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A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

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

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

NCT02488044•Aeglea Biotherapeutics

View on ClinicalTrials.gov

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.

Eligibility

Age

2 - No limit years

Sex

ALL

Healthy Volunteers

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
•PART 2 INCLUSION CRITERION:
•1\. Did not experience any safety or tolerability event in Part 1 which would preclude continued participation and dosing of AEB1102

Locations (9)

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

Key Dates

Start Date

June 1, 2016

Primary Completion Date

February 1, 2019

Completion Date

February 1, 2019

Last Updated

June 12, 2019

Enrollment

ACTUAL participants

Conditions

Arginase I DeficiencyHyperargininemia

Interventions

AEB1102

DRUG

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

NCT03921541

Arginase I DeficiencyHyperargininemia

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 12, 2019Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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