Clinical Trial: Screening for Lysosomal Acid Lipase Deficiency
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational
Official Title: SCREENING FOR LYSOSOMAL ACID LIPASE DEFICIENCY AS THE UNDERLYING SOURCE OF HEPATIC INJURY IN PEDIATRIC PATIENTS WITH EVIDENCE OF ABNORMAL CLINICAL OR BIOCHEMICAL TESTS (DE
Brief Summary: The primary outcome of this study is the development of a clinical profile of pediatric patients with LAL-D, which will enable the Sponsor to provide more focused guidance to the medical community as to which pediatric patients should be tested for LAL-D.
Detailed Summary:
Sponsor: Alexion Pharmaceuticals
Current Primary Outcome: Eligibility Criteria for LAL-D diagnosed patients [ Time Frame: Confirmed LAL-D diagnosed patients, for a period extending up to a maximum of 6 months after the date of diagnosis ]
Original Primary Outcome: Same as current
Current Secondary Outcome: LIPA gene mutations for LAL-D diagnosed patients [ Time Frame: Confirmed LAL-D diagnosed patients, for a period extending up to a maximum of 6 months after the date of diagnosis ]
Original Secondary Outcome: Same as current
Information By: Alexion Pharmaceuticals
Dates:
Date Received: October 4, 2016
Date Started: November 2016
Date Completion: June 2019
Last Updated: April 3, 2017
Last Verified: April 2017