CMPEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults with Arginase 1 Deficiency
Study ID: STU-2019-0572
CaeB1102-300a is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with aRG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension a follow-up visit for final safety assessments. Subjects will be randomized to treatment following completion of all screening assessments and confirmation of study eligibility in a 2:1 ratio to receive weekly iV infusions of pegzilarginase plus iDM or placebo plus iDM during the 24-week double blind treatment period. after completion of the 24-week double-blind treatment period, each subject will enter the long term, open-label extension, the first 8 weeks of which are blinded. (see Section 6.6). During the longterm extension, all subjects receive pegzilarginase plus iDM.
PRiMaRY: * To demonstrate the efficacy of pegzilarginase relative to placebo based on a statistically significant decrease in plasma arginine concentrations. Secondary: * To demonstrate the efficacy of pegzilarginase relative to placebo based on a statistically significantly greater proportion of clinical responders * To compare the proportions of subjects with response in the individual components of the key secondary endpoint between pegzilarginase and placebo * To compare pegzilarginase with placebo with respect to the number of individual components of the key secondary endpoint exhibiting response * To compare pegzilarginase with placebo with respect to the proportion of subjects whose endpoint arginine value falls below target guidance of 200 [MiCRo-SYMBoL]mol/L * To compare pegzilarginase with placebo with respect to the proportions of subjects whose endpoint arginine value falls within the normal range of 40-115[MiCRo-SYMBoL]mol/L * To compare pegzilarginase with placebo for chan[See protocol for complete text]