Last updated on April 2018

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

Brief description of study

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.

Clinical Study Identifier: NCT02488044

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Roberto Zori, MD

University of Florida
Gainesville, FL United States
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George Diaz, MD

Icahn School of Medicine at Mount Sinai
New York, NY United States
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Gregory Enns, MD

Stanford University School of Medicine
Stanford, CA United States
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Gerard Vockley, MD

Children's Hospital of Pittsburgh of UPMC
Pittsburgh, PA United States
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Markey McNutt, MD

Dallas, TX United States
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Nicola Longo, MD

University of Utah
Salt Lake City, UT United States
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Lawrence Merritt, MD

Seattle Children's Hospital
Seattle, WA United States
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Andreas Schultze, MD

The Hospital for Sick Children
Toronto, ON Canada
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