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

Completed · Phase 1/Phase 2

Conditions studied: Arginase I Deficiency, Hyperargininemia

In brief

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.

Key facts

Study ID

NCT02488044

Run by

Aeglea Biotherapeutics

People needed

16

Starts

2016-06-01

Expected to finish

2019-02-01

Last updated by the study team

2019-06-12

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Documented diagnosis of Arginase I deficiency
• Adequate organ function: Hgb ≥ 10 g/dL, ANC ≥ 1.5 x 109/L, plt count ≥ 100,000/µL; liver transaminase levels ≤ 2.5x ULN, total bilirubin ≤ 2.0 mg/dL; serum creatinine <1.5 x ULN
• If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
• If a sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration
• Patient or legal guardian is able and willing to provide written informed consent and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification) prior to any screening procedures

You may not qualify if…

• Transfusion of ≥ 2 u RBC within 60 days
• Active infection requiring systemic treatment
• Known infection with HIV, Hep B or Hep C
• Severe hyperammonemia requiring hospitalization within 14 days. Had more than one episode of hyperammonemia requiring hospitalization within the 30 days prior to enrollment.
• Current uncontrolled hyperammonemia
• Has a history of hypersensitivity to PEG or any other component of the AEB1102 (Co-ArgI-PEG) formulation
• If female, is lactating or breast feeding
• PART 2 INCLUSION CRITERION:
• Did not experience any safety or tolerability event in Part 1 which would preclude continued participation and dosing of AEB1102

Where it is running

• Stanford University School of Medicine — Stanford, California, United States
• University of Florida — Gainesville, Florida, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• UTSW — Dallas, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• Centro Hospitalar S. Joao — Porto, Portugal
• Great Ormond Street Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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