Clinical Trials on Lysosomal Acid Lipase Deficiency
Total 342 results
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationSevere Combined Immune Deficiency (SCID)United States
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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Eunice Kennedy Shriver National Institute of Child...Children's National Research InstituteTerminatedPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency Disease
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S.LAB (SOLOWAYS)Center of New Medical TechnologiesRecruiting
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Huilian ZhuNot yet recruiting
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Peking UniversityNational Natural Science Foundation of China; Institute of reproductive and...CompletedHyperhomocysteinemiaChina
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Baylor Research InstituteNational Institutes of Health (NIH)CompletedHyperhomocysteinemiaUnited States
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Center for Health, Exercise and Sport Sciences,...Completed
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University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
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University of Novi Sad, Faculty of Sport and Physical...Completed
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Queen Sirikit National Institute of Child HealthChulalongkorn UniversityCompletedObesity | HyperhomocysteinemiaThailand
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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London School of Hygiene and Tropical MedicineCompletedHyperhomocysteinemia | Micronutrient DeficienciesGambia
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National Institute of Cardiology, Laranjeiras,...CompletedEndothelial Dysfunction | HyperhomocysteinemiaBrazil
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UniQure Biopharma B.V.CompletedLipoprotein Lipase Deficiency | Familial Hyperlipoproteinemia Type 1 | Familial HyperchylomicronemiaGermany
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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National Center for Research Resources (NCRR)UNC Lineberger Comprehensive Cancer CenterCompletedMetabolism, Inborn Errors | Methionine Adenosyltransferase Deficiency
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IRCCS Azienda Ospedaliero-Universitaria di BolognaUniversity of BolognaCompletedInflammation | Mortality | HyperhomocysteinemiaItaly
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde Dehydrogenase Deficiency | SSADH DeficiencyUnited States
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Beijing Chest HospitalNot yet recruitingMalnutrition | Lung Cancer | Amino Acid Deficiency
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Glasgow Royal InfirmaryCompletedMagnesium Deficiency | Alcohol Withdrawal | Lactic Acidosis | Vitamin B1 Deficiency | Wernicke EncephalopathyUnited Kingdom
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Chiesi Farmaceutici S.p.A.Completed
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Zymenex A/SCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed
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Zymenex A/SEuropean CommissionCompletedAlpha-MannosidosisBelgium, Denmark, Spain, United Kingdom
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Leadiant Biosciences, Inc.CompletedSevere Combined Immunodeficiency | ADA-SCID | Adenosine Deaminase DeficiencyUnited States
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Chiesi Farmaceutici S.p.A.Not yet recruiting
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Chiesi Farmaceutici S.p.A.CromsourceCompletedAlpha-MannosidosisFrance, Denmark, Austria, Germany, Italy
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Chiesi Farmaceutici S.p.A.Withdrawn
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University Hospital HeidelbergGerman Cancer Research CenterCompletedType2 Diabetes | Fatty Acid Deficiency | Alteration of Mitochondrial MembraneGermany
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Chiesi Farmaceutici S.p.A.CompletedAlpha-MannosidosisFrance
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Zymenex A/SEuropean CommissionCompletedA Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-MannosidosisAlpha-MannosidosisDenmark, United Kingdom, France, Germany, Poland
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University of California, Los AngelesGreat Ormond Street Hospital for Children NHS Foundation TrustEnrolling by invitationAdenosine Deaminase Deficiency | Severe Combined Immunodeficiency (SCID)United States, United Kingdom
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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited 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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CENTOGENE GmbH RostockWithdrawnMetabolic Disorders | Tyrosinosis | Hepatorenal Tyrosinemia | Fumarylacetoacetase Deficiency | Fah DeficiencyGermany, Sri Lanka, India
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Great Ormond Street Hospital for Children NHS Foundation...Orchard TherapeuticsCompletedAdenosine Deaminase Deficiency | Severe Combined Immunodeficiencies (SCID)United Kingdom
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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S.LAB (SOLOWAYS)Center of New Medical Technologies; Triangel ScientificCompletedHyperlipoproteinemias | HyperhomocysteinemiaRussian Federation
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Aeglea BiotherapeuticsTerminatedArginase I Deficiency | HyperargininemiaCanada
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde | Dehydrogenase Deficiency | DiseasesUnited States
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Fresenius KabiWithdrawnMalnutrition | Pediatric ALL | Neurocognitive Deficit | Parenteral Nutrition Associated Liver Disease | Essential Fatty Acid Deficiency