Clinical Trials on Activating Mutations

Total 1062 results

  • Children's Hospital of Philadelphia
    Illumina, Inc.
    Active, not recruiting
    LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
    Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions
    United States
  • Diane George
    Active, not recruiting
    CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
    Sickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditions
    United States
  • National Heart, Lung, and Blood Institute (NHLBI)
    National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
    Completed
    National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (GenTAC)
    Marfan Syndrome | Turner Syndrome | Ehlers-Danlos Syndrome | Loeys-Dietz Syndrome | Shprintzen-Goldberg Syndrome | FBN1, TGFBR1, TGFBR2, ACTA2 or MYH11 Genetic Mutation | Bicuspid Aortic Valve Without Known Family History | Bicuspid Aortic Valve With Family History | Bicuspid Aortic Valve With Coarctation and other conditions
    United States
  • M.D. Anderson Cancer Center
    National Cancer Institute (NCI)
    Recruiting
    Personalized NK Cell Therapy in CBT
    Recurrent 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 conditions
    United States
  • St. Jude Children's Research Hospital
    Novartis Pharmaceuticals
    Active, not recruiting
    SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors
    Glioma | Neoplasms | Glioblastoma | Neoplasms, Neuroepithelial | Central Nervous System Neoplasms | Brain Cancer | Brain Tumor | Ependymoma | Medulloblastoma | Anaplastic Astrocytoma | Anaplastic Ganglioglioma | Anaplastic Ependymoma | Anaplastic Oligodendroglioma | Pineoblastoma | Recurrent Medulloblastoma | CNS Tumor | Pediatric... and other conditions
    United States
  • Masonic Cancer Center, University of Minnesota
    Recruiting
    MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
    Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions
    United States
  • Klus Pharma Inc.
    Completed
    Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene
    Cervical Cancer | Head and Neck Cancer | Mucoepidermoid Carcinoma | Pancreatic Cancer | Recurrent Prostate Cancer | Lung Cancer | Rectal Cancer | Solid Tumor | Prostate Cancer | Cholangiocarcinoma | Bile Duct Cancer | Recurrent Ovarian Carcinoma | Bladder Cancer | Skin Cancer | HER2-positive Breast Cancer | Rare Diseases | Colo-rectal Cancer and other conditions
    United States
  • Fred Hutchinson Cancer Center
    Terminated
    Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
    Recurrent Hodgkin Lymphoma | Recurrent Mantle Cell Lymphoma | Recurrent Marginal Zone Lymphoma | Burkitt Lymphoma | Secondary Acute Myeloid Leukemia | Myelodysplastic Syndrome | Refractory Plasma Cell Myeloma | Recurrent Plasma Cell Myeloma | Acute Leukemia in Remission | Acute Undifferentiated Leukemia | Acute Biphenotypic Leukemia and other conditions
    United States
  • Medicenna Therapeutics, Inc.
    Merck Sharp & Dohme LLC
    Recruiting
    A Beta-only IL-2 ImmunoTherapY Study (ABILITY-1)
    Clear Cell Renal Cell Carcinoma | Cervical Cancer | Gastric Cancer | Esophageal Cancer | Ovarian Cancer | Fallopian Tube Cancer | Solid Tumor | Squamous Cell Carcinoma of Head and Neck | Bladder Cancer | Skin Cancer | Triple Negative Breast Cancer | Advanced Solid Tumor | Primary Peritoneal Cancer | Endometrial Carcinoma and other conditions
    United States, Australia, Korea, Republic of, Canada
  • Cure CMD
    Recruiting
    Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)
    Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions
    United States
  • Sanford Health
    National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators
    Recruiting
    Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)
    Mitochondrial 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 conditions
    United States, Australia
  • RTI International
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    Early Check: Expanded Screening in Newborns
    Primary 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 conditions
    United States

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