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Clinical Trials on Substituted / Weaned or Not in United Kingdom
Total 404 results
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University of Medicine and Dentistry of New JerseyNational Cancer Institute (NCI)WithdrawnRecurrent 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 | Recurrent... and other conditionsUnited States
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)TerminatedRecurrent Adult Acute Myeloid Leukemia | Recurrent Adult Burkitt Lymphoma | 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;q22) | Adult... and other conditionsUnited States
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Xencor, Inc.ICON plcActive, not recruitingRenal Cell Carcinoma | Nasopharyngeal Carcinoma | Small Cell Lung Cancer | Hepatocellular Carcinoma | Advanced Solid Tumors | Non-small Cell Lung Carcinoma | Squamous Cell Carcinoma of the Head and Neck | Cervical Carcinoma | Endometrial Carcinoma | Urothelial Carcinoma | Colorectal Carcinoma | Pancreatic Carcinoma and other conditionsUnited States
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University of ChicagoNational Cancer Institute (NCI)CompletedRecurrent 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 | Recurrent... and other conditionsUnited States
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City of Hope Medical CenterActive, not recruitingUnspecified Childhood Solid Tumor, Protocol Specific | Solid Tumor | Ewing Sarcoma | Unspecified Adult Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Adult Brain Tumor | Recurrent... and other conditionsUnited 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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Wake Forest University Health SciencesNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | 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 and other conditionsUnited States
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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National Cancer Institute (NCI)Active, not recruitingRecurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Medulloblastoma | Recurrent Neuroblastoma | Recurrent Non-Hodgkin... and other conditionsUnited States, Puerto Rico
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Case Comprehensive Cancer CenterCompletedChronic 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 Adult Diffuse Large Cell... and other conditionsUnited States
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National Cancer Institute (NCI)Children's Oncology GroupActive, not recruitingRefractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent Medulloblastoma | Recurrent Neuroblastoma and other conditionsUnited States, Puerto Rico, Australia
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)RecruitingRecurrent Hodgkin Lymphoma | Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Recurrent Adult Acute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Secondary Acute Myeloid Leukemia | Myelodysplastic Syndrome | Recurrent Acute Myeloid Leukemia | Myelodysplastic Syndrome... 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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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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Fred Hutchinson Cancer CenterCompletedPrimary Myelofibrosis | Essential Thrombocythemia | 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... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Lymphoblastic Leukemia | Down Syndrome | Philadelphia Chromosome Positive | Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | Childhood B Acute Lymphoblastic Leukemia | Adult B Lymphoblastic Lymphoma | Ann Arbor Stage I B Lymphoblastic Lymphoma | Ann Arbor Stage... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedRecurrent 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 | Recurrent... and other conditionsUnited States, Italy
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National Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Recurrent Childhood Soft... and other conditionsUnited States
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National Cancer Institute (NCI)Active, not recruitingAdvanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Histiocytic and Dendritic Cell Neoplasm | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditionsUnited States, Puerto Rico
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National Cancer Institute (NCI)Active, not recruitingRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma and other conditionsUnited States, Puerto Rico
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)TerminatedStage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | 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... and other conditionsUnited States
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National Cancer Institute (NCI)Active, not recruitingAdvanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent Medulloblastoma | Recurrent... and other conditionsUnited States, Puerto Rico
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National Cancer Institute (NCI)Children's Oncology GroupRecruitingMalignant Solid Neoplasm | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditionsUnited States, Puerto Rico, Australia
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Martin, PaulNational 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 | 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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National Cancer Institute (NCI)Children's Oncology GroupActive, not recruitingRefractory Lymphoma | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Histiocytic and Dendritic Cell Neoplasm | Recurrent Langerhans Cell Histiocytosis | Recurrent Lymphoma | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant... and other conditionsUnited States, Puerto Rico, Australia
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedClear Cell Renal Cell Carcinoma | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma | Recurrent Grade 1 Follicular Lymphoma | Recurrent... and other conditionsUnited States, Germany
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Fred Hutchinson Cancer Research Center/University...National Cancer Institute (NCI)CompletedDepression | Fatigue | Primary Myelofibrosis | 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 Tissue | Nodal Marginal Zone... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingCastleman Disease | Lymphangioleiomyomatosis | Digestive System Carcinoma | Malignant Neoplasm | Recurrent Breast Carcinoma | Recurrent Thyroid Gland Carcinoma | Metastatic Urothelial Carcinoma | Malignant Female Reproductive System Neoplasm | Neurofibromatosis Type 2 | Recurrent Adult Soft Tissue Sarcoma 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
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... 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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National Cancer Institute (NCI)Active, not recruitingMalignant Solid Neoplasm | Recurrent Melanoma | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Medulloblastoma | Recurrent Neuroblastoma | Recurrent Non-Hodgkin... and other conditionsUnited States, Puerto Rico
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | 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, Italy
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National Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Melanoma | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Colon Cancer | Recurrent Wilms Tumor and Other Childhood... and other conditionsUnited States
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Fred Hutchinson Cancer CenterCompletedPrimary Myelofibrosis | 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 Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditionsUnited States
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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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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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Giselle ShollerTranslational Genomics Research Institute; Dell, Inc.CompletedGlioma | Renal Cell Carcinoma | Osteosarcoma | Ependymoma | Medulloblastoma | Neuroblastoma | Meningioma | Craniopharyngioma | Choroid Plexus Neoplasms | Rhabdomyosarcoma | Wilms Tumor | Ewings Sarcoma | Malignant Rhabdoid Tumor | Liver Tumors | Germ Cell Tumors | Dysembryoplastic Neuroepithelial Tumor | Clear Cell Sarcoma | ... and other conditionsUnited States, Lebanon
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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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National Cancer Institute (NCI)CompletedChronic 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 Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse... and other conditionsUnited States
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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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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...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, Germany, Italy
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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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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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National Cancer Institute (NCI)CompletedStage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Recurrent Adult Acute Myeloid Leukemia | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent Adult Immunoblastic Large Cell Lymphoma and other conditionsUnited States
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National Cancer Institute (NCI)CompletedHIV Infection | Clear Cell Renal Cell Carcinoma | Primary 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... and other conditionsUnited States
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Actuate Therapeutics Inc.Active, not recruitingSarcoma | Cancer | Breast Neoplasms | Neoplasm Metastasis | Pancreatic Neoplasms | Pancreatic Cancer | Pancreatic Adenocarcinoma | Bone Cancer | Malignant Glioma | Bone Metastases | Pancreas Cancer | Renal Cancer | Refractory Non-Hodgkin Lymphoma | Bone Neoplasm | Neoplasm, Breast | Refractory Cancer | Refractory Neoplasm | Resistant... and other conditionsUnited States, Belgium, Canada, Spain, France, Netherlands, Portugal
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsCompletedExtranodal 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 | Recurrent Adult Immunoblastic Large Cell Lymphoma and other conditionsUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States