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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02488044
Recruitment Status : Completed
First Posted : July 2, 2015
Last Update Posted : June 12, 2019
Information provided by (Responsible Party):
Aeglea Biotherapeutics

Brief 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.

Condition or disease Intervention/treatment Phase
Arginase I Deficiency Hyperargininemia Drug: AEB1102 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : June 2016
Actual Primary Completion Date : February 2019
Actual Study Completion Date : February 2019

Arm Intervention/treatment
Experimental: AEB1102

AEB1102, modified human Arginase I administered IV Part 1 Each patient may receive up to 7 doses given up to every other week over a maximum of 14 weeks.

Part 2 Each patient will receive up to 8 weeks of repeat-dose therapy.

Drug: AEB1102
modified human arginase I
Other Name: Co-ArgI-PEG

Primary Outcome Measures :
  1. Number of subjects with adverse events [ Time Frame: weekly throughout the study, up to 14 weeks ]
    Includes significant changes in hematology, chemistry and coagulation laboratory studies as well as in physical exam and vital signs

Secondary Outcome Measures :
  1. Number of subjects with a decrease from baseline in plasma arginine level [ Time Frame: Baseline to 2, 4, 6, 8 weeks ]
  2. Pharmacokinetic profile including Cmax, AUC, Tmax, T1/2 for each subject [ Time Frame: At 15 min, 1, 2, 4, 8, 12, 24, 48, 72, and 120 hours following dose escalation ]
  3. Number of subjects with a decrease from baseline in plasma guanidino compound levels [ Time Frame: Baseline to 2, 4, 6, 8 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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


1. Did not experience any safety or tolerability event in Part 1 which would preclude continued participation and dosing of AEB1102

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02488044

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United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, New York
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
United States, Texas
Dallas, Texas, United States, 75390
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Centro Hospitalar S. Joao
Porto, Portugal
United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sponsors and Collaborators
Aeglea Biotherapeutics
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Study Director: Josie Gayton Aeglea Biotherapeutics, Inc.
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Responsible Party: Aeglea Biotherapeutics Identifier: NCT02488044    
Other Study ID Numbers: CAEB1102-101A
First Posted: July 2, 2015    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: June 2019
Additional relevant MeSH terms:
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Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases