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Results for “Cholesterol Ester Storage Disease (CESD)”

37 trials

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Showing 20 of 37 results

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 applicableUnknownNCT02851550
What this trial is testing

Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients

Who this might be right for
Liver Post-transplant Patients
Hospices Civils de Lyon 100
Not applicableNo Longer AvailableNCT02376751
What this trial is testing

An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc.
Testing effectiveness (Phase 2)Study completedNCT01307098
What this trial is testing

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Alexion Pharmaceuticals, Inc. 9
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 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
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 applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT01488097
What this trial is testing

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

Who this might be right for
Cholesterol Ester Storage Disease (CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Alexion Pharmaceuticals, Inc. 8
Not applicableUnknownNCT06287658
What this trial is testing

The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence

Who this might be right for
Urinary IncontinenceMenopauseWolman Disease+2 more
Ondokuz Mayıs University 60
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
Not applicableWithdrawnNCT02383641
What this trial is testing

Biomarker for Wolman Disease (BioWolman)

Who this might be right for
Acid Lipase DeficiencyAcid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeCholesterol Ester Storage Disease
CENTOGENE GmbH Rostock
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
Testing effectiveness (Phase 2)Ended earlyNCT02193867
What this trial is testing

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 10
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 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
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
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
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 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
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