Lysosomal Acid Lipase (LAL) Deficiency Registry
Recruiting
Observational Study
Lysosomal Acid Lipase Deficiency
Cholesterol Ester Storage Disease
Wolman Disease
Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Acid Lipase Deficiency
LIPA Deficiency
LAL-Deficiency
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 300 (estimated)
- Sponsor
- Alexion Pharmaceuticals, Inc. · Industry
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About This Trial
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.
Trial Locations
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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 …
Contacts