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35 studies found for:    "niemann-pick disease"
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Rank Status Study
1 Completed Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Condition: Niemann-Pick Disease, Type C
Intervention:
2 Completed
Has Results
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Condition: Niemann-Pick Disease, Type C
Intervention: Drug: N-Acetyl Cysteine
3 Recruiting A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Condition: Niemann-Pick Disease, Type C
Intervention:
4 Terminated Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Conditions: Acid Sphingomyelinase Deficiency;   Niemann-Pick Disease
Intervention: Drug: rhASM
5 Recruiting Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Condition: Neimann-Pick Disease
Intervention: Drug: Vorinostat
6 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
7 Completed Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
Condition: Human Acid Sphingomyelinase Deficiency
Intervention: Drug: Recombinant human acid sphingomyelinase
8 Completed Miglustat in Niemann-Pick Type C Disease
Condition: Niemann-Pick Type C Disease
Intervention: Drug: miglustat
9 Recruiting Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition: Neimann-Pick Disease, Type C
Intervention:
10 Recruiting A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: GZ402665
11 Completed Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
Condition: Niemann Pick Diseases
Intervention: Drug: OGT918
12 Recruiting Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Interventions: Drug: placebo (saline);   Drug: GZ402665
13 Recruiting Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: Olipudase alfa
14 Not yet recruiting Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: Hydroxypropyl-beta-cyclodextrin
15 Not yet recruiting Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: Hydroxypropyl-beta-cyclodextrin
16 Completed Application of Miglustat in Patients With Niemann-Pick Type C
Condition: Niemann-Pick Disease Type C
Intervention: Drug: Miglustat
17 Active, not recruiting Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: VTS-270
18 Recruiting Screening of Niemann-Pick Disease, Type C in a Psychiatric Population
Condition: Psychiatric Adults Patients
Interventions: Biological: Blood sampling;   Biological: Biopsy
19 Recruiting Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: arimoclomol;   Drug: Placebo
20 Recruiting Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: VTS-270;   Drug: Sham Procedure Control

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Study has passed its completion date and status has not been verified in more than two years.