NCT06582524

Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

A Phase 3 Open-Label Study of Safety, Pharmacokinetics, and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Immedica Pharma AB · Industry
• Sex: All
• Age: 1 Day – 24 Months
• Healthy volunteers: Not accepted

Summary

This is an open-label, multicentre study to evaluate the safety, PK, and activity (PD) of weekly subcutaneous (SC) administration of pegzilarginase in subjects with ARG1-D who are \< 24 months of age. The study consists of a screening period of up to 4 weeks, a subsequent 12-week treatment period, and a safety follow-up period of 8 weeks.

Detailed description

CAEB1102-301A is an open-label, single-arm, non-controlled, repeat dosing, multicentre study to evaluate the safety, PK, and activity (PD) of weekly SC administration of pegzilarginase over 12 weeks in subjects with ARG1-D who are \< 24 months of age. This study will consist of: * A screening period of up to 4 weeks to ensure the subjects meet the study eligibility criteria and establish baseline plasma arginine * A treatment period of 12 weeks * A safety follow-up period of 8 weeks with visits 1 week and 8 weeks after the last dose.

Conditions

• Arginase 1 Deficiency

Interventions

Type	Name	Description
DRUG	Pegzilarginase	SC administration of pegzilarginase over 12 weeks in subjects with ARG1-D who are \< 24 months of age

Timeline

• Start date: 2024-08-30
• Primary completion: 2025-06-17
• Completion: 2025-06-17
• First posted: 2024-09-03
• Last updated: 2025-11-28

Locations

3 sites across 3 countries: Austria, Portugal, United Kingdom