Large-scale testing (Phase 3)Study completedNCT02534844What this trial is testingVTS-270 to Treat Niemann-Pick Type C1 (NPC1) DiseaseWho this might be right forNiemann-Pick Disease, Type C Mandos LLC 56
Not applicableActive Not RecruitingNCT07011329What this trial is testingBrain Alterations Based on Multiplex MR Imaging in Nasopharyngeal Carcinoma Patients After Treatment, and Their Correlation With Clinical Cognitive Scores.Who this might be right forNasopharyngeal Carcinoma (NPC) Hubei Cancer Hospital 60
Not applicableStudy completedNCT03883750What this trial is testingInduced Pluripotent Stem Cells for Niemann Pick DiseaseWho this might be right forNiemann-Pick Diseases CENTOGENE GmbH Rostock 40
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 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 earlyNCT01372228What this trial is testingPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersWho this might be right forHurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more Talaris Therapeutics Inc. 3
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
Large-scale testing (Phase 3)Ended earlyNCT03879655What this trial is testingOpen-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1Who this might be right forNiemann-Pick Disease, Type C Mandos LLC 2
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
Testing effectiveness (Phase 2)Study completedNCT03471143What this trial is testingStudy of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CWho this might be right forNiemann-Pick Disease, Type C Washington University School of Medicine 4
Not applicableWithdrawnNCT04189601What this trial is testingComplement Activation in the Lysosomal Storage DisordersWho this might be right forFabry DiseaseGaucher DiseaseNiemann-Pick Disease, Type C+1 more Melbourne Health
Early research (Phase 1)Study completedNCT01747135What this trial is testingHydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 DiseaseWho this might be right forNiemann-Pick Disease, Type C1 Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company 14
Testing effectiveness (Phase 2)Study completedNCT03759639What this trial is testingN-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)Who this might be right forNiemann-Pick Disease, Type C IntraBio Inc 33
Large-scale testing (Phase 3)Study completedNCT01760564What this trial is testingApplication of Miglustat in Patients With Niemann-Pick Type CWho this might be right forNiemann-Pick Disease Type C National Taiwan University Hospital 5
Not applicableLooking for participantsNCT00344331What this trial is testingEvaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type CWho this might be right forNiemann-Pick Disease, Type C Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 900
Not applicableLooking for participantsNCT07545473What this trial is testingRetinal Hyperspectral Imaging in Neurodegenerative DiseasesWho this might be right forDementiaNeurodegenerative DiseasesAlzheimer Disease+5 more Center for Eye Research Australia 930
Testing effectiveness (Phase 2)Study completedNCT00517153What this trial is testingMiglustat in Niemann-Pick Type C DiseaseWho this might be right forNiemann-Pick Type C Disease Actelion 29
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104What this trial is testingMT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisWho this might be right forMucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more Masonic Cancer Center, University of Minnesota 149
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
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40