Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
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34 studies found for:    Niemann-Pick Disease

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Rank Status Study
1 Recruiting Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Condition: Neimann-Pick Disease
Intervention: Drug: Vorinostat
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 Completed Longitudinal Study of Cognition 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 Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
6 Recruiting Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: 2-hydroxypropyl-beta-cyclodextrin
7 Recruiting Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition: Neimann-Pick Disease, Type C
Intervention:
8 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
9 Completed Miglustat in Niemann-Pick Type C Disease
Condition: Niemann-Pick Type C Disease
Intervention: Drug: miglustat
10 Completed Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
Condition: Niemann Pick Diseases
Intervention: Drug: OGT918
11 Recruiting A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: GZ402665
12 Not yet 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 Dose 1;   Drug: GZ402665 Dose 2
13 Recruiting Investigating Lysosomal Storage Diseases in Minority Groups
Conditions: Lysosomal Storage Disorders;   Gaucher Disease;   Fabry Disease;   Pompe Disease;   Niemann-Pick Disease
Intervention:
14 Unknown † Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Conditions: Sickle Cell Disease;   Thalassemia;   Anemia;   Granuloma;   Wiskott-Aldrich Syndrome;   Chediak Higashi Syndrome;   Osteopetrosis;   Neutropenia;   Thrombocytopenia;   Hurler Disease;   Niemann-Pick Disease;   Fucosidosis
Intervention: Procedure: Hematopoietic stem cell transplantation
15 Unknown † Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
16 Completed Application of Miglustat in Patients With Niemann-Pick Type C
Condition: Niemann-Pick Disease Type C
Intervention: Drug: Miglustat
17 Not yet recruiting Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: GZ402665
18 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
19 Completed
Has Results 		Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin
20 Completed PET Scan of Brain Metabolism in Relation to Age and Disease
Conditions: Alzheimer's Disease;   Brain Neoplasm;   Niemann Pick Disease
Intervention: Drug: 15 O Water
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† Indicates status has not been verified in more than two years