Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

• Status: Completed
• Trial end date: 2018-01-03
• Target enrollment: 0
• Participant gender: All

Summary

This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Inclusion Criteria:

• Participant's parent or legal guardian provided written informed consent/permission prior to any study procedures.

• Male or female child with documented decreased LAL activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis.

• Growth failure with onset before 6 months of age.

Exclusion Criteria:

• Clinically important concurrent disease or comorbidities.

• Had received an investigational product other than sebelipase alfa within 14 days prior to the first dose.

• Participant was older than 24 months of age.

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

• Previous hematopoietic stem cell or liver transplant.

• Known hypersensitivity to eggs.