Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
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.
Lysosomal Acid Lipase Deficiency
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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|
- Overall survival [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
- Survival rates at periodic intervals and median survival time. [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
- Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
|Study Start Date:||November 2011|
|Estimated Study Completion Date:||January 2015|
|Estimated Primary Completion Date:||December 2014 (Final data collection date for primary outcome measure)|
SBC-102 Weekly IV infusions of SBC-102
SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicinal product is an enzyme replacement therapy intended for treatment of patients with LAL Deficiency.
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.