Niemann-Pick Disease (DBCOND0029170)

Identifiers

Synonyms

Sphingomyelin Lipidosis / Niemann-Pick Diseases / Niemann Pick Diseases / Niemann Pick Disease / Niemann-Pick disease / Niemann-Pick disease, unspecified / Sphingomyelin/cholesterol lipidosis (disorder) / Acid Sphingomyelinase-deficient Niemann-Pick Disease / Acid sphingomyelinase deficiency / Niemann-Pick disease type A / Niemann-Pick disease, type A (disorder) / Niemann-Pick Disease, Type A

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT00001972	PET Scan of Brain Metabolism in Relation to Age and Disease	No drug interventions	Not Available	Not Available	completed
NCT00410566	Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)	No drug interventions	treatment	1	terminated
NCT01306604	Biomarker for Niemann Pick Type C Disease (BioNPC)	No drug interventions	Not Available	Not Available	withdrawn
NCT00730314	Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells	No drug interventions	treatment	1 / 2	completed
NCT01586455	Human Placental-Derived Stem Cell Transplantation	No drug interventions	treatment	1	completed
NCT02124083	Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1	Vorinostat	treatment	1 / 2	completed
NCT01372228	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	No drug interventions	treatment	1 / 2	terminated
NCT00005900	Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation	No drug interventions	Not Available	Not Available	unknown_status
NCT02120235	Investigating Lysosomal Storage Diseases in Minority Groups	No drug interventions	Not Available	Not Available	unknown_status
NCT00176904	Stem Cell Transplant for Inborn Errors of Metabolism	Busulfan Cyclophosphamide	treatment	2 / 3	completed
NCT00316498	Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease	Miglustat	treatment	1	completed
NCT03333200	Longitudinal Study of Neurodegenerative Disorders	No drug interventions	Not Available	Not Available	recruiting
NCT04469894	Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick	No drug interventions	Not Available	Not Available	completed
NCT05641103	PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"	No drug interventions	Not Available	Not Available	recruiting
NCT06192576	A 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)	Olipudase alfa	Not Available	Not Available	recruiting
NCT03883750	Induced Pluripotent Stem Cells for Niemann Pick Disease	No drug interventions	Not Available	Not Available	completed
NCT02292654	Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency	Olipudase alfa	treatment	1 / 2	completed
NCT04877132	Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)	Olipudase alfa	Not Available	Not Available	approved_for_marketing
NCT02004691	Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	Olipudase alfa	treatment	2 / 3	completed
NCT04106544	A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)	No drug interventions	other	Not Available	completed
NCT02004704	A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	Olipudase alfa	treatment	2	completed
NCT02254863	UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells	No drug interventions	treatment	1	recruiting