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Lysosomal Acid Lipase (LAL) Deficiency Registry

NCT01633489 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 300

Last updated 2026-03-06

No results posted yet for this 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.

Conditions

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

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Alexion Pharmaceuticals · Sponsor GmbH

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-05-30
Primary Completion
2029-08-30
Completion
2029-08-30

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Bulgaria
  • Canada
  • Croatia
  • Czechia
  • France
  • Germany
  • Greece
  • Ireland
  • Israel
  • Italy
  • Mexico
  • Netherlands
  • Portugal
  • Russia
  • Saudi Arabia
  • Slovenia
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01633489 on ClinicalTrials.gov