Clinical Trials on Congenital Abnormalities in Austria

Wolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-Deficiency

France, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia

Pompe Disease

United States

Fabry Disease

Italy, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland

Glycogen Storage Disease Type II | Pompe Disease

Hungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more

Cystic Fibrosis

United States

Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Recurrent Kidney Wilms Tumor | Anaplastic Kidney Wilms Tumor

United States, Canada, Puerto Rico, Australia, New Zealand, Saudi Arabia

Migraine | Migraine Without Aura | Migraine With Aura

United States

Gout | Moderate Renal Impairment

United States

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom

Low Grade Glioma | Low Grade Astrocytoma | Metastatic Low Grade Astrocytoma | Metastatic Low Grade Glioma

United States, Canada, Puerto Rico

Sphingomyelin Lipidosis

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom

Elevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVD

United States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom

Hereditary Angioedema

United States, Australia, Israel, Canada, Hungary, Romania, Russian Federation, South Africa, Ukraine

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal

Gout

United States

Hereditary Angioedema

United States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom

Migraine | Migraine Without Aura | Migraine With Aura

United States

Lennox Gastaut Syndrome (LGS) | Dravet Syndrome (DS)

United States, China, Spain, France, Belgium, Australia, Brazil, Canada, Germany, Greece, Hungary, Italy, Japan, Latvia, Netherlands, Poland, Serbia, Mexico, Russian Federation, Ukraine

Fabry Disease

United States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain

Viral Lung Infection and Acute Respiratory Failure

United States, China, Australia, Czechia, Denmark, Hungary, Israel, Spain, United Kingdom, Canada, Germany, Italy, Argentina, Bulgaria, India, Turkey, Vietnam, Japan, France, Egypt, Malaysia, Taiwan, Thailand, Brazil, Greece, Belgium, Hong... and more

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more

Tic Disorder | Tourette's Disorder

United States, Germany, Spain, Mexico, Romania, Canada, Hungary, Italy, Sweden

Unspecified Childhood Solid Tumor, Protocol Specific | Ewing Sarcoma of Bone | Ewing Sarcoma | Childhood Acute Lymphoblastic Leukemia | Wilms Tumor and Other Childhood Kidney Tumors | Childhood Rhabdomyosarcoma | Childhood Soft Tissue Sarcoma | Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor...

United States, Australia, Canada

Adv Solid Malig - H&N SCC, ATM Pro / Def NSCLC, Gastric, Breast and Ovarian Cancer

Spain, United States, Belgium, United Kingdom, France, Hungary, Canada, Korea, Republic of, Australia

Fallopian Tube Cancer | Epithelial Ovarian Cancer | Primary Peritoneal | Newly Diagnosed | FIGO Stage III-IV | Partial Response | Complete Response

United States, United Kingdom, Korea, Republic of, Russian Federation, Canada, Denmark, Taiwan, New Zealand, Ireland, Japan, Spain, Australia, Israel, Italy, Greece, Sweden, Czechia, Finland, Poland, Belgium, Germany, Romania, Singa... and more

Fabry Disease

Croatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more

Intracranial Meningioma | Recurrent Meningioma | NF2 Gene Mutation

United States

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more

Recurrent Adult Acute Myeloid Leukemia | Adult Acute Monoblastic Leukemia (M5a) | Adult Acute Monocytic Leukemia (M5b) | Adult Acute Myeloblastic Leukemia With Maturation (M2) | Adult Acute Myeloblastic Leukemia Without Maturation (M1) | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities and other conditions

United States, Canada, Australia

Niemann-Pick Disease, Type C1

United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany

Juvenile Myelomonocytic Leukemia | Neurofibromatosis Type 1

United States

Malignant Solid Neoplasm | Neuroendocrine Neoplasm | Myeloproliferative Neoplasm | Desmoid Fibromatosis | Lymphoproliferative Disorder | Carcinoma In Situ | Central Nervous System Neoplasm | Ganglioneuroma | Congenital Mesoblastic Nephroma | Childhood Immature Teratoma | Childhood Langerhans Cell Histiocytosis and other conditions

United States, Puerto Rico, Canada, Australia, New Zealand, Saudi Arabia

Sarcoma | Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Brain and Central Nervous System Tumors | Neuroblastoma | Retinoblastoma

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland

Clear 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 conditions

United States, Canada, Australia, New Zealand, Puerto Rico

Unspecified Childhood Solid Tumor, Protocol Specific | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Secondary Acute Myeloid Leukemia | Childhood Acute Myeloid Leukemia in Remission | Mucositis | Childhood Acute Lymphoblastic Leukemia in Remission | Childhood Chronic Myelogenous... and other conditions

United States, Canada, Australia

Recurrent Thyroid Cancer | Recurrent Childhood Rhabdomyosarcoma | Papillary Thyroid Cancer | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Previously Treated Childhood Rhabdomyosarcoma | Recurrent Childhood Liver Cancer | Childhood Hepatocellular Carcinoma

United States, Canada, Australia

Glioma | 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 conditions

United States, Lebanon

Neurofibromatosis Type 1 | Low Grade Glioma | Visual Pathway Glioma

United States, Canada, Puerto Rico

Lynch Syndrome | Stage III Colon Cancer AJCC v8 | Colon Adenocarcinoma | DNA Repair Disorder

United States, Puerto Rico, Germany

Stage III Adult Soft Tissue Sarcoma | Stage IV Adult Soft Tissue Sarcoma | Stage I Adult Soft Tissue Sarcoma | Stage II Adult Soft Tissue Sarcoma | Dermatofibrosarcoma Protuberans | Adult Angiosarcoma | Adult Epithelioid Sarcoma | Adult Extraskeletal Osteosarcoma | Adult Fibrosarcoma | Adult Leiomyosarcoma and other conditions

United States, Canada, Australia, New Zealand, Puerto Rico

Malignant 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 conditions

United States, Puerto Rico, Australia

Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Previously Treated Childhood Rhabdomyosarcoma | Recurrent Childhood Soft Tissue Sarcoma | Adult Rhabdomyosarcoma | Neuroectodermal Tumor and other conditions

United States, Canada, Australia

Glioblastoma | 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 conditions

United States, Belgium, Spain, Denmark, Germany, Czechia, Italy, Australia, Russian Federation, France, Brazil, Netherlands, Japan, Canada, Finland, Sweden, United Kingdom, Argentina, Israel

Refractory 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 conditions

United States, Puerto Rico, Australia

Malignant Glioma | Rhabdoid Tumor | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditions

United States, Puerto Rico, Australia, Canada

Refractory 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 conditions

United States, Puerto Rico, Australia

Recurrent Neuroblastoma | Recurrent Rhabdomyosarcoma | Wilms Tumor | Recurrent Malignant Peripheral Nerve Sheath Tumor | Recurrent Synovial Sarcoma | Pleuropulmonary Blastoma

United States

Lynch 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 v7

United States, Canada, Puerto Rico, Peru, South Africa

Soft Tissue Sarcoma | Osteosarcoma | Ewing Sarcoma | Malignant Glioma | Ependymoma | Rhabdoid Tumor | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Rhabdomyosarcoma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma | Refractory Neuroblastoma | Refractory Non-Hodgkin Lymphoma and other conditions

United States, Puerto Rico

Recurrent 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 conditions

United States, Puerto Rico