Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Summary

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; and a follow up visit for final safety assessments.

Timeline

Start

2019-04-10

Primary completion

2023-01-27

Completion

2023-01-27

Publications

• Russo RS, Gasperini S, Bubb G, Neuman L, Sloan LS, Diaz GA, Enns GM; PEACE Investigators. Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial. EClinicalMedicine. 2024 Jan 12;68:102405. doi: 10.1016/j.eclinm.2023.102405. eCollection 2024 Feb.

Drugs

Indications