NCT01633489 (ALX-LALD-501) is a clinical trial in Lysosomal Acid Lipase Deficiency, sponsored by Alexion Pharmaceuticals, Inc..

Who is sponsoring NCT01633489?

NCT01633489 is sponsored by Alexion Pharmaceuticals, Inc..

What condition does NCT01633489 study?

NCT01633489 studies Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, LIPA Deficiency, LAL-Deficiency.

Is NCT01633489 still recruiting?

NCT01633489 is currently recruiting now. Last updated 6 March 2026.

Last reviewed 2026-03-06 · How we verify

NCT01633489: ALX-LALD-501

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Lysosomal Acid Lipase (LAL) Deficiency Registry

Recruiting now Last updated 6 March 2026

What this trial tests

trial in Lysosomal Acid Lipase Deficiency in 300 participants. Currently enrolling.

Timeline

30 May 2013

Primary endpoint
30 August 2029

30 August 2029

Quick facts

Lead sponsor	Alexion Pharmaceuticals, Inc.
Status	Recruiting now
Study type	OBSERVATIONAL
Enrollment	300
Start date	30 May 2013
Primary completion	30 August 2029
Estimated completion	30 August 2029
Sites	104 locations across Italy, Ireland, Croatia, Netherlands, Russia, Belgium, Mexico, Bulgaria

Conditions studied

Lysosomal Acid Lipase Deficiency — all drugs for Lysosomal Acid Lipase Deficiency →
Cholesterol Ester Storage Disease — all drugs for Cholesterol Ester Storage Disease →
Wolman Disease — all drugs for Wolman Disease →
Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 — all drugs for Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 →

Sponsor

Alexion Pharmaceuticals, Inc. — full company profile →

Who can join

Eligibility, any sex, with Lysosomal Acid Lipase Deficiency or Cholesterol Ester Storage Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Lysosomal acid lipase deficiency manifestations in children and adults: Baseline data from an international registry.
Balwani M, Balistreri W, D'Antiga L, Evans J, et al · · 2023 · cited 10× · PMID 37222260 · DOI 10.1111/liv.15620
From LAL-D to MASLD: Insights into the role of LAL and Kupffer cells in liver inflammation and lipid metabolism.
Bradić I, Kuentzel KB, Pirchheim A, Rainer S, et al · · 2025 · cited 6× · PMID 39486573 · DOI 10.1016/j.bbalip.2024.159575
Clinical outcome of a patient with lysosomal acid lipase deficiency and first results after initiation of treatment with Sebelipase alfa: A case report.
Soll D, Spira D, Hollstein T, Haberbosch L, et al · · 2019 · cited 3× · PMID 31249784 · DOI 10.1016/j.ymgmr.2019.100479
Sebelipase Alfa Improves Aminotransferase Levels in Lysosomal Acid Lipase Deficiency: Data From an International Registry.
D'Antiga L, Evans J, Ros E, Abel F, et al · · 2025 · cited 2× · PMID 40781810 · DOI 10.1111/liv.70279
Survival, growth, and safety findings in patients with rapidly progressive, infantile-onset LAL-D: Results from the international LAL-D registry.
Vijay S, Evans J, Lacaille F, Abel F, et al · · 2025 · PMID 41270440 · DOI 10.1016/j.ymgme.2025.109290
Abstract
· 2025
ESPGHAN 57th Annual Meeting Abstracts
· 2025
A216 CLINICAL MANIFESTATIONS OF LYSOSOMAL ACID LIPASE DEFICIENCY (LAL-D): THE INTERNATIONAL LAL-D REGISTRY
Francis M, Balwani M, Balistreri W, D’Antiga L, et al · · 2021

Verify or expand the search:

Other recruiting trials for Lysosomal Acid Lipase Deficiency

Currently open trials in the same condition.

NCT07455864 — Lysosomal Acid Lipase Deficiency in Risk Groups · recruiting

Other Alexion Pharmaceuticals, Inc. trials

Trials by the same sponsor.

NCT06015750 — Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia · Phase 4 · withdrawn
NCT07352423 — Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants · Phase 1 · recruiting
NCT07221838 — A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab · Phase 4 · recruiting
NCT07413250 — Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis o · active not recruiting
NCT07308574 — Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS · Phase 4 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01633489 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
Last refreshed: 6 March 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01633489.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing