Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102

Lysosomal Acid Lipase Deficiency Clinical Trial

Official title: An Open Label Multicenter Extension Study to Evaluate the Long-term Efficacy and Safety of SBC-102 in Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102

Status Terminated

Clinical Trial Summary

This phase 2/3, open-label extension study will evaluate the long-term efficacy and safety of intravenous (IV) infusions of SBC-102 in children with Lysosomal Acid Lipase (LAL) Deficiency who previously received treatment with SBC-102.

Clinical Trial Description

Early onset LAL Deficiency is a very rare form of LAL Deficiency, with an estimated prevalence of less than 2 lives per million (Meikle et al., 1999). This form of the disease, named after the physician who first described it (Abramov et al., 1956), is the most aggressive presentation of LAL Deficiency and is characterized by gastrointestinal and hepatic manifestations including marked growth failure, malabsorption, steatorrhea, and hepatomegaly. Early onset LAL Deficiency is rapidly progressive and fatal usually within the first year of life (Assmann & Seedorf, 2001).

The primary objective of the study is to evaluate the effect of SBC-102 therapy on overall survival at 12 months of age in children with growth failure due to LAL Deficiency.

All subjects will receive repeat IV infusions of SBC-102, beginning at least 1 week after the preceding infusion in study LAL-CL03 or under an expanded access treatment regimen. ;

Related Conditions & MeSH terms

• Lysosomal Acid Lipase Deficiency
• Wolman Disease

• NCT number: NCT01473875
• Study type: Interventional
• Source: Alexion Pharmaceuticals
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: November 2011
• Completion date: January 2015