Not applicableActive Not RecruitingNCT07274826What this trial is testingDiagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)Who this might be right forAcid Sphingomyelinase Deficiency (ASMD) Sohag University 7
Testing effectiveness (Phase 2)Study completedNCT02004704What this trial is testingA Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 25
Large-scale testing (Phase 3)Study completedNCT02004691What this trial is testingEfficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 36
Early research (Phase 1)Study completedNCT01722526What this trial is testingTolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency PatientsWho this might be right forHuman Acid Sphingomyelinase Deficiency Genzyme, a Sanofi Company 5
Not applicableLooking for participantsNCT06192576What this trial is testingA Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)Who this might be right forNiemann-Pick DiseasesAcid Sphingomyelinase Deficiency Sanofi 10
Not applicableLooking for participantsNCT05992532What this trial is testingGammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple MyelomaWho this might be right forGaucher DiseaseAcid SphingoMyelinase Deficiency Fundación Española de Hematología y Hemoterapía 210
Not applicableUnknownNCT05904366What this trial is testingMagnetic Resonance Spectroscopy in Acid Sphingomyelinase DeficiencyWho this might be right forAsmd, Visceral Type Eline C. B. Eskes 34
Not applicableEnrolling By InvitationNCT05368038What this trial is testingScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramWho this might be right forAcid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Ended earlyNCT02193867What this trial is testingClinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 10
Not applicableStudy completedNCT04106544What this trial is testingA Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)Who this might be right forSphingomyelin Lipidosis Sanofi 84
Not applicableNot Yet RecruitingNCT06985212What this trial is testingNational Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and ChildrenWho this might be right forAcid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease Wladimir MAUHIN, Dr 200
Not applicableStudy completedNCT05641103What this trial is testingPREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"Who this might be right forGaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more Fundación Española de Hematología y Hemoterapía 122
Not applicableStudy completedNCT04845958What this trial is testingA Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged SpleenWho this might be right forGaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency Sanofi 60
Not applicableStudy completedNCT05359276What this trial is testingData Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in FranceWho this might be right forAcid Sphingomyelinase Deficiency (ASMD) Sanofi 40
Early research (Phase 1)Ended earlyNCT00410566What this trial is testingSafety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)Who this might be right forAcid Sphingomyelinase DeficiencyNiemann-Pick Disease Genzyme, a Sanofi Company 11
Not applicableApproved For MarketingNCT04877132What this trial is testingCompassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)Who this might be right forSphingomyelin Lipidosis Sanofi
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableNot Yet RecruitingNCT06869499What this trial is testingStudy of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung DiseaseWho this might be right forSplenomegalySplenectomyThrombopenia+2 more Wladimir MAUHIN, Dr 200
Large-scale testing (Phase 3)Study completedNCT01371825What this trial is testingSafety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase DeficiencyWolman Disease Alexion Pharmaceuticals, Inc. 9
Not applicableStudy completedNCT00001972What this trial is testingPET Scan of Brain Metabolism in Relation to Age and DiseaseWho this might be right forAlzheimer's DiseaseBrain NeoplasmNiemann Pick Disease National Institute of Neurological Disorders and Stroke (NINDS) 123