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30 studies found for:    "Niemann-Pick disease type C1"
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Rank Status Study
1 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
2 Completed Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Condition: Niemann-Pick Disease, Type C
Intervention:
3 Active, not recruiting Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: VTS-270
4 Recruiting Screening of Niemann-Pick Disease, Type C in a Psychiatric Population
Condition: Psychiatric Adults Patients
Interventions: Biological: Blood sampling;   Biological: Biopsy
5 Recruiting Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Condition: Neimann-Pick Disease
Intervention: Drug: Vorinostat
6 Recruiting Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: arimoclomol;   Drug: Placebo
7 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
8 Recruiting A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Condition: Niemann-Pick Disease, Type C
Intervention:
9 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
10 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
11 Recruiting Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition: Neimann-Pick Disease, Type C
Intervention:
12 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
13 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
14 Completed Miglustat in Niemann-Pick Type C Disease
Condition: Niemann-Pick Type C Disease
Intervention: Drug: miglustat
15 Terminated
Has Results
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Conditions: Hurler's Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Sphingolipidoses;   Krabbe Disease;   Wolman's Disease;   Niemann-Pick Disease Type B;   Niemann-Pick Disease, Type C
Interventions: Procedure: Stem Cell Transplantation;   Drug: Cyclophosphamide;   Drug: Campath-1H;   Drug: Busulfan
16 Recruiting Genetic Characterization of Movement Disorders and Dementias
Conditions: Ataxia;   Dystonia;   Parkinson's Disease;   Amyotrophic Lateral Sclerosis;   Corticobasal Degeneration;   Multiple System Atrophy;   Alzheimer's Disease;   Lewy Body Dementia;   Parkinson Disease-Dementia;   Dentatorubral-pallidoluysian Atrophy;   Creutzfeldt-Jakob Disease and Fatal Familial Insomnia;   Fragile X-associated Tremor/Ataxia Syndrome;   Krabbe's Disease;   Niemann-Pick Disease, Type C;   Neuronal Ceroid Lipofuscinosis
Intervention:
17 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
18 Recruiting A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: GZ402665
19 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
20 Completed Application of Miglustat in Patients With Niemann-Pick Type C
Condition: Niemann-Pick Disease Type C
Intervention: Drug: Miglustat

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