- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in South Africa
Total 1109 results
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HealthCore-NERINational Heart, Lung, and Blood Institute (NHLBI); FDA Office of Orphan Products... and other collaboratorsCompletedMarfan SyndromeUnited States, Canada, Belgium
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Belgium, Netherlands, United Kingdom, Canada, Germany, Australia, France, Italy, Czechia, Austria, Greece, Sweden
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Proteostasis Therapeutics, Inc.CompletedCystic Fibrosis | Healthy VolunteerUnited States, Canada, Denmark, Germany, Sweden
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Mylan Inc.Cystic Fibrosis FoundationCompletedPseudomonas Aeruginosa in Cystic FibrosisUnited States
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Nuon Therapeutics, Inc.CompletedHyperuricemia | Moderate to Severe GoutUnited States
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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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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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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, Ireland, Italy, Japan, Latvia, Malaysia, Mexico, Po... and more
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Novartis PharmaceuticalsCompletedAcute GoutColombia, Switzerland, Germany, Russian Federation, Singapore, Australia, Belgium, Canada, Estonia, Guatemala, Latvia, Lithuania, Mexico, Norway, Poland, Sweden, Ukraine
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Zogenix International Limited, Inc., a subsidiary...CompletedLennox Gastaut SyndromeUnited States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Spain, Sweden
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Vertex Pharmaceuticals IncorporatedActive, not recruitingCystic FibrosisUnited States, Spain, United Kingdom, Belgium, Netherlands, Denmark, Israel, New Zealand, France, Australia, Ireland, Germany, Sweden, Canada, Czechia, Switzerland, Portugal, Italy, Austria, Hungary, Norway, Poland, Greece
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Australia, Canada, France, United Kingdom, Germany, Spain, Switzerland, Belgium, Israel, Netherlands, Denmark, Italy, Austria, Ireland, Sweden
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Novartis PharmaceuticalsCompletedIron Overload | Sickle Cell Disease | Hemolytic AnemiaUnited States, Canada
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Spruce BiosciencesRecruitingCongenital Adrenal Hyperplasia | 21-OHDUnited States
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GlycoMimetics IncorporatedTerminatedSickle Cell Disease | Sickle Cell Anemia | Vaso-occlusive Crisis | Pain Crisis | Sickle Cell DisordersUnited States, Canada
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Gilead SciencesCystic Fibrosis FoundationCompleted
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Amalia MagaretUniversity of Texas; Cystic Fibrosis Foundation; National Jewish HealthRecruitingPregnancy Related | Cystic FibrosisUnited States
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Anthera PharmaceuticalsCompletedCystic Fibrosis | Exocrine Pancreatic InsufficiencyUnited States
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Armata Pharmaceuticals, Inc.Cystic Fibrosis FoundationCompletedCystic Fibrosis | Pseudomonas Aeruginosa | Lung Infection | Pseudomonas | Lung Infection PseudomonalUnited States
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Nicole HamblettCystic Fibrosis FoundationRecruitingCystic FibrosisUnited States
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, Australia, United Kingdom, Germany, Ireland
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AmgenCompleted
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Nivalis Therapeutics, Inc.Medidata SolutionsCompletedCystic FibrosisUnited States
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, Germany, United Kingdom
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Baxalta now part of ShireTakeda Development Center Americas, Inc.RecruitingVon Willebrand DiseaseUnited States, Belgium, Spain, Italy, Turkey, Germany, Austria, France, Czechia, Russian Federation, Ukraine, United Kingdom, Netherlands
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Regeneron PharmaceuticalsCompleted
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Horizon Pharma USA, Inc.Forest LaboratoriesCompletedTrial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients (TIS)Cystic FibrosisUnited States, Ireland, United Kingdom, France, Germany, Israel
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Vertex Pharmaceuticals IncorporatedCompletedAlpha1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland, Sweden, United Kingdom
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NewAmsterdam PharmaRecruitingDyslipidemias | Hypercholesterolemia | Familial Hypercholesterolemia | High Cholesterol | ASCVDUnited States
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Mast Therapeutics, Inc.CompletedSickle Cell Disease | Vaso-occlusive CrisisUnited States, Belgium, Brazil, Dominican Republic, Jamaica, Jordan, Lebanon, Oman, Panama, Spain, Turkey
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, United Kingdom, New Zealand, Israel, Australia, Ireland, Germany, Sweden, Czechia, Portugal, Hungary
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Lundbeck LLCCompletedEpilepsy | Epilepsy, Generalized | SeizuresUnited States, Belarus, India, Australia, Lithuania
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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SK Life Science, Inc.RecruitingSeizures | Lennox Gastaut SyndromeUnited States, Korea, Republic of, Taiwan, Colombia, Australia, Poland, Serbia, Mexico, Spain, Greece, Italy, Portugal, Germany, Argentina, Israel
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Novo Nordisk A/SCompletedObesity | Metabolism and Nutrition DisorderFrance, United States, Turkey, Italy, Australia, New Zealand, Canada, Netherlands
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CymaBay Therapeutics, Inc.CompletedGoutUnited States, Canada, Georgia
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Baxalta now part of ShireCompletedVon Willebrand DiseaseNetherlands, Spain, United States, Belgium, Australia, United Kingdom, Austria, Italy, Bulgaria, Japan, Poland, Germany, India, Canada, Russian Federation, Sweden
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, France, Germany
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Gilead SciencesCompleted
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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireCompletedHemophilia AUnited States, Spain, Netherlands, Australia, Czechia, Korea, Republic of, Taiwan, Poland, Bulgaria, United Kingdom, Germany, Turkey, Israel, Malaysia, Ukraine, Russian Federation, Austria, Switzerland, Japan, Hong Kong, Lithuania, Roman... and more
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Horizon Pharma USA, Inc.CompletedCystic Fibrosis (CF)Germany, United States, Netherlands
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AbbVieVIVUS LLC; Cystic Fibrosis Foundation; Digestive Care, Inc.; Nestle Health ScienceTerminated
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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingDown Syndrome | Myeloid Leukemia Associated With Down Syndrome | B Acute Lymphoblastic Leukemia Associated With Down SyndromeUnited States
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BiomX, Inc.Active, not recruitingCystic Fibrosis | Chronic Pseudomonas Aeruginosa InfectionUnited States, Israel, Spain, Czechia, Netherlands
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Eisai Inc.TerminatedLennox-Gastaut Syndrome (LGS)Korea, Republic of, United States, Australia, Belgium, Japan, Czechia, India
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TakedaOvid Therapeutics Inc.CompletedEpilepsy | Dravet Syndrome | Lennox-Gastaut SyndromeUnited States, Canada, Israel, Poland, Spain, Australia, China, Portugal
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Novartis PharmaceuticalsActive, not recruitingChronic Myeloid Leukemia (CML) Philadelphia Chromosome PositiveUnited States, China, Hungary, Japan, Belgium, Germany, Canada, Singapore, Australia, Israel, Netherlands, Spain, Italy, Korea, Republic of, Bulgaria, France, United Kingdom, Czechia, Sweden, India, Malaysia, Denmark, Taiwan, Norway, Fi... and more
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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...