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Completed

NCT Number: NCT03378531

A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

The purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.

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Key information

Conditions

Arginase I Deficiency Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hyperargininemia Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Urea Cycle Disorders, Inborn

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Hospital for Sick Children, Toronto, Ontario, Canada

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About this study

Purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing
  • Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102
  • If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
  • If 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 where required assent, 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

  • Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances

Treatment and study plan

AEB1102

Drug

modified human arginase I

Other names: Co-ArgI-PEG, Pegzilarginase

Primary outcomes

  1. Incidence of treatment-related adverse events

    Time frame: up to 4 years

    Incidence of treatment-related adverse events

Secondary outcomes

  1. Cmax Cmin

    Time frame: up to 4 years

    Cmax Cmin

Sponsors and collaborators

Lead sponsor

Aeglea Biotherapeutics

Industry

Registry information

Official study title

An Open-label, Multicentre Extension Study to Evaluate the Long-Term Safety, Tolerability and Effects of Intravenous AEB1102 in Patients With Arginase I Deficiency Who Previously Received Treatment in Study CAEB1102-101A

Acronym: AEB1102

Important dates

Study start
2017
Primary completion
2022
Study completion
2022
First posted
Dec 20, 2017
Registry last updated
Jul 27, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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