Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

NCT06582524 | Completed Phase 3 Results

• 1 other identifier: CAEB1102-301A (Europe)
• Study Type: interventional
• Target: 3
• Locations: 3 countries
• Sites: 3

Brief Summary

This is an open-label, multicentre study to evaluate the safety, PK, and activity (PD) of weekly subcutaneous (SC) administration of pegzilarginase in subjects with ARG1-D who are \< 24 months of age. The study consists of a screening period of up to 4 weeks, a subsequent 12-week treatment period, and a safety follow-up period of 8 weeks.

Conditions

Arginase 1 Deficiency

Outcome Measures

Primary Outcomes (1)

• To Evaluate the Effect of Pegzilarginase

  To evaluate the effect of pegzilarginase on plasma arginine concentrations in subjects \<24 months of age with arginase 1 deficiency (ARG1-D)

  From baseline up to 12 weeks

Secondary Outcomes (7)

• To Characterize the Pharmacokinetic (PK) Profile of Pegzilarginase

  From baseline up to 12 weeks

• To Characterize the Pharmacokinetic (PK) Profile of Pegzilarginase

  From baseline up to 12 weeks

• To Characterize the Pharmacokinetic (PK) Profile of Pegzilarginase

  From baseline up to 12 weeks

• To Characterize the Pharmacokinetic (PK) Profile of Pegzilarginase

  From baseline up to 12 weeks

• To Evaluate the Pharmacodynamic (PD) Response of Pegzilarginase

  From baseline up to 12 weeks

Study Arms (1)

Weekly subcutaneous (SC) administration of pegzilarginase

EXPERIMENTAL

All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks

Drug: Pegzilarginase

Interventions

Pegzilarginase DRUG

SC administration of pegzilarginase over 12 weeks in subjects with ARG1-D who are \< 24 months of age

Also known as: Loargys

Weekly subcutaneous (SC) administration of pegzilarginase

Eligibility Criteria

• Age: 1 Day - 24 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects must be \< 24 months of age on the date of informed consent
• Confirmed diagnosis of ARG1-D documented in medical records by at least 1 of the following methods:
• elevated plasma arginine levels
• a mutation analysis revealing a pathogenic variant
• red blood cell (RBC) arginase activity
• Subjects must weigh \> 8 kg due to clinical trial related blood collection volumes required
• Written informed consent by parent/legal guardian, in accordance with national stipulations, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
• At least one value of plasma arginine ≥ 180 μM during screening
• Documented confirmation from the Investigator and/or dietitian that the subject can:
• attempt to maintain a stable, age-appropriate level of protein consumption, including natural protein, and EAA supplementation within approximately ± 15% of dietitian recommended diet
• attempt to maintain current use of ammonia scavengers, if prescribed

You may not qualify if:

• Other medical condition(s) or comorbidity(ies) that, in the opinion of the Investigator, would interfere with study compliance or data interpretation
• Hyperammonaemic episode (plasma ammonia levels \> 100 μM) with ≥ 1 symptom related to hyperammonaemia requiring hospitalisation or emergency room management within the 4 weeks before the first dose of study drug
• Active infection requiring anti-infective therapy within \< 2 weeks before first dose of study drug
• Known active infection with human immunodeficiency virus, hepatitis B, or hepatitis C
• History of hypersensitivity to polyethylene glycol (PEG) or any of the excipients included in the study drug that, in the judgment of the Investigator, puts the subject at unacceptable risk for AEs
• Currently participating in another therapeutic clinical study or has received any investigational agent within 30 days (or 5 half-lives, whichever is longer) prior to first dose of study drug
• Previous liver or haematopoietic stem cell transplant
• Use of botulinum toxin within 16 weeks prior to first dose

Sponsors & Collaborators

• Immedica Pharma AB: lead

Study Sites (3)

Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität

Graz, A-8036, Austria

Location

Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria

Lisbon, Portugal

Location

Bradford Royal Infirmary Duckworth Lane

Bradford, BD9 6RJ, United Kingdom

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

Results Point of Contact

• Title: VP Head of Global Integrated Evidence Generation
• Organization: Immedica Pharma AB

clinical@immedica.com

0046 8 533 39 500

Study Officials

• Mattias Rudebeck, PhD MSc BMedSc

  Immedica Pharma AB

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

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: August 20, 2024
• First Posted: September 3, 2024
• Study Start: August 30, 2024
• Primary Completion: June 17, 2025
• Study Completion: June 17, 2025
• Last Updated: April 30, 2026
• Results First Posted: April 30, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

PT (1); AU (1); GB (1)