Lysosomal Acid Lipase (LAL) Deficiency Registry

NCT01633489 · tracked via the Priya Life Science USA tracker

Sponsor

Alexion Pharmaceuticals, Inc.

Phase

Observational

Started

2013-05-30

Last updated

2026-03-06

Condition(s) studied

Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2Acid Lipase DeficiencyLIPA DeficiencyLAL-Deficiency

Study summary

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee. Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Primary outcome measure(s)

Understanding of the variability, progression, identification and natural history of LAL Deficiency. — Ongoing

Trial sites (104)

Facility	City	Region	Status
Clinical Trial Site	Phoenix	Arizona	Recruiting
Clinical Trial Site	Stanford	California	Recruiting
Clinical Trial Site	Miramar	Florida	Recruiting
Clinical Trial Site	Orlando	Florida	Recruiting
Clinical Trial Site	Atlanta	Georgia	Recruiting
Clinical Trial Site	Chicago	Illinois	Recruiting
Clinical Trial Site	Boston	Massachusetts	Recruiting
Clinical Trial Site	Detroit	Michigan	Recruiting
Clinical Trial Site	Minneapolis	Minnesota	Recruiting
Clinical Trial Site	Jackson	Mississippi	Recruiting
Clinical Trial Site	Hackensack, NJ, 7601	New Jersey	Recruiting
Clinical Trial Site	New York	New York	Recruiting
Clinical Trial Site	The Bronx	New York	Recruiting
Clinical Trial Site	Philadelphia	Pennsylvania	Recruiting
Clinical Trial Site	Philadelphia	Pennsylvania	Recruiting
Clinical Trial Site	Philadelphia	Pennsylvania	Recruiting
Clinical Trial Site	Houston	Texas	Recruiting
Clinical Trial Site	Houston	Texas	Recruiting
Clinical Trial Site	Snyder	Texas	Recruiting
Clinical Trial Site	Tacoma	Washington	Recruiting
Clinical Trial Site	New Lambton Heights	New South Wales	Recruiting
Clinical Trial Site	Ghent	East Flanders	Recruiting
Clinical Trial Site	Campinas	São Paulo	Recruiting
Clinical Trial Site	São Paulo	Brazil	Recruiting
Clinical Trial Site	São Paulo	Brazil	Recruiting
Clinical Trial Site	Sofia	Sofia-Grad	Recruiting
Clinical Trial Site	Edmonton	Alberta	Recruiting
Clinical Trial Site	Halifax	Nova Scotia	Recruiting
Clinical Trial Site	London	Ontario	Recruiting
Clinical Trial Site	Québec	Canada	Recruiting
Clinical Trial Site	Zagreb	Croatia	Recruiting
Clinical Trial Site	Prague	Central Bohemia	Recruiting
Clinical Trial Site	Olomouc	Czechia	Recruiting
Clinical Trial Site	Bron	Auvergne-Rhône-Alpes	Recruiting
Clinical Trial Site	Clermont-Ferrand	Auvergne-Rhône-Alpes	Recruiting
Clinical Trial Site	Clermont-Ferrand	Auvergne-Rhône-Alpes	Recruiting
Clinical Trial Site	Grenoble	Auvergne-Rhône-Alpes	Recruiting
Clinical Trial Site	La Rochelle	New Aquitaine	Recruiting
Clinical Trial Site	Niort	New Aquitaine	Recruiting
Clinical Trial Site	Caen	Normandy	Recruiting

+ 64 more sites — see the full list on the official registry below.

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT01633489 on ClinicalTrials.gov ↗ ← All trials in the USA