A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency
Status:
Completed
Trial end date:
2019-02-01
Target enrollment:
Participant gender:
Summary
A Phase 1/2 Open-label Study in Patients with Arginase I Deficiency to Investigate the
Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102. This study is designed
to evaluate the safety and tolerability of IV administration of AEB1102 for the treatment of
pediatric and adult patients with Arginase I deficiency and hyperargininemia. This study will
be conducted in 2 parts: Part 1 (Single Ascending Dose Escalation) and Part 2 (Repeated
Dosing). Each part will be preceded by a baseline assessment of arginine levels. All patients
who participate in Part 1 may continue AEB1102 dosing in Part 2 if they qualify for continued
dosing. A data safety monitoring board (DSMB) will provide independent review of study safety
data and recommend whether the sponsor should continue the study as planned, modify the study
protocol, or discontinue the study.