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

Primary Purpose

Arginase I Deficiency, Hyperargininemia

Status
Terminated
Phase
Phase 3
Locations
Canada
Study Type
Interventional
Intervention
Pegzilarginase
Sponsored by
Aeglea Biotherapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Arginase I Deficiency focused on measuring ARG1-D

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date 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 Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active 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: Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study

Sites / Locations

  • Hospital for Sick Children

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Pegzilarginase

Arm Description

Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks

Outcomes

Primary Outcome Measures

Treatment emergent adverse events
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)

Secondary Outcome Measures

Full Information

First Posted
December 21, 2022
Last Updated
July 25, 2023
Sponsor
Aeglea Biotherapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT05676853
Brief Title
A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
Official Title
A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
Sponsor Decision
Study Start Date
April 4, 2023 (Actual)
Primary Completion Date
April 27, 2023 (Actual)
Study Completion Date
April 27, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aeglea Biotherapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Arginase I Deficiency, Hyperargininemia
Keywords
ARG1-D

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pegzilarginase
Arm Type
Experimental
Arm Description
Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks
Intervention Type
Drug
Intervention Name(s)
Pegzilarginase
Other Intervention Name(s)
Co-ArgI-PEG, AEB1102
Intervention Description
Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
Primary Outcome Measure Information:
Title
Treatment emergent adverse events
Description
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)
Time Frame
Baseline through week 54

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date 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 Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active 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: Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Cortney Caudill
Organizational Affiliation
Aeglea BioTherapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G1X8
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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