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Clinical Trials on Inborn Errors of Metabolism in United States
Total 3211 results
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BioCryst PharmaceuticalsCompletedHereditary Angioedema (HAE)Canada, United Kingdom, Germany, Spain, Australia, Hungary, Italy, Austria, North Macedonia, Denmark, Switzerland
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | Prophylaxis | HAEAustria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
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St. Jude Children's Research HospitalRecruitingPancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma and other conditionsUnited States
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Mayo ClinicCompletedMultiple Sclerosis | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Uveitis | Vasculitis | Psoriasis | Graves Disease | Systemic Sclerosis | Celiac Disease | Crohn Disease | Type 1 Diabetes Mellitus | Myasthenia Gravis | Ulcerative Colitis | Dermatomyositis | Polymyositis | Vitiligo | Hepatitis, Autoimmune | Polymyalgia... and other conditionsUnited States
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Sanguine BiosciencesRecruitingHeart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedClear Cell Renal Cell Carcinoma | Stage III Renal Cell Cancer | Stage IV Renal Cell Cancer | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Rhabdoid Tumor of the Kidney | Stage I Renal Cell Cancer | Childhood Renal Cell Carcinoma | Stage II Renal Cell Cancer | Stage I Renal Wilms Tumor and other conditionsUnited States, Canada, Australia, New Zealand, Puerto Rico
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University of WashingtonNational Institute on Aging (NIA)RecruitingPromoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers (PICSI-M)Liver Cirrhosis | Quality of Life | Neoplasm Metastasis | Pulmonary Disease, Chronic Obstructive | Peripheral Vascular Diseases | Dementia | Kidney Failure, Chronic | Chronic Disease | Lung Diseases, Interstitial | Health Communication | Lung Neoplasm | Heart Failure,Congestive | Patient Care | Health Care Quality... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Adult Kidney Wilms Tumor | Beckwith-Wiedemann Syndrome | Childhood Kidney Wilms Tumor | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Stage V Kidney...United States, Canada, Australia, New Zealand, Puerto Rico, Israel
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Global Healthy Living FoundationUniversity of Alabama at BirminghamRecruitingHeart Failure | Stroke | Epilepsy | Multiple Sclerosis | Obesity | Diabetes | Chronic Obstructive Pulmonary Disease | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Peripheral Arterial Disease | Inflammatory Bowel Diseases | Amyotrophic Lateral Sclerosis | Psoriasis | Asthma | Muscular Dystrophy | Fibromyalgia | Alzheimer... and other conditionsUnited States
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Wave Life Sciences Ltd.TerminatedALS | FTDCanada, Belgium, New Zealand, Netherlands, Australia, Sweden, United Kingdom, Ireland
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DePuy InternationalTerminatedOsteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Nonunion of Femoral FracturesAustria, Czech Republic, Finland, Germany, Norway, Portugal, Switzerland, United Kingdom
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TauRx Therapeutics LtdTerminatedAlzheimer's Disease | Behavioral Variant Frontotemporal DementiaKorea, Republic of, United States, Spain, Taiwan, Canada, Belgium, Malaysia, United Kingdom, Australia, Singapore, Croatia, Finland, France, Germany, Netherlands, Romania, Russian Federation
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University of SienaRecruitingBehcet Syndrome | Uveitis | Hereditary Autoinflammatory Diseases | Schnitzler Syndrome | Scleritis | PFAPA Syndrome | Still Disease | Autoinflammatory Syndrome, Unspecified | Vexas SyndromeEgypt, Spain, Turkey, Italy, Saudi Arabia, Greece, Belgium, United States, Algeria, Brazil, Germany, Ghana, Iran, Islamic Republic of, Lebanon, Morocco, Poland, Portugal, Romania, Taiwan
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Fog Pharmaceuticals, Inc.RecruitingCancer | Gastric Cancer | Colorectal Cancer | Solid Tumor | Metastatic Cancer | Adenomatous Polyposis Coli | Non-small Cell Lung Cancer | Non-small Cell Lung Cancer Metastatic | Non-small Cell Carcinoma | Locally Advanced Solid Tumor | Non-small Cell Lung Cancer Stage IIIB | Gastroesophageal-junction Cancer | Non-Small... and other conditionsUnited States
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AmgenCompletedLymphoma | Kidney Cancer | Breast Cancer | Head and Neck Cancer | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Non-Small Cell Lung Cancer | Lung Cancer | Metastatic Cancer | Colon Cancer | Thyroid Cancer | Renal Cancer | Parathyroid Neoplasms | Endocrine Cancer | Hypercalcemia of MalignancyUnited States, Italy, France, Canada, Poland
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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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Center for International Blood and Marrow Transplant...National Marrow Donor ProgramRecruitingAutoimmune Diseases | Hodgkin Lymphoma | Hematologic Malignancies | Acute Lymphoblastic Leukemia (ALL) | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Inherited Disorders of Metabolism | Inherited Abnormalities of Platelets | Histiocytic Disorders | Acute Myelogenous Leukemia (AML or ANLL) | Other Acute... and other conditionsUnited States
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ShireTakeda Development Center Americas, Inc.RecruitingHereditary Angioedema (HAE)France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
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Dana-Farber Cancer InstituteRecruitingLung Cancer | Cirrhosis | Barrett Esophagus | Hematologic Malignancy | Non-Alcoholic Fatty Liver Disease | Cancer Risk | Vulvar Intraepithelial Neoplasia | Childhood Cancer Survivors | Lung; Node | Non Alcoholic Steatohepatitis | Osteochondroma | Cancer Predisposition Syndrome | Hereditary Cancer Prediction | Adult... and other conditionsUnited States
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NestléNestec Ltd.; Regulatory and Clinical Research Institute Inc; Cytel Inc.TerminatedStroke | Multiple Sclerosis | Parkinson Disease | Dementia | Alzheimer Disease | Oropharyngeal DysphagiaUnited States, Finland
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CENTOGENE GmbH RostockCompletedAbdominal Pain | Functional Abdominal PainUnited Kingdom, Germany, Turkey, Poland, Italy, Japan
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Southwest Oncology GroupNational Cancer Institute (NCI)CompletedMultiple Myeloma | Amyloidosis | Waldenstrom's Macroglobulinemia | Monoclonal Gammopathy of Undetermined Significance | Smoldering MyelomaUnited States
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King's College LondonRecruitingLiver Diseases | Heart Diseases | Heart Failure | Stroke | Cerebrovascular Disorders | Epilepsy | Pain | Multiple Sclerosis | Hypertension | Obesity | Diabetes | Cancer | Arthritis | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Migraine | Hepatitis | Atrial Fibrillation | Asthma | Endometriosis | Dementia | Fibromyalgia | Celiac... and other conditionsUnited Kingdom
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Pharvaris Netherlands B.V.CompletedHereditary 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 conditionsBulgaria, United States, Spain, Israel, Germany, Canada, Czechia, France, Hungary, Italy, Netherlands, Poland, United Kingdom
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National Cancer Institute (NCI)RecruitingLynch Syndrome | Malignant Glioma | Recurrent Childhood Ependymoma | Recurrent Medulloblastoma | Refractory Ependymoma | Refractory Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Refractory Diffuse Intrinsic Pontine Glioma | Recurrent Brain Neoplasm | Refractory Brain Neoplasm | Constitutional...United States, Canada
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Novartis PharmaceuticalsRecruitingGlioblastoma | Astrocytoma | Anaplastic Astrocytoma | Neurofibromatosis Type 1 | Anaplastic Ganglioglioma | Anaplastic Pleomorphic Xanthoastrocytoma | Ganglioglioma | Pleomorphic Xanthoastrocytoma | Diffuse Astrocytoma | Anaplastic Oligodendroglioma | Pilocytic Astrocytoma | Gangliocytoma | Central Neurocytoma | Oligodendroglioma... and other conditionsUnited States, Belgium, Spain, Denmark, Germany, Czechia, Italy, Australia, Russian Federation, France, Brazil, Netherlands, Japan, Canada, Finland, Sweden, United Kingdom, Argentina, Israel
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Novo Nordisk A/SCompletedChronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational AgeIsrael, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
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Bellicum PharmaceuticalsTerminatedLymphoma, Non-Hodgkin | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Myelodysplastic Syndrome | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Osteopetrosis | Leukemia, Acute Myeloid (AML), ChildUnited Kingdom, Italy
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National Cancer Institute (NCI)WithdrawnHematopoietic and Lymphoid Cell Neoplasm | Lynch Syndrome | Refractory Lymphoma | Refractory Malignant Solid Neoplasm | Recurrent Lymphoma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma | Refractory Neuroblastoma | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary Central... and other conditionsUnited States, Canada
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National Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditionsUnited States
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Roswell Park Cancer InstituteCompletedPrimary Myelofibrosis | Polycythemia Vera | Chronic 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 and other conditionsUnited States
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Taproot HealthRecruitingMelanoma | Sarcoma | Kidney Cancer | Adenocarcinoma | Renal Cell Carcinoma | Cervical Cancer | Acute Myeloid Leukemia | Stomach Cancer | Breast Cancer | Head and Neck Neoplasms | Nasopharyngeal Carcinoma | Glioblastoma | Astrocytoma | Multiple Myeloma | Colorectal Cancer | Pancreatic Cancer | Esophageal Cancer | Small Bowel Cancer and other conditionsUnited States
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Novoic LimitedKing's College LondonCompletedParkinson Disease | Motor Neuron Disease | Amyotrophic Lateral Sclerosis | Alzheimer Disease | Bipolar Disorder | Mild Cognitive Impairment | Major Depressive Disorder | Frontotemporal Dementia | Primary Progressive Aphasia | Dementia, Vascular | Dementia With Lewy BodiesUnited Kingdom
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Natera, Inc.Children's Hospital of Philadelphia; Montefiore Medical Center; University of... and other collaboratorsCompletedPrader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat SyndromeUnited States, Australia, Ireland, Spain, Sweden, United Kingdom
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Hyunseok Kang, MDNRG OncologyCompletedClinical Stage IV HPV-Mediated (p16-Positive) Oropharyngeal Carcinoma AJCC v8 | Stage III Hypopharyngeal Carcinoma AJCC v8 | Stage III Laryngeal Cancer AJCC v8 | Stage III Lip and Oral Cavity Cancer AJCC v8 | Stage III Oropharyngeal (p16-Negative) Carcinoma AJCC v8 | Stage IV Hypopharyngeal... and other conditionsUnited States
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OMNI Medical Services, LLCOMNI Medical Services IncRecruitingDepression | COVID-19 | Epilepsy | Multiple Sclerosis | Glaucoma | Cancer | Covid19 | Chronic Pain | Parkinson Disease | Inflammatory Bowel Diseases | Hepatitis C | Insomnia | HIV/AIDS | Amyotrophic Lateral Sclerosis | Fibromyalgia | Anxiety | Bipolar Disorder | Seizures | Crohn Disease | Traumatic Brain Injury | Ulcerative Colitis | Sickle... and other conditionsUnited States
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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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National Cancer Institute (NCI)Active, not recruitingLynch Syndrome | Colon Mucinous Adenocarcinoma | Colon Signet Ring Cell Adenocarcinoma | Stage IIA Colon Cancer AJCC v7 | Stage IIB Colon Cancer AJCC v7 | Stage IIC Colon Cancer AJCC v7United States, Canada, Puerto Rico, Peru, South Africa
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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingRenal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Chronic 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... and other conditionsUnited States
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City of Hope Medical CenterTerminatedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Iron Overload | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditionsUnited States
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Wake Forest University Health SciencesNational Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Burkitt Lymphoma | Secondary... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingMelanoma | Neuroblastoma | Pancreatic Ductal Adenocarcinoma | Refractory Malignant Solid Neoplasm | Endometrial Carcinoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Malignant Solid Neoplasm | Recurrent Non-Hodgkin Lymphoma | Recurrent Osteosarcoma | Refractory Ewing Sarcoma | Refractory... and other conditionsUnited States
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Adult Acute Myeloid 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 Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma and other conditionsUnited States
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City of Hope Medical CenterNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid... and other conditionsUnited States
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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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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)CompletedStage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic 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... and other conditionsUnited States, Italy
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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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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal... and other conditionsUnited States
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)CompletedHIV Infection | Unspecified Childhood Solid Tumor, Protocol Specific | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal... and other conditionsUnited States