1
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Condition(s): Acid Sphingomyelinase Deficiency; Niemann-Pick Disease Last Updated: August 3, 2009Terminated
19 Clinical Trials for: “Niemann-Pick Diseases”, Page 1 of 1
Hide Studies Not Seeking New Volunteers
2
Biomarker for Niemann Pick Type C Disease
Condition(s): Niemann-Pick Disease; Niemann-Pick Disease, Type C Last Updated: March 15, 2013Recruiting
3
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Condition(s): Niemann-Pick Disease, Type C Last Updated: April 30, 2013Completed
4
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Condition(s): 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 Last Updated: November 6, 2012Terminated
5
Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
Condition(s): Human Acid Sphingomyelinase Deficiency Last Updated: April 3, 2013Recruiting
6
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition(s): Neimann-Pick Disease Last Updated: December 19, 2012Recruiting
7
Miglustat in Niemann-Pick Type C Disease
Condition(s): Niemann-Pick Type C Disease Last Updated: March 24, 2010Completed
8
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition(s): Niemann-Pick Disease, Type C1 Last Updated: February 14, 2013Recruiting
9
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
Condition(s): Niemann Pick Diseases Last Updated: August 16, 2007Completed
10
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Condition(s): 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 Last Updated: June 23, 2005Active, not recruiting
11
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Condition(s): Sickle Cell Disease; Thalassemia; Anemia; Granuloma; Wiskott-Aldrich Syndrome; Chediak Higashi Syndrome; Osteopetrosis; Neutropenia; Thrombocytopenia; Hurler Disease; Niemann-Pick Disease; Fucosidosis Last Updated: June 22, 2011Recruiting
12
Application of Miglustat in Patients With Niemann-Pick Type C
Condition(s): Niemann-Pick Disease Type C Last Updated: January 3, 2013Completed
13
PET Scan of Brain Metabolism in Relation to Age and Disease
Condition(s): Alzheimer's Disease; Brain Neoplasm; Niemann Pick Disease Last Updated: March 3, 2008Completed
14
Stem Cell Transplant for Inborn Errors of Metabolism
Condition(s): Adrenoleukodystrophy; Metachromatic Leukodystrophy; Globoid Cell Leukodystrophy; Gaucher's Disease; Fucosidosis; Wolman Disease; Niemann-Pick Disease; Batten Disease; GM1 Gangliosidosis; Tay Sachs Disease; Sandhoff Disease Last Updated: November 6, 2012Completed
15
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Condition(s): Hurler Syndrome (MPS I); Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to ERT; Hunter Syndrome (MPS II); Sanfilippo Syndrome (MPS III); Krabbe Disease (Globoid Leukodystrophy); Metachromatic Leukodystrophy (MLD); Adrenoleukodystrophy (ALD and AMN); Sandhoff Disease; Tay Sachs Disease; Pelizaeus Merzbacher (PMD); Niemann-Pick Disease; Alpha-mannosidosis Last Updated: December 21, 2012Active, not recruiting
16
Human Placental-Derived Stem Cell Transplantation
Condition(s): Mucopolysaccharidosis I; Mucopolysaccharidosis VI; Adrenoleukodystrophy; Niemann-Pick Disease; Metachromatic Leukodystrophy; Wolman Disease; Krabbe's Disease; Gaucher's Disease; Fucosidosis; Batten Disease; Severe Aplastic Anemia; Diamond-Blackfan Anemia; Amegakaryocytic Thrombocytopenia; Myelodysplastic Syndrome; Acute Myelogenous Leukemia; Acute Lymphocytic Leukemia Last Updated: April 18, 2013Recruiting
17
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Condition(s): Inherited Metabolic Diseases; Lysosomal Storage Disease; Peroxisomal Storage Diseases; Inborn Errors of Metabolism; Mucopolysaccharidosis Last Updated: January 10, 2011Suspended
18
Trial in Children With Growth Failure Due to Early Onset Lysosomal Acid Lipase (LAL) Deficiency/Wolman Disease
Condition(s): Lysosomal Acid Lipase Deficiency; Wolman Disease Last Updated: May 6, 2013Recruiting
19
Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
Condition(s): Lysosomal Acid Lipase Deficiency; Wolman Disease Last Updated: January 18, 2013Terminated
