Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2025-10-01
Target enrollment:
Participant gender:
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.