A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency

July 25, 2023 updated by: Aeglea Biotherapeutics

A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency

This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 3

Contacts and Locations

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

Study Contact

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G1X8
        • Hospital for Sick Children

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date
  2. Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
  3. Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active
  4. If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device, or intrauterine hormone-releasing system

Exclusion Criteria:

  1. Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose
  2. Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study

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: Pegzilarginase
Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks
Drug: Pegzilarginase
Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
Other Names:
  • Co-ArgI-PEG
  • AEB1102

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment emergent adverse events
Time Frame: Baseline through week 54
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)
Baseline through week 54

Collaborators and Investigators

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

Sponsor

Aeglea Biotherapeutics

Investigators

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

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)

April 4, 2023

Primary Completion (Actual)

April 27, 2023

Study Completion (Actual)

April 27, 2023

Study Registration Dates

First Submitted

December 21, 2022

First Submitted That Met QC Criteria

January 6, 2023

First Posted (Actual)

January 9, 2023

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

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAEB1102-305

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

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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