Last updated on July 2020

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency


Brief description of study

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.

Detailed Study Description

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. After 8 weeks of the LTE study, patients have the option to receive treatment by subcutaneous administration (SC).

Clinical Study Identifier: NCT03921541

Find a site near you

Start Over

Seattle Children's Hospital

Seattle, WA United States
  Connect »

LKH Bregenz

Bregenz, Austria
  Connect »

Salford Royal

Salford, United Kingdom
  Connect »

Hopital Purpan

Toulouse Cedex 9, France
  Connect »

Harvey Pediatrics

Rogers, AR United States
  Connect »

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


Volunteer Sign-up

Sign up for our FREE service to receive email notifications when clinical trials are posted in the medical category of interest to you.