Example: Heart Attack

Clinical Trials on Niemann-Pick Diseases

Total 48 results

NCT03883750

Completed

Induced Pluripotent Stem Cells for Niemann Pick Disease

Conditions: Niemann-Pick Diseases
NCT01899950

Completed

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

Conditions: Niemann-Pick Disease, Type C
NCT02612129

Active, not recruiting

Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C

Conditions: Niemann-Pick Disease, Type C
NCT03893071

Recruiting

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Conditions: Niemann-Pick Disease, Type C1
NCT03471143

Recruiting

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Conditions: Niemann-Pick Disease, Type C
NCT03887533

Recruiting

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Conditions: Niemann-Pick Disease, Type C1
NCT02435030

Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Conditions: Niemann-Pick Disease, Type C
NCT00975689

Completed

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Conditions: Niemann-Pick Disease, Type C
NCT03759639

Active, not recruiting

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Conditions: Niemann-Pick Disease, Type C
NCT02124083

Completed

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

Conditions: Neimann-Pick Disease
NCT00410566

Terminated

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Conditions: Acid Sphingomyelinase Deficiency; Niemann-Pick Disease
NCT01306604

Active, not recruiting

Biomarker for Niemann Pick Type C Disease (BioNPC)

Conditions: Niemann-Pick Disease; Niemann-Pick Disease, Type C
NCT03643562

Active, not recruiting

Adrabetadex for Patients With Nerve Symptoms of Niemann-Pick Type C Disease (NPC)

Conditions: Niemann-Pick Type C Disease
NCT04469894

Completed

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

Conditions: Niemann-Pick Diseases
NCT01722526

Completed

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

Conditions: Human Acid Sphingomyelinase Deficiency
NCT00517153

Completed

Miglustat in Niemann-Pick Type C Disease

Conditions: Niemann-Pick Type C Disease
NCT00316498

Completed

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

Conditions: Niemann Pick Diseases
NCT04106544

Recruiting

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Conditions: Sphingomyelin Lipidosis
NCT04316637

Available

Early Access Program With Arimoclomol in US Patients With NPC

Conditions: Niemann-Pick Disease, Type C
NCT00344331

Recruiting

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Conditions: Neimann-Pick Disease, Type C
NCT03910621

Recruiting

Safety and Efficacy of Miglustat in Chinese NPC Patients

Conditions: Niemann-Pick Disease, Type C
NCT02004704

Active, not recruiting

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Conditions: Sphingomyelin Lipidosis
NCT02292654

Completed

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Conditions: Sphingomyelin Lipidosis
NCT02004691

Active, not recruiting

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Conditions: Sphingomyelin Lipidosis
NCT02939547

Completed

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Conditions: Niemann-Pick Disease, Type C1