NCT02488044

A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

A Phase 1/2 Open-label Study in Patients With Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Aeglea Biotherapeutics · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Arginase I Deficiency
• Hyperargininemia

Interventions

Type	Name	Description
DRUG	AEB1102	modified human arginase I

Timeline

• Start date: 2016-06-01
• Primary completion: 2019-02-01
• Completion: 2019-02-01
• First posted: 2015-07-02
• Last updated: 2019-06-12

Locations

9 sites across 4 countries: United States, Canada, Portugal, United Kingdom