Clinical Trials on GNE Myopathy
Total 1144 results
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Royal Marsden NHS Foundation TrustPfizer; Breast Cancer NowActive, not recruitingGastric Cancer | Triple Negative Breast Cancer | Lobular Breast Carcinoma | CDH1 Gene MutationUnited Kingdom
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Stanford UniversityCompletedRecurrent Non-Small Cell Lung Carcinoma | KRAS Gene Mutation | Metastatic Malignant Neoplasm in the Brain | Stage IV Non-Small Cell Lung Cancer AJCC v7United States
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National Institute of Allergy and Infectious Diseases...TerminatedIFN-Gamma Therapy | CGD Gene Mutation | CGD Response to IFNg | CGD - Chronic Granulomatous Disease | Immunodeficiency DiseaseUnited States
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University of Sao Paulo General HospitalCompletedRheumatoid Arthritis | Systemic Sclerosis | Granulomatosis With Polyangiitis | Juvenile Idiopathic Arthritis | Sjogren's Syndrome | Takayasu Arteritis | Spondyloarthritis | Juvenile Dermatomyositis | Mixed Connective Tissue Disease | Systemic Lupus | Inflammatory MyopathyBrazil
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Mayo ClinicNational Institute of Neurological Disorders and Stroke (NINDS); National Institute... and other collaboratorsRecruitingAmyotrophic Lateral Sclerosis | Progressive Supranuclear Palsy (PSP) | Corticobasal Degeneration (CBD) | GRN Related Frontotemporal Dementia | Behavioral Variant Frontotemporal Dementia (bvFTD) | Semantic Variant Primary Progressive Aphasia (svPPA) | Nonfluent Variant Primary Progressive Aphasia... and other conditionsUnited States, Canada
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingFatigue | Overweight | Lynch Syndrome | Chronic Lymphocytic Leukemia | BRCA1 Gene Mutation | BRCA2 Gene Mutation | Breast Carcinoma | Cancer Survivor | Ovarian Carcinoma | Health Status Unknown | Body Mass Index 25 or GreaterUnited States
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Vanderbilt University Medical CenterRecruitingIdiopathic Pulmonary Arterial Hypertension | Heritable Pulmonary Arterial Hypertension | Unaffected Mutation Carriers: Healthy Participants With a Known BMPR2 Gene Mutation and Normal Pulmonary Pressure and RV Function on Echo | Healthy Individuals With no Cardiopulmonary DiseaseUnited States
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Roswell Park Cancer InstituteCompletedStage IIIA Non-Small Cell Lung Cancer | Stage IIIB Non-Small Cell Lung Cancer | Recurrent Non-Small Cell Lung Carcinoma | Stage IV Non-Small Cell Lung Cancer | EGFR Gene MutationUnited States
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Johns Hopkins UniversityMassachusetts General Hospital; Washington University School of Medicine; Emory... and other collaboratorsCompletedMotor Neuron Disease | Amyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Progressive Muscular Atrophy | Healthy Controls | Flail Arm ALS | Monomelic Amyotrophy | Asymptomatic ALS Gene CarriersUnited States
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National Cancer Institute (NCI)CompletedTherapy-Related Acute Myeloid Leukemia | Acute Myeloid Leukemia With FLT3/ITD Mutation | FLT3 Gene Mutation | Acute Promyelocytic Leukemia With PML-RARA | Acute Myeloid Leukemia With Inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11 | Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1United States
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Shanghai Chest HospitalUnknownNon-Small Cell Lung Cancer | Brain Metastases | EGFR Gene MutationChina
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Massachusetts General HospitalActive, not recruitingCovid19 | Diabetes Mellitus, Type I | Autoimmune Diabetes | Diabetes Mellitus, Type OneUnited States
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Instituto Nacional de Cancerologia de MexicoRecruitingNon-small Cell Lung Cancer | Brain Metastases | EGFR Gene MutationMexico
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Radboud University Medical CenterA-T Children's Project; Twan foundation (https://twanfoundation.nl)CompletedAtaxia Telangiectasia | ATM Gene MutationNetherlands
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Jacobio Pharmaceuticals Co., Ltd.RecruitingSolid Tumors | ER+ Breast Cancer | ARID1A Gene Mutation | Triple Negative Breast Cancer, TNBC | Small Cell Lung Cancer, SCLCUnited States, China
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Memorial Sloan Kettering Cancer CenterAstraZenecaRecruitingNSCLC | Lung Cancer | Non-small Cell Lung Cancer Stage III | Non-small Cell Lung Cancer | Non-small Cell Lung Cancer Stage I | PD-L1 Gene Mutation | Non-small Cell Lung Cancer Stage IIIA | Non-small Cell Lung Cancer Stage IIUnited States
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RWTH Aachen UniversityBayerCompletedAtrial Fibrillation or Pulmonary Embolism | Need of Long Term Oral Anticoagulation Therapy (OAT) | Existent Coronary or Valvular Calcification, or Both and Agatston Score > 50 in at Least One LocationGermany
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingStage IVA Lung Cancer AJCC v8 | Stage IVB Lung Cancer AJCC v8 | Lung Non-Small Cell Carcinoma | Advanced Lung Carcinoma | Stage IV Lung Cancer AJCC v8 | Recurrent Lung Non-Small Cell Carcinoma | ALK Gene RearrangementUnited States
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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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LMU KlinikumUniversity of Pisa; German Federal Ministry of Education and Research; European...RecruitingMitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEOAustria, Germany, Italy
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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University of California, DavisNational Cancer Institute (NCI); Merck Sharp & Dohme LLC; NovartisActive, not recruitingKRAS Gene Mutation | Stage IV Non-Small Cell Lung Cancer AJCC v7 | Recurrent Non-Squamous Non-Small Cell Lung Carcinoma | Metastatic Non-Squamous Non-Small Cell Lung CarcinomaUnited States
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Lei LiRecruitingEpithelial Ovarian Cancer | Homologous Recombination Deficiency | Homologous Recombination Repair Gene Mutation | Drug Resistance | Overall Survival | Progression-free Survival | Poly(ADP-ribose) Polymerase InhibitorChina
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Postgraduate Institute of Medical Education and...RecruitingPortal Vein Thrombosis | Anticoagulants and Bleeding Disorders | Hepatic Vein Thromboses | Hepatic Venous Outflow Obstruction | Portal Hypertension, Noncirrhotic | Portal Vein Occlusion | Portal Vein Embolism | JAK2 Mutation | CALR Gene Mutation | Prothrombin G20210AIndia
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Shaare Zedek Medical CenterRecruitingParkinson Disease | Gaucher Disease | GBA Gene MutationIsrael
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Pacific Parkinson's Research CentreUniversity of British Columbia; Oregon Health and Science University; University... and other collaboratorsRecruitingParkinson Disease | Gaucher Disease | GBA Gene MutationUnited States, Canada
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National Heart, Lung, and Blood Institute (NHLBI)CompletedHypertension | Cardiovascular Disease | Williams Syndrome | Multisystem Developmental Disorder | Elastin Gene DeletionUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)RecruitingChronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Recurrent Acute Myeloid Leukemia | Refractory Acute Myeloid Leukemia | IDH2 Gene Mutation | Acute Bilineal Leukemia | Acute Biphenotypic LeukemiaUnited States
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Turning Point Therapeutics, Inc.TerminatedNon Small Cell Lung Cancer | Advanced Solid Tumor | Metastatic Solid Tumor | Medullary Thyroid Cancer | RET Gene MutationUnited States, Korea, Republic of
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RWJ Barnabas Health at Jersey City Medical CenterUnknownLynch Syndrome | Endometrial Cancer | Somatic Mutation | Cancer Gene MutationUnited States
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Newcastle UniversityLudwig-Maximilians - University of Munich; LGMD2i Research Fund; CureLGMD2iRecruitingLimb Girdle Muscular Dystrophy | Congenital Muscular Dystrophy | LGMDR9 | LGMD2I | Walker-Warburg Syndrome | Muscle-Eye-Brain Disease | FKRP Gene MutationUnited Kingdom
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PYC TherapeuticsNot yet recruitingAutosomal Dominant Optic Atrophy | Kjer Optic Atrophy | OPA1 Gene Mutation | Hereditary Optic Atrophies
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingCardiovascular Disease | Arterial Stiffness | Germline Mutation in the NOTCH 3 Gene | Pathogenesis of CADASIL | Clinical Phenotype of CADASILUnited States
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CAI HongbingRecruitingOvarian Cancer | Epithelial Ovarian Cancer | Ovarian Cancer, Epithelial | Hyperthermic Intraperitoneal Chemotherapy(HIPEC) | Homologous Recombination Repair Gene MutationChina
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William ShomaliIncyte CorporationRecruitingEosinophilia | Hypereosinophilic Syndrome | Blasts More Than 5 Percent of Bone Marrow Nucleated Cells | Splenomegaly | Hepatomegaly | Chronic Eosinophilic Leukemia, Not Otherwise Specified | JAK2 Gene Mutation | BCR-JAK2 Fusion Protein Expression | Blasts 20 Percent or Less of Peripheral Blood White... and other conditionsUnited States
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University of ArkansasWithdrawnPancreatic Neoplasms | Colorectal Neoplasms, Hereditary Nonpolyposis | BRCA1 Gene Mutation | BRCA2 Gene Mutation | Hereditary Pancreatitis | Ataxia Telangiectasia | Peutz-Jegher's Syndrome | Familial Atypical Mole-Malignant Melanoma SyndromeUnited States
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Hadassah Medical OrganizationRecruitingNSCLC | Non-small Cell Lung Cancer | EGF-R Positive Non-Small Cell Lung Cancer | Brain Metastases | EGFR Gene MutationIsrael
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCardiovascular Disease | Arterial Stiffness | Germline Mutation in the NOTCH 3 Gene | Pathogenesis of CADASIL | Clinical Phenotype of CADASILUnited States
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Blueprint Medicines CorporationActive, not recruitingNeoplasms | Neoplasms by Histologic Type | Lung Diseases | Neoplasms by Site | Adenocarcinoma | Carcinoma | Carcinoma, Non-Small-Cell Lung | Lung Neoplasms | Respiratory Tract Neoplasms | Thoracic Neoplasms | Carcinoma, Bronchogenic | Bronchial Neoplasms | Neoplasms, Nerve Tissue | EGF-R Positive Non-Small Cell Lung... and other conditionsUnited States, Canada, Taiwan, Korea, Republic of, Singapore, Japan, United Kingdom, France, Netherlands, Spain
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Northwestern UniversityNational Cancer Institute (NCI); Millennium Pharmaceuticals, Inc.Active, not recruitingDiffuse Large B-Cell Lymphoma | Plasmablastic Lymphoma | B-Cell Lymphoma, Unclassifiable, With Features Intermediate Between Diffuse Large B-Cell Lymphoma and Burkitt Lymphoma | Adult Burkitt Lymphoma | MYC Gene MutationUnited States
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N.N. Petrov National Medical Research Center of...RecruitingMelanoma | Lung Cancer | Pancreas Cancer | Refractory Cancer | Colorectal Cancer Recurrent | RAS Mutation | Ras (Kras or Nras) Gene MutationRussian Federation
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Kura Oncology, Inc.RecruitingAcute Myeloid Leukemia | Acute Myeloid Leukemia, in Relapse | Myeloid Sarcoma | Acute Myeloid Leukemia Recurrent | Mixed Phenotype Acute Leukemia | Mixed Lineage Acute Leukemia | Refractory AML | NPM1 Mutation | Mixed Lineage Leukemia Gene Mutation | AML With Mutated NPM1 | KMT2ArUnited States
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Cothera Bioscience, IncRecruitingLymphoma, B-Cell | Lymphoma, Large B-Cell, Diffuse | Lymphatic Diseases | Burkitt Lymphoma | High-grade B-cell Lymphoma | C-MYC/BCL6 Double-Hit High-Grade B-Cell Lymphoma | C-MYC/BCL2 Double-Hit High-Grade B-Cell Lymphoma | Lymphoma, High-Grade | C-Myc Gene RearrangementKorea, Republic of, China
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University of California, San FranciscoTerminatedALK Fusion Protein Expression | Stage III Cutaneous Melanoma | Stage IIIA Cutaneous Melanoma | Stage IIIB Cutaneous Melanoma | Stage IIIC Cutaneous Melanoma | Stage IV Cutaneous Melanoma | ROS1 Fusion Positive | BRAF wt Allele | Invasive Skin Melanoma | MET Fusion Gene Positive | NRAS wt Allele | NTRK1 Fusion... and other conditionsUnited States
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Stanford UniversityNational Institutes of Health (NIH)RecruitingStomach Cancer | Esophageal Cancer | Gastrointestinal Stromal Tumor (GIST) | Gastric (Stomach) Cancer | Gastro-Esophageal(GE) Junction CancerUnited States
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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Ikena OncologyActive, not recruitingSolid Tumor | Solid Tumors, Adult | Malignant Pleural Mesothelioma (MPM) | Epithelioid Hemangioendothelioma (EHE) | NF2 Deficient Mesothelioma | Other NF2 Deficient Solid Tumors and Solid Tumors With YAP1/TAZ Fusion Genes | NF2 Deficiency | YAP1 or TAZ Gene FusionsUnited States, United Kingdom
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI); ExelixisActive, not recruitingRenal Cell Carcinoma | Metastatic Renal Cell Carcinoma | Stage IV Renal Cell Cancer AJCC v8 | Sarcomatoid Renal Cell Carcinoma | Chromophobe Renal Cell Carcinoma | Papillary Renal Cell Carcinoma | Malignant Neoplasms of Urinary Tract | Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3... and other conditionsUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedFLT3 Gene Mutation Negative | FLT3 Internal Tandem Duplication Positive | Recurrent Acute Myeloid Leukemia | Recurrent Chronic Myelomonocytic Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Chronic Myelomonocytic Leukemia | Refractory Myelodysplastic...United States
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Tel-Aviv Sourasky Medical CenterUnknownFamilial Adenomatous Polyposis | FAP | FAP Gene Mutation