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Clinical trial condition

Lysosomal Acid Lipase Deficiency

Explore clinical trials studying Lysosomal Acid Lipase Deficiency. Study availability and eligibility vary by location and protocol.

10 public trials 0 recruiting studies
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Lysosomal Acid Lipase Deficiency trial results

Enrolling by Invitation

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

NCT05368038

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
Locations
  • Maimonides Medical Center Brooklyn, New York, United States
  • NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
  • North Shore University Hospital Manhasset, New York, United States
  • NYU Langone Health - Tisch Hospital New York, United States
View Trial Details
Completed

Baby Detect : Genomic Newborn Screening

NCT05687474

Conditions
3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
Locations
  • CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
View Trial Details
Completed

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

NCT01757184

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Tucson, Arizona, United States
  • Palo Alto, California, United States
  • San Francisco, California, United States
  • Wilmington, Delaware, United States
View all 40 locations
View Trial Details
Completed

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

NCT02112994

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Chicago, Illinois, United States
  • Shreveport, Louisiana, United States
  • Cincinnati, Ohio, United States
  • Westmead, Australia
View all 19 locations
View Trial Details
Completed

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

NCT01307098

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease(CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Stanford, California, United States
  • New York, United States
  • Pittsburgh, Pennsylvania, United States
  • Prague, Czechia
View Trial Details
Completed

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

NCT01488097

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease (CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Eureka, California, United States
  • Sacramento, California, United States
  • San Francisco, California, United States
  • Minneapolis, Minnesota, United States
View Trial Details
Completed

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

NCT01528917

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease(CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • Stanford University Stanford, California, United States
  • Children's Memorial Hospital Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Morgan Stanley Children's Hospital of New York-Presbyterian New York, United States
View all 16 locations
View Trial Details
Completed

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

NCT01358370

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Cedars-Siani Medical Center Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Columbia University New York, United States
View all 14 locations
View Trial Details
Active, Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
View Trial Details

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