Last updated on September 2017

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


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

Detailed Study Description

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.

Clinical Study Identifier: TX152713

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University of Florida

University of Florida
Gainesville, FL USA
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Mt. Sinai

Mt. Sinai
New York, NY USA
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Stanford

Stanford
Palo Alto, CA USA
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University of Utah

University of Utah
Salt Lake City, UT USA
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Children’s National

Children’s National
Washington, DC USA
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Children’s Hospital of Michigan

Children’s Hospital of Michigan
Detroit, MI USA
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SickKids

SickKids
Toronto, XX Canada
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University of Texas Southwestern

University of Texas Southwestern
Dallas, TX USA
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Children’s Hospital of Pittsburgh

Children’s Hospital of Pittsburgh
Pittsburgh, PA USA
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Seattle Children’s Hospital

Seattle Children’s Hospital
Seattle, WA USA
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