Overview

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.