial Sloan Kettering Cancer CenterPfizer; Dana-Farber Cancer Institute; University of Pittsburgh; University of VirginiaCompletedNeurofibromatosis | Meningioma | CNS Cancer | Hemangioblastoma | Intracranial HemangiopericytomaUnited States
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Baylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of MedicineRecruitingLiposarcoma | Liver Cancer | Rhabdomyosarcoma | Wilms Tumor | Malignant Rhabdoid Tumor | Yolk Sac TumorUnited States
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Baylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of MedicineNot yet recruitingLiposarcoma | Liver Cancer | Rhabdomyosarcoma | Wilms Tumor | Malignant Rhabdoid Tumor | Yolk Sac TumorUnited 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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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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Universitaire Ziekenhuizen KU LeuvenKU LeuvenUnknownObesity | Healthy Volunteers | Prader-Willi Syndrome | Muscle Hypotonia | Hypotonia-Cystinuria Syndrome | Dwarfism, Growth Hormone DeficiencyBelgium
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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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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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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University of MichiganNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health...RecruitingObstructive Sleep Apnea | Down Syndrome | Tolerance | Trisomy 21 | Hypertonia, Muscle | Nasal Airway ObstructionUnited States
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Fondation Ophtalmologique Adolphe de RothschildRecruitingTuberous Sclerosis | Focal Cortical Dysplasia | Hemimegalencephaly | Refractory Focal Epilepsy | Mild Malformation of Cortical Development With Oligodendroglial Hyperplasia in Epilepsy (MOGHE)France
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National Taiwan University HospitalNational Cheng-Kung University HospitalUnknownMELAS Syndrome | Noninsulin-dependent Diabetes Mellitus With DeafnessTaiwan
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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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Novartis PharmaceuticalsCompletedHypereosinophilic Syndrome | Chronic Myelogenous Leukemia | Systemic Mastocytosis | Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive)United States, Poland, Italy, Belgium, Canada, France, Australia, Netherlands, Sweden, Switzerland, United Kingdom, Spain, Korea, Republic of, Austria, Germany, Denmark, Finland, Singapore, Taiwan, Hong Kong, New Zealand, Norway
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National Cancer Institute (NCI)Childrens Oncology Group (COG); LLS PedAL LLCRecruitingDown Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...United States
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National Cancer Institute (NCI)CompletedThrombocytopenia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13... and other conditionsUnited States
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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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7 Hills Pharma, LLCRecruitingMelanoma | Kidney Cancer | Renal Cell Carcinoma | Hepatocellular Carcinoma | Colorectal Cancer | NSCLC | Non Small Cell Lung Cancer | Skin Cancer | Metastasis | Advanced Solid Tumor | Advanced Cancer | Renal Cell Cancer | Hepatocellular Cancer | Mismatch Repair Deficiency | Pleural Mesothelioma | MSI-High | Anaplastic Lymphoma... and other conditionsUnited States
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Shirley Ryan AbilityLabRecruitingMultiple Sclerosis | Paralysis | Muscular Dystrophy | Spinal Cord Injuries | Cerebrovascular Accident | Post-polio SyndromeUnited 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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St. Jude Children's Research HospitalUniversity of MiamiCompletedNeuroblastoma | Central Nervous System Tumors | Wilms Tumor | LymphomasUnited States
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Pediatric Brain Tumor ConsortiumNational Cancer Institute (NCI); American Lebanese Syrian Associated Charities...RecruitingLow Grade Glioma (LGG) of Brain With BRAF Aberration | High Grade Glioma (HGG) of the Brain With BRAF Aberration | Low Grade Glioma of Brain With Neurofibromatosis Type 1United States
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Children's Oncology GroupRecruitingLymphoblastic Lymphoma | B Acute Lymphoblastic Leukemia | B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | T Acute Lymphoblastic Leukemia | Mixed Phenotype Acute Leukemia | B Acute Lymphoblastic Leukemia, BCR-ABL1-LikeUnited States, Canada
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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...CompletedUrogenital Abnormalities | Mental Retardation | Wilm's Tumor | WAGR Syndrome | AniridiaUnited 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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Richard FryeAutism Discovery and Treatment FoundationRecruitingEpilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal InfectionUnited 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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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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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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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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Memorial Sloan Kettering Cancer CenterCompleted
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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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Sun Yat-sen UniversityRecruitingBreast Cancer | Cognitive Impairment | Brain Metastases | Radiotherapy Side Effect | Gangliosidosis, GM1China
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NYU Langone HealthRecruitingMLH1 Gene Mutation | RAD51C Gene Mutation | BRIP1 Gene Mutation | MSH6 Gene Mutation | PMS2 Gene Mutation | BRCA-Mutated Ovarian Carcinoma | MSH2 A636P | EPCAMUnited States
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Duke UniversityNational Institute of Nursing Research (NINR); National Institutes of Health...Active, not recruitingChronic Kidney Diseases | Diabetes Mellitus, Type 1 | Systemic Lupus Erythematosus | Inflammatory Bowel Diseases | Cystic Fibrosis | Sickle Cell Disease | Stem Cell Transplant | Childhood Cancer | Organ TransplantUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)TerminatedAccelerated Phase Chronic Myelogenous Leukemia (CML) | Blast Phase Chronic Myelogenous Leukemia (CML) | Chronic Phase Phase Chronic Myelogenous Leukemia (CML) | Philadelphia Chromosome Positive (Ph+) Phase Chronic Myelogenous Leukemia (CML)United States
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Puerto Rico
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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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Atara BiotherapeuticsRecruitingLymphoproliferative Disorders | Leiomyosarcoma | Stem Cell Transplant Complications | Solid Organ Transplant Complications | Epstein-Barr Virus (EBV)-Associated Diseases | EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD) | Allogeneic Hematopoietic Cell Transplant | EBV+ Sarcomas | EBV+ Lymphoproliferative... and other conditionsUnited States, Austria, Belgium, France, Italy, Spain, United Kingdom