Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

• Status: Terminated
• Trial end date: 2018-10-30
• Target enrollment: 0
• Participant gender: All

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 sebelipase alfa for up to 3 years.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Inclusion Criteria:

1. Participant's parent or legal guardian (if applicable) consent to participation in the study

2. 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

3. 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 Criteria:

1. Clinically important concurrent disease

2. Participant was > 8 months of age at the time of first dosing

3. Participant received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in this study

4. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation

5. Previous hematopoietic stem cell or liver transplant

6. Known hypersensitivity to eggs