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

July 25, 2023 updated by: Aeglea Biotherapeutics

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

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.

Study Overview

Status

Completed

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
      • Porto, Portugal
        • Centro Hospitalar S. Joao
      • London, United Kingdom
        • Great Ormond Street Hospital
    • California
      • Stanford, California, United States, 94305
        • Stanford University School of Medicine
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • New York
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai
    • Texas
      • Dallas, Texas, United States, 75390
        • UTSW

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing
  2. Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102
  3. If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
  4. 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
  5. 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:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AEB1102
Each patient may receive AEB1102 administered IV for up to approximately 4 years.
modified human arginase I
Other Names:
  • Co-ArgI-PEG
  • Pegzilarginase

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment-related adverse events
Time Frame: up to 4 years
Incidence of treatment-related adverse events
up to 4 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cmax Cmin
Time Frame: up to 4 years
Cmax Cmin
up to 4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Cortney Caudill, Aeglea BioTherapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 7, 2017

Primary Completion (Actual)

December 15, 2022

Study Completion (Actual)

December 15, 2022

Study Registration Dates

First Submitted

December 11, 2017

First Submitted That Met QC Criteria

December 14, 2017

First Posted (Actual)

December 20, 2017

Study Record Updates

Last Update Posted (Actual)

July 27, 2023

Last Update Submitted That Met QC Criteria

July 25, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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