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

NCT ID: NCT05676853

Last Updated: 2023-07-27

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-04-04
• Study Completion Date: 2023-04-27

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.

Conditions

Arginase I Deficiency; Hyperargininemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Pegzilarginase

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

Group Type: EXPERIMENTAL

Pegzilarginase

Intervention Type: DRUG

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

Interventions

Pegzilarginase

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

Intervention Type: DRUG

Other Intervention Names

Co-ArgI-PEG; AEB1102

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Aeglea Biotherapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Cortney Caudill

Role: STUDY_DIRECTOR

Aeglea BioTherapeutics, Inc.

Locations

Hospital for Sick Children

Toronto, Ontario, Canada

Countries

Canada

Other Identifiers

CAEB1102-305

Identifier Type: -

Identifier Source: org_study_id