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

Hyperargininemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05676853
• Other study ID #: CAEB1102-305
• Status: Terminated
• Phase: Phase 3
• Start date: April 4, 2023
• Est. completion date: April 27, 2023

Study information

• Verified date: July 2023
• Source: Aeglea Biotherapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 3
• Est. completion date: April 27, 2023
• Est. primary completion date: April 27, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Arginase I Deficiency
• Hyperargininemia

Intervention

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

Locations

Country	Name	City	State
Canada	Hospital for Sick Children	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
Aeglea Biotherapeutics

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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