NCT03921541

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Aeglea Biotherapeutics · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Detailed 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).

Conditions

• Arginase I Deficiency
• Hyperargininemia

Interventions

Type	Name	Description
DRUG	Pegzilarginase	Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
DRUG	Placebo	Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

Timeline

• Start date: 2019-04-10
• Primary completion: 2023-01-27
• Completion: 2023-01-27
• First posted: 2019-04-19
• Last updated: 2024-11-19
• Results posted: 2024-11-19

Locations

32 sites across 7 countries: United States, Austria, Canada, France, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study