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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Arginase I Deficiency, Hyperargininemia

Intervention / Treatment
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* Drug: Pegzilarginase


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pegzilarginase<br>Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks | Drug: Pegzilarginase<br>* Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated<br>* Other names: AEB1102;|

What is the study measuring?
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Primary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ARG1-D