Arginase 1 Deficiency Treatment with Pegzilarginase: The PEACE Study – Phase II

PEACE (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

Protocol Description

The objective of this international multi-center study is to see if the drug pegzilarginase can help people with arginase 1 deficiency. Arginase 1 deficiency is a rare disease caused by a defect in a specific gene that causes a deficiency of the enzyme called arginase 1 in the body. This enzyme’s job is to help break down the amino acid arginine. When arginase is not working, arginine can build up in the blood and can lead to serious effects on growth, health, and intellect.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, candidates must have a confirmed diagnosis of arginase 1 deficiency.
Males and Females: Ages 2 years and older

Requirements

Participants will be randomly assigned to a group that receives either a placebo or the study drug via intravenous infusion, weekly for 24 weeks. After that period all participants will receive weekly infusions of pegzilarginase for an additional 150 weeks along with individualized disease management.
Visits: About 178
Duration: About 3-1/2 years

Status: Open to Enrollment

Source(s) of Support

Aeglea BioTherapeutics

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Jessica Lindenberger, MSN, CRNP, FNP-C
412-692-7530