Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

 Hyperargininemia / Posted 11 months ago

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.

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.

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 individualized disease management (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. During the long-term extension, all subjects receive pegzilarginase plus IDM.

  • Study start date : 01/04/2019
  • Study end date : 01/03/2021
  • Wales-Based Study Contact : please speak to your clinician
Contact details

University Hospital of Wales Cardiff Birmingham Children's Hospital Birmingham Great Ormond Street Hospital for Children London Salford Royal SalfordUniversity Hospital of Wales,Cardiff raredisease@aegleabio.com bepartofresearch.nihr.ac.uk

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