A Phase III Multicenter Study of SBC-102 (sebelipase alfa) in patients with Lysosomal Acid Lipase (LAL) Deficiency

A Phase III Multicenter Study of SBC-102 (sebelipase alfa) in patients with Lysosomal Acid Lipase (LAL) Deficiency

Lysosomal Acid Lipase Deficiency (LALD) is a genetic disease which is characterized by abnormal lipid accumulation in many parts of the body due to a marked decrease in activity of the enzyme lysosomal acid lipase (LAL). Although a single disease, LALD presents with two major forms: early onset and late onset. Early onset LALD, also known as Wolman Disease, is characterized by severe malabsorption, growth failure, and hepatic failure and is usually fatal within the first year of life.
 
The late onset form of the disease, also known as Cholesteryl Ester Storage Disease (CESD), occurs in both children and adults and is an under-appreciated cause of cirrhosis, liver failure and dyslipidemia. Late onset LAL Deficiency is characterized by an enlarged liver, abnormal liver function tests (LFTs) and elevated cholesterol levels. Some patients may also have an enlarged spleen.
 
The ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) clinical study will evaluate the safety and efficacy of sebelipase alfa (SBC-102, recombinant human LAL) administered every other week as an intravenous infusion in patients with late onset LAL Deficiency.
 
For entry into this study, the patient must be 4 years of age or older.  The patient would be excluded if he/she has had a previous hematopoietic or liver transplant procedure.
 
Patients diagnosed with LAL Deficiency, may be eligible for enrollment.  If you have any questions about the study, you may contact clinicaltrials@synageva.com.

Additional details of the study, including the location of clinical trial sites, are also available at www.clinicaltrials.gov/ct2/show/NCT01757184 or www.clinicaltrials.gov (search term "sebelipase alfa").


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