Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Stopped early · Phase 2

Conditions studied: Lysosomal Acid Lipase Deficiency

In brief

This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.

Key facts

Study ID

NCT02193867

Run by

Alexion Pharmaceuticals, Inc.

People needed

10

Starts

2014-06-06

Expected to finish

2018-10-30

Last updated by the study team

2019-11-18

Who can join

Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participant's parent or legal guardian (if applicable) consent to participation in the study
• Confirmation of documented decreased LAL activity relative to the normal range of the lab performing the assay or confirmation of LAL-D diagnosis as determined by a Sponsor-approved central laboratory
• Substantial clinical concerns, in the opinion of Investigator and Sponsor, of rapid disease progression requiring urgent medical intervention including, but not restricted to the following:
• Marked abdominal distension and hepatomegaly
• Failure to thrive
• Disturbance of coagulation
• Severe anemia
• Sibling with rapidly progressive course of LAL-D

You may not qualify if…

• Clinically important concurrent disease
• Participant was > 8 months of age at the time of first dosing
• Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study
• Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation
• Previous hematopoietic stem cell or liver transplant
• Known hypersensitivity to eggs

Where it is running

• Study site — Phoenix, Arizona, United States
• Study site — Kuopio, Finland
• Study site — Naples, Italy
• Study site — Birmingham, United Kingdom
• Study site — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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