Not applicableAvailableNCT04316637What this trial is testingEarly Access Program With Arimoclomol in US Patients With NPCWho this might be right forNiemann-Pick Disease, Type C ZevraDenmark
Testing effectiveness (Phase 2)Study completedNCT00975689What this trial is testingBiomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl CysteineWho this might be right forNiemann-Pick Disease, Type C Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Large-scale testing (Phase 3)Study completedNCT02612129What this trial is testingArimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type CWho this might be right forNiemann-Pick Disease, Type C ZevraDenmark 50
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
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 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 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 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 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 completedNCT02124083What this trial is testingPhase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1Who this might be right forNeimann-Pick Disease Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 12
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
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
Not applicableUnknownNCT02120235What this trial is testingInvestigating Lysosomal Storage Diseases in Minority GroupsWho this might be right forLysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more O & O Alpan LLC 20,000
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Large-scale testing (Phase 3)Looking for participantsNCT07054515What this trial is testingEvaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 GangliosidosisWho this might be right forNiemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis Azafaros A.G. 147
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
Early research (Phase 1)UnknownNCT03893071What this trial is testingOpen-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type CWho this might be right forNiemann-Pick Disease, Type C1 Cyclo Therapeutics, Inc. 12
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
Large-scale testing (Phase 3)Active Not RecruitingNCT04860960What this trial is testingPhase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1Who this might be right forNiemann-Pick Disease, Type C1 Cyclo Therapeutics, Inc. 94
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