Clinical Trials on Hypercholesterolemia, Familial, 4, Autosomal Recessive in United States
Total 131 results
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University of Texas Southwestern Medical CenterWithdrawnFamilial HypercholesterolemiaUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedParkinson Disease 6, Early-Onset | Parkinson Disease (Autosomal Recessive, Early Onset) 7, Human | Parkinson Disease Autosomal Recessive, Early Onset | Parkinson Disease, Autosomal Recessive Early-Onset, Digenic, Pink1/Dj1United States
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedMetabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditionsUnited States
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedMetabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditionsUnited States, Taiwan, Brazil, Canada, South Africa, United Kingdom, Singapore
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AstraZenecaCompletedFamilial HypercholesterolaemiaUnited States, Netherlands, Norway, Canada, Belgium
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedMetabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditionsUnited States, Canada, Brazil, Taiwan, Singapore, South Africa, United Kingdom
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Wayne State UniversityUnknownAneuploidy | Autosomal Recessive DisorderUnited States
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney (ARPKD)United States, United Kingdom, Belgium, Poland, France, Germany, Italy
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National Heart, Lung, and Blood Institute (NHLBI)CompletedHomozygous Familial HypercholesterolemicUnited States
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NeuroBo Pharmaceuticals Inc.CompletedHypercholesteremiaUnited States, Canada, Israel
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Kadmon Corporation, LLCCompletedPolycystic Kidney, Autosomal RecessiveUnited States
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Novartis PharmaceuticalsActive, not recruitingFamilial Hypercholesterolemia - HomozygousGreece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
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SanofiRegeneron PharmaceuticalsCompletedHypercholesterolaemiaNorway, Russian Federation, Sweden, South Africa, Czechia, United States, Canada, France, Netherlands, Spain
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SanofiRegeneron PharmaceuticalsCompletedHypercholesterolaemiaNetherlands, United States, Canada, Russian Federation, South Africa
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Geisinger ClinicCompletedHypercholesterolemia | Hypercholesterolemia, Familial | Hypercholesteremia in Children | Hyperlipidemia in ChildrenUnited States
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney Disease (ARPKD)United States, Belgium, Spain, Germany, United Kingdom, Poland
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Novartis PharmaceuticalsRecruitingHeterozygous or Homozygous Familial HypercholesterolemiaNetherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
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SanofiRegeneron PharmaceuticalsCompletedHypercholesterolaemiaCzechia, France, Denmark, Netherlands, Austria, Mexico, Spain, United States, Argentina, Brazil, Bulgaria, Canada, Finland, Hungary, Italy, Lebanon, Norway, Poland, Russian Federation, Slovenia, South Africa, Sweden, Taiwan, Turkey
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Aegerion Pharmaceuticals, Inc.University of Pennsylvania; Doris Duke Charitable FoundationCompletedHomozygous Familial HypercholesterolemiaUnited States
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REGENXBIO Inc.National Heart, Lung, and Blood Institute (NHLBI)TerminatedHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Italy, Netherlands
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HDL TherapeuticsMedStar Heart and Vascular InstituteUnknownHomozygous Familial Hypercholesterolemia | HoFHUnited States
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Regeneron PharmaceuticalsSanofiTerminatedHeterozygous Familial Hypercholesterolemia | Non-familial HypercholesterolemiaUnited States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine
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Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemiaFrance, United States
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The Rogosin InstituteRecruitingHomozygous Familial HypercholesterolemiaUnited States
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The Medicines CompanyCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Novartis PharmaceuticalsCompletedElevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVDUnited States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Canada
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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University of PennsylvaniaUniversity of Amsterdam; University of Cape Town; University of Witwatersrand...RecruitingHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Arrowhead PharmaceuticalsActive, not recruitingHomozygous Familial HypercholesterolemiaAustralia, Canada, United States, South Africa
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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LIB Therapeutics LLCMedpace, Inc.CompletedHeterozygous Familial HypercholesterolemiaUnited States, Israel, Norway, South Africa, Turkey
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REGENXBIO Inc.CompletedHomozygous Familial Hypercholesterolemia (HoFH)United States
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Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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Regeneron PharmaceuticalsSanofiCompletedHeterozygous Familial HypercholesterolemiaUnited States, Germany
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingBethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1United Kingdom
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Australia, Austria, Netherlands, Taiwan, Ukraine
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Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemia | Heterozygous Familial HypercholesterolemiaUnited States, Canada
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LIB Therapeutics LLCCompletedHomozygous Familial HypercholesterolemiaUnited States, Israel, Norway, South Africa, Turkey, India
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Regeneron PharmaceuticalsSanofiCompletedHomozygous Familial HypercholesterolemiaUnited States, South Africa, Canada, Greece, Japan, Ukraine, Austria, Czechia, France, Germany, Italy, Taiwan, Turkey
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Australia, Austria, Canada, France, Greece, Italy, Japan, South Africa, Ukraine
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Novartis PharmaceuticalsActive, not recruitingFamilial Hypercholesterolemia - HeterozygousUnited States, Germany, South Africa, Greece, Italy, Czechia, Taiwan, Slovakia, Turkey, France, Russian Federation, Canada, Spain, Jordan, Switzerland, Hungary, Poland, Netherlands, Norway, United Kingdom, Lebanon, Argentina, Brazil, ... and more
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REGENXBIO Inc.Active, not recruitingHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Netherlands
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency (MoCD) | Rare Autosomal Recessive Disorder | Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)United States
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AmgenCompletedHomozygous Familial HypercholesterolemiaNew Zealand, South Africa, France, United States, Spain, Belgium, Czechia, Italy, Netherlands, Hong Kong, Canada, Lebanon
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NYU Langone HealthCompletedAnxiety Disorders | Familial Dysautonomia | Dysthymia | Paroxysmal Hypertension | Autosomal Recessive DiseaseUnited States
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AmgenCompletedFamilial HypercholesterolemiaCanada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
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AmgenCompletedSevere Familial HypercholesterolemiaItaly, Czechia, United States, Israel, New Zealand, South Africa, France, Spain, Australia, Belgium, Canada, Netherlands, Hong Kong, Brazil, United Kingdom, Greece, Japan, Lebanon
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AmgenCompletedHeterozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Norway, Belgium, Turkey, Australia, New Zealand, Austria, Czechia, South Africa, Poland, Spain, Switzerland, Netherlands, Taiwan, Finland, Brazil, Portugal, Slovenia, Russian Federation, Hungary, United... and more