A Study to Identify & Characterize LAL-D Patients in High-risk Populations

Updated 06/21/2024

Status Terminated

Clinical Trial Summary

The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.

Clinical Trial Description

n/a

Study Design

Observational Model: Case-Only

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02345421
Study type	Observational
Source	Alexion Pharmaceuticals
Contact
Status	Terminated
Phase	N/A
Start date	December 2014
Completion date	October 2015

See also
	Status	Clinical Trial	Phase
	Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
	Recruiting	`NCT03984149` - Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)
	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	`NCT02926872` - Screening for Lysosomal Acid Lipase Deficiency	N/A
	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
	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