Lysosomal Acid Lipase Deficiency Clinical Trial
— ALX-LALD-501Official title:
An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
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.
Status | Recruiting |
Enrollment | 1000 |
Est. completion date | June 30, 2029 |
Est. primary completion date | June 30, 2029 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Alexion |
United States, Australia, Belgium, Brazil, Canada, Croatia, Czechia, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Mexico, Netherlands, Poland, Portugal, Saudi Arabia, Slovenia, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Understanding of the variability, progression, identification and natural history of LAL Deficiency. | Ongoing |
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