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.
FEATURED Cannabix Technologies and Omega Laboratories Inc. Advance Marijuana Breathalyzer Technology - Dr. Bruce Goldberger to Present at Society of Forensic Toxicologists Conference • Sep 24, 2024 8:50 AM
FEATURED Integrated Ventures, Inc Announces Strategic Partnership For GLP-1 (Semaglutide) Procurement Through MedWell USA, LLC. • Sep 24, 2024 8:45 AM
Avant Technologies Accelerates Creation of AI-Powered Platform to Revolutionize Patient Care • AVAI • Sep 24, 2024 8:00 AM
VHAI - Vocodia Partners with Leading Political Super PACs to Revolutionize Fundraising Efforts • VHAI • Sep 19, 2024 11:48 AM
Dear Cashmere Group Holding Co. AKA Swifty Global Signs Binding Letter of Intent to be Acquired by Signing Day Sports • DRCR • Sep 19, 2024 10:26 AM
HealthLynked Launches Virtual Urgent Care Through Partnership with Lyric Health. • HLYK • Sep 19, 2024 8:00 AM