Status:
TERMINATED
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Lysosomal Acid Lipase Deficiency
Eligibility:
All Genders
Up to 8 years
Phase:
PHASE2
Brief Summary
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 sebe...
Detailed Description
Lysosomal acid lipase deficiency is a rare autosomal recessive lipid storage disorder that is caused by a marked decrease or complete absence of the LAL enzyme, leading to the accumulation of lipids, ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
June 6 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 30 2018
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT02193867
Start Date
June 6 2014
End Date
October 30 2018
Last Update
November 18 2019
Active Locations (5)
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1
Phoenix, Arizona, United States, 85016
2
Kuopio, Finland
3
Naples, Italy
4
Birmingham, United Kingdom