Not applicableUnknownNCT05904366What this trial is testingMagnetic Resonance Spectroscopy in Acid Sphingomyelinase DeficiencyWho this might be right forAsmd, Visceral Type Eline C. B. Eskes 34
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 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 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
Testing effectiveness (Phase 2)Study completedNCT02292654What this trial is testingSafety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 20
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
Not applicableEnrolling By InvitationNCT03655223What 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 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
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
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 applicableNot Yet RecruitingNCT06258577What this trial is testingScreening for Gaucher Disease and Acid Sphingomyelinase DeficiencyWho this might be right forGaucher Disease Chung-Hsing Wang 50
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
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
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 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
Not applicableActive Not RecruitingNCT05641103What 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
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 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
Testing effectiveness (Phase 2)Study completedNCT06949358What this trial is testingEvaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in FranceWho this might be right forNiemann-Pick Disease Sanofi 3
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