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