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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

Brief 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.

Detailed Summary:

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.


Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Overall survival [ Time Frame: 12 months ]

Original Primary Outcome: Overall survival at approximately 12 months of age [ Time Frame: Approximately 12 months ]

Current Secondary Outcome:

  • Survival rates at periodic intervals and median survival time. [ Time Frame: 3 years ]
  • Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency [ Time Frame: 3 years ]


Original Secondary Outcome:

  • Survival rates at periodic intervals and median survival time. [ Time Frame: Approximately 3 years ]
  • Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency [ Time Frame: Approximately 3 years ]


Information By: Alexion Pharmaceuticals

Dates:
Date Received: November 10, 2011
Date Started: November 2011
Date Completion:
Last Updated: February 17, 2017
Last Verified: February 2017