Clinical Trials on Lipid Metabolism, Inborn Errors
Total 7135 results
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Rambam Health Care CampusUnknown
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National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
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Massachusetts General HospitalActive, not recruiting
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SanofiCompleted
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AVROBIOWithdrawn
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, United States
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SanofiCompletedGaucher DiseaseUnited States
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
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National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed
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Eunice Kennedy Shriver National Institute of Child...RecruitingBatten Disease | Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)United States
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NewAmsterdam PharmaActive, not recruitingDyslipidemias | Hypercholesterolemia | Familial Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | High CholesterolUnited States, Poland, Denmark, Czechia, China, Netherlands, Japan, Georgia
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LIB Therapeutics LLCMedpace, Inc.Enrolling by invitationFamilial Hypercholesterolemia | Elevated Cholesterol | Cardiovascular Disease With Mention of ArteriosclerosisUnited States, Israel, Norway, South Africa, Turkey, India
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Amicus TherapeuticsTerminatedBatten Disease | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3United States
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Amicus TherapeuticsCompletedVariant Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1United States, Canada, Germany, Israel, Italy, South Africa, Spain, United Kingdom, Brazil, France, Netherlands
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1Canada
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Novartis PharmaceuticalsTerminatedFamilial Chylomicronemia Syndrome (FCS) (HLP Type I)South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
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Hospices Civils de LyonUnknownPatients Waiting for a Liver Transplant.France
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Nationwide Children's HospitalMassachusetts General HospitalCompletedNeuronal Ceroid Lipofuscinosis | Batten's DiseaseUnited States
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Isfahan University of Medical SciencesCompletedTangier Disease | Body Mass Index Quantitative Trait Locus 7 DisorderIran, Islamic Republic of
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
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University of PennsylvaniaSuspendedLipid Metabolism, Inborn Errors | Cholesterol, HDL | Tangier Disease | LCAT Deficiency | Cholesteryl Ester Transfer Protein (CETP) DeficiencyUnited States
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The Medicines CompanyCompletedDiabetes | Familial Hypercholesterolemia | Atherosclerotic Cardiovascular DiseaseCanada, Netherlands, United States, United Kingdom, Germany
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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Instituto Aragones de Ciencias de la SaludHospital Clinic of Barcelona; Instituto de Salud Carlos IIICompletedObesity | Weight Loss | Familial Hypercholesterolemias | Familial Combined Hypercholesterolemia
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Maastricht University Medical CenterCompletedNeutral Lipid Storage DiseaseNetherlands
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Verve Therapeutics, Inc.Not yet recruitingHypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial HypercholesterolemiaNew Zealand, United Kingdom
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Verve Therapeutics, Inc.Active, not recruitingHypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial HypercholesterolemiaNew Zealand, United Kingdom
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BioMarin PharmaceuticalActive, not recruitingLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2United States
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REGENXBIO Inc.WithdrawnNeuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedInfantile Neronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Isfahan University of Medical SciencesCompletedTangier Disease | Body Mass Index Quantitative Trait Locus 5 DisorderIran, Islamic Republic of
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Translational Research Center for Medical Innovation...RecruitingNeutral Lipid Storage DiseaseChina, United States, France, Austria, Germany, Italy, Japan, Netherlands, United Kingdom
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Regeneron PharmaceuticalsAvailablePartial Lipodystrophy | Generalized Lipodystrophy | Monogenic Obesity Due to LEP or LEPR Loss of Function
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University of British ColumbiaMedical University of Graz; Hospital de Clinicas de Porto AlegreUnknownMorquio B Disease | GM1 Gangliosidosis Type IIICanada
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Kinderkrankenhaus auf der BultCompletedType 1 Diabetes | Familial Hypercholesterolemia
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Bellicum PharmaceuticalsNo longer availableHurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage DisorderUnited States
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States