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

NCT05676853 | Terminated Phase 3 FDA Drug

• 1 other identifier: CAEB1102-305
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Arginase I Deficiency; Hyperargininemia

Keywords

ARG1-D

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Pegzilarginase

EXPERIMENTAL

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

Drug: Pegzilarginase

Interventions

Pegzilarginase DRUG

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

Also known as: Co-ArgI-PEG, AEB1102

Pegzilarginase

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Aeglea Biotherapeutics: lead

Study Sites (1)

Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

Location

MeSH Terms

Conditions

Hyperargininemia

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, Inborn; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Cortney Caudill

  Aeglea BioTherapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2022
• First Posted: January 9, 2023
• Study Start: April 4, 2023
• Primary Completion: April 27, 2023
• Study Completion: April 27, 2023
• Last Updated: July 27, 2023

Record last verified: 2023-07

Locations

CA (1)