Cholesterol Ester Storage Disease - 25 Studies Found
No longer available |
: An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency : Lysosomal Acid Lipase Deficiency : 2015-02-25 : Drug: sebelipase alfa Other Name: SBC-102, recombinant human lysosomal acid lipase, rhLAL |
Active, not recruiting |
: Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency : Lysosomal Acid Lipase Deficiency : 2014-03-20 : Drug: sebelipase alfa Intravenous infusion of sebelipase alfa: 1mg/kg every other week |
Active, not recruiting |
: Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency : Lysosomal Acid Lipase Deficiency : 2014-07-07 : Drug: sebelipase alfa Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). The investig |
Terminated |
: Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 :
: 2011-11-10 : Drug: SBC-102 SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicina |
Active, not recruiting |
: SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa :
: 2011-06-09 : Drug: Sebelipase alfa (SBC-102) Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Th |
Active, not recruiting |
: Screening for Lysosomal Acid Lipase Deficiency : Lysosomal Acid Lipase Deficiency : 2016-10-04 : Other: There is no intervention in the study |
Recruiting |
: Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant. : Patients Waiting for a Liver Transplant. : 2016-07-28 |
Recruiting |
: Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients : Liver Post-transplant Patients : 2016-07-28 |
Terminated |
: A Study to Identify and Characterize LAL-D Patients in High-risk Populations : Lysosomal Acid Lipase Deficiency : 2015-01-19 |
Completed |
: A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype :
: 2011-04-13 |