Trial ID or NCT#

NCT02488044

Status

RECRUITING

Purpose

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.

Official Title

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

Eligibility Criteria

Ages Eligible for Study: Older than 2 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No

Investigator(s)

Gregory Enns
Geneticist
Professor of Pediatrics (Genetics) at the Lucile Salter Packard Children's Hospital
Chung Lee
Clinical Assistant Professor, Pediatrics - Medical Genetics

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CONTACT

Gregory Enns, MD