Clinical Trial: Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Lysosomal Acid Lipase Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency

Brief Summary: This is the first clinical study of SBC-102 (sebelipase alfa) for the treatment of LAL Deficiency. It is an open label dose escalation study in adult patients with liver dysfunction due to Lysosomal Acid Lipase (LAL) Deficiency and will examine three doses of SBC-102 (sebelipase alfa). The targeted number for this study is 9 evaluable subjects.

Detailed Summary:

Cholesteryl Ester Storage Disease (CESD) is the late onset phenotype for Lysosomal Acid Lipase (LAL) Deficiency, a Lysosomal Storage Disorder, which also has an early onset phenotype known as Wolman Disease that primarily affects infants. CESD can present in childhood but often goes unrecognized until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are common to patients with other liver conditions.

CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly, persistently abnormal liver function tests (LFTs) and type II hyperlipidemia. Splenomegaly and evidence of mild hypersplenism may affect some patients. Untreated, CESD may lead to fibrosis, cirrhosis, liver failure and death.

Disease Risk In Families:

  • 25 per million incidence
  • Autosomal recessive disorder, LAL deficiency is carried on chromosome 10
  • Parents with an affected son or daughter have a 1 in 4 chance of having another affected child

Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Safety and Tolerability of SBC-102 (sebelipase alfa) [ Time Frame: 4 weeks ]

The safety and tolerability of weekly infusions of SBC-102 (sebelipase alfa) will be assessed by routine monitoring of patients for adverse events (AEs) and monitoring changes from baseline in physical examination findings, vital signs, clinical laboratory evaluations, immunogenicity tests and concomitant therapies.


Original Primary Outcome: Safety and Tolerability of SBC-102 [ Time Frame: 4 weeks ]

The safety and tolerability of weekly infusions of SBC-102 will be assessed by routine monitoring of patients for adverse events (AEs) and monitoring changes from baseline in physical examination findings, vital signs, clinical laboratory evaluations, immunogenicity tests and concomitant therapies.


Current Secondary Outcome: Pharmacokinetics of SBC-102 (sebelipase alfa) [ Time Frame: 4 weeks ]

Characterize the pharmacokinetics of SBC-102 (sebelipase alfa) delivered by IV infusion after single and multiple doses.


Original Secondary Outcome: Pharmacokinetics of SBC-102 [ Time Frame: 4 weeks ]

Characterize the pharmacokinetics of SBC-102 delivered by IV infusion after single and multiple doses.


Information By: Alexion Pharmaceuticals

Dates:
Date Received: March 1, 2011
Date Started: May 2011
Date Completion:
Last Updated: July 26, 2016
Last Verified: June 2014