Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Trial end date:
SummaryThis 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
- 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.
- 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.