8:53AM Synageva Biopharma provides clinical trial updates with Sebelipase ALFA in LAL deficiency (GEVA) 66.13 : Co announces total enrollment is complete in ARISE (Acid Lipase Replacement Investigating Safety and Efficacy), a global Phase 3 trial with sebelipase alfa in children and adults with lysosomal acid lipase deficiency (LAL Deficiency). Enrollment exceeded the original target of 50 patients with 66 randomized as of December 31, 2013. As previously announced, the company expects to report top-line results from the ARISE trial during the second half of 2014.
In addition, the company announces that it met the enrollment target in the Phase 2/3 trial in infants with LAL Deficiency. Nine patients enrolled in this open-label trial and preliminary results will be presented at the 10th Annual Lysosomal Disease Network WORLD Symposium being held February 11-13 in San Diego, California.
Phase 3 ARISE trial with sebelipase alfa in children and adults with LAL Deficiency
The ARISE trial is a randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with LAL Deficiency and is designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with the disease. Patients enrolled in the trial are randomized to infusions of sebelipase alfa (1 mg/kg, every other week) or placebo for the 20-week, double-blind treatment period and then allowed to enter into a long-term, open-label extension period. The efficacy and safety results from the double-blind treatment period are expected to support global submissions for product registration.
Phase 2/3 trial with sebelipase alfa in infants with LAL Deficiency
The Phase 2/3 trial is an open-label, multicenter study of sebelipase alfa in infants with LAL Deficiency. Infants with growth failure before six months of age were eligible to enroll and receive weekly infusions with sebelipase alfa. The primary endpoint of the trial is survival at 12 months of age.
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