Clinical Trials on Nonsense Mutations

Total 935 results

  • Nuvectis Pharma, Inc.
    GOG Foundation; European Network of Gynaecological Oncological Trial Groups...
    Recruiting
    A Phase 1 Clinical Study of NXP800 in Subjects With Advanced Cancers and Expansion in Subjects With Ovarian Cancer
    Ovarian Cancer | Ovarian Endometrioid Adenocarcinoma | Advanced Solid Tumor | Ovarian Clear Cell Carcinoma | Ovarian Clear Cell Adenocarcinoma | Ovarian Clear Cell Tumor | Ovarian Endometrioid Tumor | ARID1A Gene Mutation
    United States, United Kingdom
  • Royal Marsden NHS Foundation Trust
    Recruiting
    Inherited Cancer Early Diagnosis (ICED) Study (ICED)
    Lynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene Mutation
    United Kingdom
  • Memorial Sloan Kettering Cancer Center
    Recruiting
    A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors
    Solid Tumor | Rhabdoid Tumor | Wilms Tumor | Nephroblastoma | Malignant Peripheral Nerve Sheath Tumors | MPNST | XPO1 Gene Mutation
    United States
  • Mohamed Abdel-Rahman
    Recruiting
    Frequency and Clinical Phenotype of BAP1 Hereditary Predisposition Syndrome
    Renal Cell Carcinoma | Hepatocellular Carcinoma | Mesothelioma | Cholangiocarcinoma | Uveal Melanoma | Cutaneous Melanoma | Meningioma Atypical | BAP1 Gene Mutation
    United States
  • Joshua Mann, MPH
    National Organization for Rare Disorders
    Recruiting
    MyVHL: Patient Natural History Study (MyVHL)
    Von Hippel-Lindau Disease | Hereditary Leiomyomatosis and Renal Cell Cancer | Birt-Hogg-Dube Syndrome | SDHB Gene Mutation
    United States
  • Apollomics Inc.
    Recruiting
    APL-101 Study of Subjects With NSCLC With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors (SPARTA)
    Gastric Cancer | Pancreatic Cancer | NSCLC | Lung Cancer | Brain Tumor | Glioblastoma Multiforme | Advanced Cancer | Colon Cancer | Solid Tumors | Thyroid Cancer | Renal Cancer | Gastroesophageal Junction Adenocarcinoma | EGFR Gene Mutation | MET Amplification | HGF | MET Alteration | MET Fusion | Exon 14 Skipping
    United States, Spain, France, United Kingdom, Taiwan, Australia, Finland, Italy, Singapore, Hungary, Canada, Puerto Rico, Russian Federation
  • Peloton Therapeutics, Inc., a subsidiary of Merck...
    Active, not recruiting
    A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)
    VHL - Von Hippel-Lindau Syndrome | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL-Associated Renal Cell Carcinoma | VHL-Associated Clear Cell Renal Cell Carcinoma
    United States, Denmark, United Kingdom, France
  • Peloton Therapeutics, Inc., a subsidiary of Merck...
    National Institutes of Health (NIH)
    Completed
    PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma
    Clear 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 of
    United States
  • Abramson Cancer Center at Penn Medicine
    Enrolling by invitation
    Sequential EHR Based Interventions to Increase Genetic Testing for Breast and Ovarian Cancer Predisposition
    Ovarian Cancer | Genetic Predisposition to Disease | Breast Cancer Female | Hereditary Diseases | Gene Mutation-Related Cancer | Hereditary Breast and Ovarian Cancer | Hereditary Cancer Syndrome
    United States
  • Children's Hospital of Philadelphia
    Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators
    Recruiting
    The Myelin Disorders Biorepository Project (MDBP)
    Mucopolysaccharidoses | Leukoencephalopathies | 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... and other conditions
    United States
  • Calithera Biosciences, Inc
    Completed
    Study of the Glutaminase Inhibitor CB-839 in Solid Tumors
    Renal Cell Carcinoma | Non Small Cell Lung Cancer | Mesothelioma | Solid Tumors | Triple-Negative Breast Cancer | Fumarate Hydratase (FH)-Deficient Tumors | Succinate Dehydrogenase (SDH)-Deficient Gastrointestinal Stromal Tumors (GIST) | Succinate Dehydrogenase (SDH)-Deficient Non-gastrointestinal... and other conditions
    United States
  • Biomea Fusion Inc.
    Recruiting
    Study of Covalent Menin Inhibitor BMF-219 in Adult Patients With KRAS Driven Non-Small Cell Lung Cancer, Pancreatic Cancer, and Colorectal Cancer
    Colorectal Cancer | Pancreatic Cancer | NSCLC | Non Small Cell Lung Cancer | Stage III Pancreatic Cancer | Stage IV Pancreatic Cancer | CRC | KRAS Mutation-Related Tumors | Relapsed Cancer | Refractory Cancer | Stage IV Non-small Cell Lung Cancer | Stage IV Colorectal Cancer | Stage III Non-small Cell Lung Cancer | Stage III NSCLC and other conditions
    United States, Korea, Republic of
  • M.D. Anderson Cancer Center
    National Cancer Institute (NCI)
    Active, not recruiting
    Olaparib in Treating Patients With Newly Diagnosed BRCA-Mutant Ovarian, Primary Peritoneal, or Fallopian Cancer Before Surgery
    BRCA1 Gene Mutation | BRCA2 Gene Mutation | Primary Peritoneal High Grade Serous Adenocarcinoma | Stage III Fallopian Tube Cancer AJCC v8 | Stage III Ovarian Cancer AJCC v8 | Stage III Primary Peritoneal Cancer AJCC v8 | Stage IIIA Fallopian Tube Cancer AJCC v8 | Stage IIIA Ovarian Cancer AJCC v8 | Stage... and other conditions
    United States
  • Academic and Community Cancer Research United
    National Cancer Institute (NCI)
    Recruiting
    Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations
    Bile Duct Adenocarcinoma | Fanconi Anemia Complementation Group Gene Mutation | Metastatic Bile Duct Carcinoma | PTEN Gene Deletion
    United States
  • Memorial Sloan Kettering Cancer Center
    Recruiting
    A Study of Avutometinib for People With Solid Tumor Cancers
    Solid Tumor | Neuroblastoma | Solid Tumor, Adult | Primary Brain Tumor | Solid Carcinoma | Low-grade Glioma | Central Nervous System Tumor | Refractory Cancer | CNS Tumor, Adult | CNS Tumors | NF1 | Plexiform Neurofibroma | CNS Tumor, Childhood | Optic Pathway Gliomas | MAP Kinase Family Gene Mutation
    United States
  • NeoTX Therapeutics Ltd.
    AstraZeneca
    Active, not recruiting
    Naptumomab Estafenatox in Combination With Durvalumab in Subjects With Selected Advanced or Metastatic Solid Tumors
    Melanoma | Renal Cell Carcinoma | Hepatocellular Carcinoma | Pancreatic Adenocarcinoma | Ovarian Cancer | NSCLC | Prostate Cancer | Mesothelioma | Bladder Cancer | Triple Negative Breast Cancer | Endometrial Cancer | Colorectal Cancer Metastatic | Head and Neck Squamous Cell Carcinoma | Urothelial Cancer | Cervical Squamous... and other conditions
    Israel
  • Epizyme, Inc.
    Completed
    A Study of Tazemetostat in Adult Participants With Soft Tissue Sarcoma
    Synovial Sarcoma | Epithelioid Sarcoma | Renal Medullary Carcinoma | Malignant Rhabdoid Tumors (MRT) | Rhabdoid Tumors of the Kidney (RTK) | Atypical Teratoid Rhabdoid Tumors (ATRT) | Selected Tumors With Rhabdoid Features | INI1-negative Tumors | Malignant Rhabdoid Tumor of Ovary | Poorly Differentiated... and other conditions
    France, United States, Australia, Taiwan, Belgium, United Kingdom, Canada, Germany, Italy
  • Khondrion BV
    Julius Clinical; ProPharma Group; Certara
    Completed
    The KHENEREXT Study
    Mitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS) | Chronic Progressive External Ophthalmoplegia (CPEO)
    Netherlands, Denmark, United Kingdom, Germany
  • QuantumLeap Healthcare Collaborative
    Byondis B.V.
    Withdrawn
    ISPY-P1.01:Evaluating the Safety of Weekly Paclitaxel With Trastuzumab Duocarmazine (SYD985) in Patients With Metastatic Cancer
    Ovarian Cancer | Metastatic Cancer | Metastatic Breast Cancer | Bladder Cancer | Triple Negative Breast Cancer | HER2-positive Breast Cancer | Gastroesophageal Adenocarcinoma | Ovarian Carcinoma | HER2-positive Gastric Cancer | Metastatic Gastrointestinal Carcinoid Tumor | HER2-positive Metastatic Breast... and other conditions
    United States
  • Roswell Park Cancer Institute
    Active, not recruiting
    Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
    Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions
    United States
  • City of Hope Medical Center
    National Cancer Institute (NCI)
    Active, not recruiting
    Vaccine Therapy and Pembrolizumab in Treating Patients With Solid Tumors That Have Failed Prior Therapy
    Melanoma | Soft Tissue Sarcoma | Renal Cell Carcinoma | Hepatocellular Carcinoma | Head and Neck Squamous Cell Carcinoma | Bladder Carcinoma | Colon Carcinoma | Pancreatic Carcinoma | Rectal Carcinoma | Estrogen Receptor Negative | HER2/Neu Negative | Progesterone Receptor Negative | Triple-Negative Breast Carcinoma and other conditions
    United States
  • 23andMe, Inc.
    Active, not recruiting
    A Phase 1/2a Study of 23ME-00610 in Patients With Advanced Solid Malignancies
    Clear Cell Renal Cell Carcinoma | Fallopian Tube Cancer | Solid Tumor | Neuroendocrine Tumors | Epithelial Ovarian Cancer | Primary Peritoneal Carcinoma | Extensive-stage Small-cell Lung Cancer | MSI-H Cancer | Cancer With A High Tumor Mutational Burden
    United States, Canada
  • McGill University Health Centre/Research Institute...
    Recruiting
    Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
    Peroxisome Biogenesis Disorder | Zellweger Spectrum Disorder | RCDP - Rhizomelic Chondrodysplasia Punctata | D-Bifunctional Protein Deficiency | Alpha-Methylacyl-CoA Racemase Deficiency | Peroxisomal Acyl-CoA Oxidase Deficiency | Peroxisomal Acyl-CoA Oxidase 2 Deficiency | ATP Binding Cassette Subfamily... and other conditions
    Canada
  • City of Hope Medical Center
    National Cancer Institute (NCI)
    Completed
    Trametinib and Trifluridine and Tipiracil Hydrochloride in Treating Patients With Colon or Rectal Cancer That is Advanced, Metastatic, or Cannot Be Removed by Surgery
    Stage IV Colorectal Cancer AJCC v7 | Stage IVA Colorectal Cancer AJCC v7 | Stage IVB Colorectal Cancer AJCC v7 | Metastatic Colorectal Carcinoma | Stage III Colon Cancer AJCC v7 | Stage III Rectal Cancer AJCC v7 | Stage IIIA Colon Cancer AJCC v7 | Stage IIIA Rectal Cancer AJCC v7 | Stage IIIB Colon... and other conditions
    United States
  • 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
  • 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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