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

Completed · Phase 2/Phase 3

Conditions studied: Lysosomal Acid Lipase Deficiency, Wolman Disease

In brief

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.

Key facts

Study ID

NCT01371825

Run by

Alexion Pharmaceuticals, Inc.

People needed

9

Starts

2011-05-04

Expected to finish

2018-01-03

Last updated by the study team

2019-01-30

Who can join

Age: any, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Study site — Irvine, California, United States
• Study site — Cairo, Egypt
• Study site — Grenoble, France
• Study site — Paris, France
• Study site — Dublin, Ireland
• Study site — London, United Kingdom
• Study site — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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