Clinical Trials on Ornithine Carbamoyltransferase Deficiency Disease

Bile Duct Adenocarcinoma | Fanconi Anemia Complementation Group Gene Mutation | Metastatic Bile Duct Carcinoma | PTEN Gene Deletion

United States

Sarcoma | Pancreatic Adenocarcinoma | Ewing Sarcoma | Gastric Adenocarcinoma | Chondrosarcoma | Solid Tumors | Malignant Pleural Mesothelioma | Colorectal Adenocarcinoma | GIST | SDH-deficient Solid Tumors

United States

Melanoma | Breast Cancer | Head and Neck Cancer | Pancreatic Cancer | Lung Cancer | Endometrial Cancer | Solid Tumors | Renal Cell Carcinoma (RCC) | Colorectal Cancer (CRC) | UC (Urothelial Cancer) | MMR-deficient Tumors

United States

Autoimmune Diseases | Systemic Lupus Erythematosus | Primary Immunodeficiency | Autoimmune Hepatitis | Juvenile Idiopathic Arthritis | IBD | Autoimmune Rheumatologic Disease | Autoimmune Diabetes | APECED | IPEX | Hemophagocytic Lymphohistiocytoses | Autoimmune Lymphoproliferative Syndrome | Autoimmune Thrombocytopenia and other conditions

Melanoma | Kidney Cancer | Renal Cell Carcinoma | Hepatocellular Carcinoma | Colorectal Cancer | NSCLC | Non Small Cell Lung Cancer | Skin Cancer | Metastasis | Advanced Solid Tumor | Advanced Cancer | Renal Cell Cancer | Hepatocellular Cancer | Mismatch Repair Deficiency | Pleural Mesothelioma | MSI-High | Anaplastic Lymphoma... and other conditions

United States

Melanoma | Renal Cell Carcinoma | Cervical Cancer | Hepatocellular Carcinoma | Non Small Cell Lung Cancer | Mesothelioma | Squamous Cell Carcinoma of Head and Neck | Esophageal Squamous Cell Carcinoma | Urothelial Carcinoma | Microsatellite Instability High | Mismatch Repair Deficiency

United States

Epithelioid Sarcoma | Atypical Teratoid/Rhabdoid Tumor | Malignant Rhabdoid Tumor | Rhabdoid Tumor of the Kidney | Chordoma (Poorly Differentiated or De-differentiated) | Other INI1 Negative Tumors (With PI Approval) | Other SMARCA4-deficient Malignant Tumors (With PI Approval)

United States

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

United States

Sarcoma | Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Myelodysplastic Syndromes | Leukemia | Fanconi Anemia | Neuroblastoma | Childhood Langerhans Cell Histiocytosis

United States

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States

Mucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy Syndrome

United States

Non-Small Cell Lung Cancer | DNA Repair-Deficiency Disorders | Head and Neck Squamous Cell Carcinoma | Urothelial Carcinoma Bladder | Classical Hodgkin Lymphoma | Malignant Melanoma

United States

Thrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell Aplasia

United States

Melanoma | Lymphoma | Kidney Cancer | Leukemia | Cancer | Breast Cancer | Head and Neck Cancer | Ovarian Cancer | Lynch Syndrome | Lung Cancer | Colon Cancer | Meningioma | BRCA1 Mutation | BRCA2 Mutation | Myeloma | Mutation | Uterine Cancer | Cowden Syndrome

Canada

Fanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic Anemia

United States

Tuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Xeroderma Pigmentosum | Birt-Hogg-Dube Syndrome | Palmoplantar Keratoses

Netherlands

Melanoma | Hepatocellular Carcinoma (HCC) | Renal Cell Carcinoma (RCC) | Colorectal Carcinoma (CRC) | Microsatellite Instability - High (MSI-H) | Mismatch Repair Deficient (dMMR)

United States, Brazil, Canada, Australia, Slovakia, Norway, Mexico

Pancreatic 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 conditions

United States

Mucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell Disease

United States

Lymphoproliferative Disorders | Stem Cell Transplant Complications | Epstein-Barr Virus (EBV) Infections | EBV+ Associated Lymphoma | EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD) | Epstein-Barr Viremia | Lymphoma, AIDS-related | Epstein-Barr Virus-associated Lymphoproliferative... and other conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States

Melanoma | Renal Cell Carcinoma | Cervical Cancer | Small Cell Lung Cancer | Gastric Cancer | Colorectal Cancer | Esophageal Cancer | Non-small Cell Lung Cancer | Head and Neck Squamous Cell Carcinoma | Esophageal Squamous Cell Carcinoma | Urothelial Carcinoma | Merkel Cell Carcinoma | Gastroesophageal Junction... and other conditions

United States

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom

HIV Infections | Acquired Immunodeficiency Syndrome | Sexually Transmitted Diseases, Viral | Pneumonia | Psoriasis | Weight Loss | Thrombocytopenia | Leukopenia | Dermatitis, Seborrheic | Herpes Zoster | Lymphadenopathy | Uterine Cervical Dysplasia | Primary Health Care | Diarrhea Chronic | Candida Infection | Infectious... and other conditions

Netherlands

Myelodysplastic Syndromes | Primary Immunodeficiency Diseases | Hemoglobinopathies | Chronic Myeloid Leukemia | Cytopenia | Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Acute Myeloid Leukemia in Remission | Hemophagocytic Lymphohistiocytoses | Acute Lymphoblastic Leukemia in Remission | Severe...

United States

Granuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler Disease

United States

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 Acute Myeloid Leukemia With t(8;21)(q22;q22) | Fanconi Anemia | Adult Acute... and other conditions

United States

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore

Malignant Solid Neoplasm | Lynch Syndrome | Hematopoietic and Lymphoid System Neoplasm | Breast Ductal Carcinoma In Situ | Hereditary Neoplastic Syndrome | BRCA1/2-Associated Hereditary Breast and Ovarian Cancer Syndrome

United States

Nutrition Disorders | Chronic Pain | Postoperative Pain | Anesthesia, Local | Postoperative Cognitive Dysfunction | Scoliosis | Coagulation Disorder | Acute Pain | Neuropathic Pain | Anesthesia | Postoperative Nausea and Vomiting | Ventilator-Induced Lung Injury | Intraoperative Complications | Hyperalgesia | Regional... and other conditions

Ukraine

Niemann-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

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

Melanoma | Renal Cell Carcinoma | Cervical Cancer | Stomach Cancer | Small Cell Lung Cancer | Hepatocellular Carcinoma | Gastric Cancer | Esophageal Cancer | Ovarian Cancer | NSCLC | Mesothelioma | Squamous Cell Carcinoma of the Head and Neck | Non-small Cell Lung Cancer | Gastroesophageal Junction Cancer | Urothelial... and other conditions

United States

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

Stage IVA Lung Cancer AJCC v8 | Stage IVB Lung Cancer AJCC v8 | Refractory Pancreatic Carcinoma | Stage II Pancreatic Cancer AJCC v8 | Stage III Pancreatic Cancer AJCC v8 | Stage IV Pancreatic Cancer AJCC v8 | Lung Non-Small Cell Carcinoma | Stage III Lung Cancer AJCC v8 | Stage IV Lung Cancer AJCC v8 | Stage IIIA Lung Cancer AJCC... and other conditions

United States

Melanoma | Renal Cell Carcinoma | Cervical Cancer | Small Cell Lung Cancer | Hepatocellular Carcinoma | Gastric Cancer | Colorectal Cancer | Non-Small Cell Lung Cancer | Head and Neck Squamous Cell Carcinoma | Urothelial Carcinoma | Merkel Cell Carcinoma | Microsatellite Instability | Mismatch Repair Deficiency

United States

Melanoma | Renal Cell Carcinoma | Hodgkin Lymphoma | Gastric Adenocarcinoma | Non Small Cell Lung Cancer | Squamous Cell Carcinoma of the Head and Neck | Diffuse Large B Cell Lymphoma | Urothelial Carcinoma | Gastroesophageal Junction Adenocarcinoma | Squamous Cell Carcinoma of the Skin | Squamous Cell Carcinoma... and other conditions

United States, Canada, Belgium, Spain

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

Chronic Myelomonocytic Leukemia | Fanconi Anemia | Childhood Acute Erythroleukemia (M6) | Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute... and other conditions

United States

Hepatocellular Carcinoma | Triple Negative Breast Cancer | Non-small Cell Lung Cancer | Soft-tissue Sarcoma | Urothelial Carcinoma | Thyroid Cancer | Gastro-enteropancreatic Neuroendocrine Tumor | Biliary Tract Cancer | HPV-Related Carcinoma | Solid Tumor, Adult | Mesotheliomas Pleural | GIST | Renal Carcinoma | Colorectal... and other conditions

France

HIV Infection | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma | Recurrent Adult Lymphoblastic Lymphoma | Recurrent Grade 1 Follicular Lymphoma | Recurrent Grade 2 Follicular... and other conditions

United States

Melanoma | Follicular Lymphoma | Carcinoma | Myelodysplastic Syndromes | Leukemia | Cancer | Breast Cancer | Myeloproliferative Disorders | Glioblastoma | Myelofibrosis | Ovarian Cancer | Mantle Cell Lymphoma | Marginal Zone Lymphoma | Neuroendocrine Tumors | Non Small Cell Lung Cancer | COVID | Non Hodgkin Lymphoma | Cholang... and other conditions

United States

Melanoma | Sarcoma | Kidney Cancer | Leukemia | Breast Cancer | Laryngeal Cancer | Hypopharyngeal Cancer | Nasopharyngeal Cancer | Multiple Myeloma | Gastric Cancer | Pancreatic Cancer | Esophageal Cancer | Small Intestine Cancer | Ovarian Cancer | Lynch Syndrome | Lung Cancer | Rectal Cancer | Prostate Cancer | Gastrointestinal... and other conditions

United States

Primary 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 conditions

United States

HIV Infection | Unspecified Adult Solid Tumor, Protocol Specific | Stage IV Breast Cancer | Stage IV Ovarian Epithelial Cancer | Recurrent Breast Cancer | Recurrent Ovarian Epithelial Cancer | Stage IV Gastric Cancer | Recurrent Gastric Cancer | Recurrent Metastatic Squamous Neck Cancer With Occult... and other conditions

United States

HIV Infection | Metastatic Renal Cell Carcinoma | Anatomic Stage III Breast Cancer AJCC v8 | Recurrent Ovarian Carcinoma | Metastatic Lung Non-Small Cell Carcinoma | Anatomic Stage IV Breast Cancer AJCC v8 | Metastatic Melanoma | Recurrent Head and Neck Carcinoma | Recurrent Renal Cell Carcinoma | Advanced... and other conditions

United States

HIV 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 conditions

United States

Primary 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 conditions

United States