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Results for “Cholesterol Ester Storage Disease(CESD)”

36 trials

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Showing 20 of 36 results

Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
Not applicableUnknownNCT03984149
What this trial is testing

Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

Who this might be right for
Lysosomal Acid Lipase Deficiency
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) 1,000
Testing effectiveness (Phase 2)Study completedNCT02112994
What this trial is testing

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 31
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableUnknownNCT01716728
What this trial is testing

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage Disease
Massachusetts General Hospital 60
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Large-scale testing (Phase 3)Study completedNCT01757184
What this trial is testing

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 66
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableEnded earlyNCT02345421
What this trial is testing

Identify and Characterize LAL-D Patients in High-risk Populations

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 640
Not applicableStudy completedNCT01358370
What this trial is testing

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 40
Not applicableUnknownNCT01791452
What this trial is testing

Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study

Who this might be right for
Non-alcoholic Fatty Liver DiseaseCholesterol Ester Storage Disease
Assy Nimer
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Not applicableUnknownNCT03564002
What this trial is testing

Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity

Who this might be right for
Lysosomal Acid Lipase DeficiencyObesityKetogenic Dieting+1 more
University Of Perugia 60
Not applicableStudy completedNCT01528917
What this trial is testing

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Who this might be right for
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 49
Not applicableUnknownNCT04792671
What this trial is testing

Prevalence and Risk Factors of Women Mental Health Disorders

Who this might be right for
Psychiatric ProblemWolman Disease
Assiut University 200
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableStudy completedNCT04652713
What this trial is testing

Breakfast for Young Women

Who this might be right for
ObesityWolman DiseaseNutrition
University of Aarhus 30
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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