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

Phase 3

Terminated

• Conditions: Arginase I Deficiency; Hyperargininemia
• Interventions: Drug: Pegzilarginase

• Registration Number: NCT05676853
• Lead Sponsor: Aeglea Biotherapeutics

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.

Study Timeline

• Study First Submitted: 2022-12-21
• Study First Submitted QC: 2023-01-06
• Study First Posted: 2023-01-09
• Study Start: 2023-04-04
• Primary Completion: 2023-04-27
• Study Completion: 2023-04-27
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-25
• Last Update Posted: 2023-07-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pegzilarginase	Pegzilarginase	Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks

Primary Outcome Measures

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

Trial Locations

Locations (1)

Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada