Status:
RECRUITING
Lysosomal Acid Lipase (LAL) Deficiency Registry
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Lysosomal Acid Lipase Deficiency
Cholesterol Ester Storage Disease
Eligibility:
All Genders
Brief 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 pati...
Detailed Description
Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lysosomal storage disorder (LSD) that is caused by a marked decrease of lysosomal acid lipase (LAL), the enzyme that breaks down ch...
Eligibility Criteria
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.
Key Trial Info
Start Date :
May 30 2013
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 30 2029
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT01633489
Start Date
May 30 2013
End Date
August 30 2029
Last Update
March 6 2026
Active Locations (104)
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1
Clinical Trial Site
Phoenix, Arizona, United States, 85016
2
Clinical Trial Site
Stanford, California, United States, 94305
3
Clinical Trial Site
Miramar, Florida, United States, 33025
4
Clinical Trial Site
Orlando, Florida, United States, 32806