- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in Australia
Total 966 results
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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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Bristol-Myers SquibbCompletedChronic Myeloid Leukemia | Leukemia, Lymphoblastic, Acute, Philadelphia-positiveNetherlands, United States, Italy, Argentina, Austria, Brazil, Canada, Germany, Peru, United Kingdom, France, Korea, Republic of, Sweden, Finland, Australia, Israel, Belgium, Denmark, Norway, Switzerland
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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CelgeneRecruitingBeta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated MyelofibrosisUnited States, Japan, Israel, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon, Netherlands
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Gilead SciencesCompletedCystic FibrosisUnited States, France, Spain, Germany, Austria, Belgium, Italy, Netherlands, Poland
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Netherlands, Spain, Switzerland, Taiwan, Turkey, United Kingdom, Croatia, Germany, Brazil, France, Japan, Australia, Hungary, Israel, Korea, Republic of, Lithuania, Portugal, Ukraine, Greece, Italy, Denmark, Norway, Canada and more
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, United Kingdom, Croatia, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Italy, Japan, Norway, Bulgaria
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited 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
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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited 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
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Pharvaris Netherlands B.V.Active, not recruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
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Otsuka Pharmaceutical Development & Commercialization...CompletedChronic Kidney Disease | Autosomal Dominant Polycystic Kidney DiseaseAustralia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania
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TakedaRecruitingLennox 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
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Novo Nordisk A/SRecruitingHaemophilia A With Inhibitors | Haemophilia ATaiwan, Canada, Spain, Netherlands, Korea, Republic of, India, United States, United Kingdom, Portugal, Lithuania, Turkey, China, South Africa, Ireland, Italy, France, Japan, Belgium, Germany, Malaysia, Poland, Austria, Bulgaria, Denmark and more
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AstraZenecaRecruitingAdv Solid Malig - H&N SCC, ATM Pro / Def NSCLC, Gastric, Breast and Ovarian CancerSpain, United States, Belgium, United Kingdom, France, Hungary, Canada, Korea, Republic of, Australia
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Otsuka Pharmaceutical Development & Commercialization...Otsuka Pharmaceutical Co., Ltd.CompletedAutosomal Dominant Polycystic Kidney Disease (ADPKD)United States, Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
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Medical University of ViennaMedical University of Graz; Charite University, Berlin, Germany; Hannover Medical... and other collaboratorsCompletedSarcoma | Hematologic Diseases | Leukemia | Brain Tumor | Neurofibromatoses | NeuroblastomaAustria, Germany, Switzerland, Italy
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Bristol-Myers SquibbOno Pharmaceutical Co. LtdRecruitingMetastatic Colorectal CancerUnited States, Belgium, China, Canada, Denmark, France, Norway, Romania, Spain, United Kingdom, Austria, Brazil, Germany, Netherlands, Argentina, Czechia, Ireland, Italy, Turkey, Chile, Greece, Puerto Rico, Australia, Japan
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedUnspecified 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
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | HAEUnited States, Austria, Canada, Czechia, France, Germany, Hungary, North Macedonia, Romania, Spain, United Kingdom
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Novo Nordisk A/SRecruitingHaemophilia A With Inhibitors | Haemophilia AUnited States, Korea, Republic of, France, Spain, Romania, Canada, Taiwan, China, Lithuania, Netherlands, India, South Africa, Russian Federation, Austria, Belgium, Czechia, Denmark, Germany, Ireland, Italy, Japan, Latvia, Malaysia, M... and more
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Gilead SciencesTerminatedCystic Fibrosis | Pseudomonas Aeruginosa Respiratory Tract Infection/ColonizationUnited States, Spain, United Kingdom, Denmark, Belgium, France, Italy, Germany, Israel, Netherlands, Austria, Greece
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European Huntington's Disease NetworkCompletedHuntington Disease | Huntington's DiseaseFinland, United Kingdom, Austria, Belgium, Czechia, Denmark, France, Germany, Italy, Netherlands, Norway, Poland, Portugal, Russian Federation, Spain, Sweden, Switzerland
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The Hospital for Sick ChildrenLabatt Family Heart CentreRecruitingAtrial Flutter | Tachycardia, Supraventricular | Tachycardia, Atrioventricular Nodal Reentry | Tachycardia Atrial | Tachycardia, Reciprocating | Tachycardia, Paroxysmal | Fetal Hydrops | Tachycardia, Atrial EctopicUnited States, Hong Kong, Canada, Netherlands, Sweden, Australia, Brazil, Finland, France, Russian Federation, Spain, Czechia, Switzerland, United Kingdom
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedMyelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative NeoplasmsUnited States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
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Novartis PharmaceuticalsCompletedTuberous Sclerosis Complex-associated Refractory SeizuresUnited States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage III Kidney Wilms Tumor | Stage IV Kidney Wilms TumorUnited States, Canada, Australia, New Zealand, Puerto Rico, Israel, Switzerland
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Novartis PharmaceuticalsCompletedHypereosinophilic Syndrome | Chronic Myelogenous Leukemia | Systemic Mastocytosis | Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive)United States, Poland, Italy, Belgium, Canada, France, Australia, Netherlands, Sweden, Switzerland, United Kingdom, Spain, Korea, Republic of, Austria, Germany, Denmark, Finland, Singapore, Taiwan, Hong Kong, New Zealand, Norway
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Genzyme, a Sanofi CompanyRecruitingMucopolysaccharidosis 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
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms TumorUnited States, Canada, Australia, New Zealand, Puerto Rico, Israel, Switzerland
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Genzyme, a Sanofi CompanyRecruitingGaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency DiseaseHungary, 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
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Otsuka Pharmaceutical Development & Commercialization...Otsuka Pharmaceutical Co., Ltd.CompletedPolycystic Kidney Disease, Autosomal DominantUnited States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
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CENTOGENE GmbH RostockAlnylam PharmaceuticalsRecruitingCardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyGermany, Austria, Switzerland
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Denmark, Austria, Spain, Japan, Brazil, Algeria, Lithuania, Portugal, Russian Federation, Serbia, Turkey, Greece, Hong Kong, Puerto Rico, Hungary, China, Poland
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National Cancer Institute (NCI)RecruitingDown Syndrome | B Acute Lymphoblastic Leukemia | B Lymphoblastic LymphomaUnited States, Canada, Puerto Rico, Australia, New Zealand
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Children's Oncology GroupNational Cancer Institute (NCI); EsPhALL Network/ BFM Study GroupRecruitingAcute Lymphoblastic Leukemia | B Acute Lymphoblastic Leukemia | T Acute Lymphoblastic Leukemia | Mixed Phenotype Acute LeukemiaUnited States, Canada, Puerto Rico, Australia, Finland, New Zealand, Saudi Arabia, Germany, Austria, Belgium, Chile, Czechia, France, Italy, Netherlands, Sweden, Switzerland, Hong Kong, Israel
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Spruce BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Estonia, Germany, Korea, Republic of, Latvia, Lithuania, Poland, Romania, Spain, Sweden, Turkey, Canada, Italy, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, Japan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Puerto Rico, Norway, Korea, Republic of, Bulgaria, Russian...
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National Cancer Institute (NCI)Active, not recruitingB Acute Lymphoblastic Leukemia | Central Nervous System Leukemia | Testicular Leukemia | B Acute Lymphoblastic Leukemia, BCR-ABL1-LikeUnited States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedMedulloblastoma | Nevoid Basal Cell Carcinoma Syndrome | Desmoplastic/Nodular Medulloblastoma | Medulloblastoma With Extensive NodularityUnited States, Canada, Puerto Rico, Australia
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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingStage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Recurrent Kidney Wilms Tumor | Anaplastic Kidney Wilms TumorUnited States, Canada, Puerto Rico, Australia, New Zealand, Saudi Arabia
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National Cancer Institute (NCI)CompletedNeurofibromatosis Type 1 | Recurrent Childhood Pilocytic Astrocytoma | Recurrent Childhood Visual Pathway GliomaUnited States, Canada, Australia, New Zealand
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Hoffmann-La RocheCompletedBasal Cell CarcinomaUnited States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada
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National Cancer Institute (NCI)SuspendedDown Syndrome | Recurrent B Acute Lymphoblastic LeukemiaUnited States, Canada, Puerto Rico, Australia
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedUnspecified 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 conditionsUnited States, Canada, Australia
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EMD Serono Research & Development Institute, Inc.Merck KGaA, Darmstadt, GermanyCompletedFirst Line Non-Small Cell Lung CancerSpain, United States, Hungary, Israel, China, Korea, Republic of, Taiwan, Czechia, Russian Federation, Australia, Brazil, Lebanon, Serbia, Thailand, Canada, Slovakia, Ukraine, Bulgaria, Italy, Netherlands, New Zealand, Poland, Belgium, Turk... and more
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Myeloid Leukemia | Down Syndrome | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | Myeloid Leukemia Associated With Down SyndromeUnited States, Canada, Saudi Arabia, Puerto Rico, Australia, New Zealand
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Novartis PharmaceuticalsCompletedMyelogenous Leukemia, ChronicSouth Africa, Italy, Belgium, Japan, Taiwan, Turkey, Canada, Germany, Spain, Egypt, United States, Singapore, Austria, France, Netherlands, Sweden, United Kingdom, Korea, Republic of, Hong Kong, Thailand, Russian Federation, Brazil, Hungar... and more
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Adult Kidney Wilms Tumor | Beckwith-Wiedemann Syndrome | Childhood Kidney Wilms Tumor | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Stage V Kidney...United States, Canada, Australia, New Zealand, Puerto Rico, Israel
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Reata, a wholly owned subsidiary of BiogenTerminatedChronic Kidney Diseases | Autosomal Dominant Polycystic Kidney | Alport SyndromeUnited States, France, Australia, Japan, Spain, Puerto Rico
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National Cancer Institute (NCI)CompletedRecurrent 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 CarcinomaUnited States, Canada, Australia