Screening for Lysosomal Acid Lipase Deficiency

Updated 06/21/2024

Status Terminated

Clinical Trial Summary

The primary outcome of this study is the development of a clinical profile of pediatric patients with LAL-D, which will enable the Sponsor to provide more focused guidance to the medical community as to which pediatric patients should be tested for LAL-D.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02926872
Study type	Observational
Source	Alexion Pharmaceuticals
Contact
Status	Terminated
Phase	N/A
Start date	November 2016
Completion date	June 5, 2017

See also
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	Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
	Recruiting	`NCT03564002` - Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity
	Terminated	`NCT01473875` - Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102	Phase 2/Phase 3
	Completed	`NCT01358370` - A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype	N/A
	Completed	`NCT01371825` - Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency	Phase 2/Phase 3
	Enrolling by invitation	`NCT05368038` - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
	Terminated	`NCT02193867` - Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency	Phase 2
	Completed	`NCT02112994` - Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency	Phase 2
	Completed	`NCT01528917` - An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype	N/A
	Completed	`NCT01757184` - Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency	Phase 3
	Recruiting	`NCT01633489` - Lysosomal Acid Lipase (LAL) Deficiency Registry
	Terminated	`NCT02345421` - A Study to Identify and Characterize LAL-D Patients in High-risk Populations	N/A
	Completed	`NCT01488097` - Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency	Phase 2
	Enrolling by invitation	`NCT01716728` - Identification of Undiagnosed Lysosomal Acid Lipase Deficiency	N/A
	Completed	`NCT01307098` - Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency	Phase 1/Phase 2
	No longer available	`NCT02376751` - An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency	N/A