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Sanofi Genzyme Begins Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency

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Additional Relevant Details ASCEND is a Phase 2/3 multi-national, multi-center, double-blinded, placebo-controlled trial to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in adult patients with ASMD, specifically NPD B. The Phase 2/3 trial will assess the effect of olipudase alfa on spleen size, lung function and other important clinical parameters. Thirty-six patients are expected to be enrolled in the study and receive olipudase alfa or a placebo. Upon completion of the 52 week primary analysis period, all patients will receive treatment in an extension period.
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Catalyst Date
Occurred on:
Jul 06, 2016
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Related Keywords Olipudase Alfa, Acid Sphingomyelinase Deficiency, Niemann-pick Disease Type B, Enzyme Acid Sphingomyelinase, Ascend