Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

NCT ID: NCT06582524

Last Updated: 2025-11-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-30
• Study Completion Date: 2025-06-17

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.

Detailed Description

CAEB1102-301A is an open-label, single-arm, non-controlled, repeat dosing, multicentre study to evaluate the safety, PK, and activity (PD) of weekly SC administration of pegzilarginase over 12 weeks in subjects with ARG1-D who are < 24 months of age.

This study will consist of:

• A screening period of up to 4 weeks to ensure the subjects meet the study eligibility criteria and establish baseline plasma arginine
• A treatment period of 12 weeks
• A safety follow-up period of 8 weeks with visits 1 week and 8 weeks after the last dose.

Conditions

Arginase 1 Deficiency

Study Design

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

Study Groups

Weekly subcutaneous (SC) administration of pegzilarginase

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

Group Type: EXPERIMENTAL

Pegzilarginase

Intervention Type: DRUG

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

Interventions

Pegzilarginase

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

Intervention Type: DRUG

Other Intervention Names

Loargys

Eligibility Criteria

Inclusion Criteria

1. Subjects must be < 24 months of age on the date of informed consent

2. Confirmed diagnosis of ARG1-D documented in medical records by at least 1 of the following methods:

1. elevated plasma arginine levels

2. a mutation analysis revealing a pathogenic variant

3. red blood cell (RBC) arginase activity

3. Subjects must weigh > 8 kg due to clinical trial related blood collection volumes required

4. 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

5. At least one value of plasma arginine ≥ 180 μM during screening

6. Documented confirmation from the Investigator and/or dietitian that the subject can:

1. attempt to maintain a stable, age-appropriate level of protein consumption, including natural protein, and EAA supplementation within approximately ± 15% of dietitian recommended diet

2. attempt to maintain current use of ammonia scavengers, if prescribed

Exclusion Criteria

1. Other medical condition(s) or comorbidity(ies) that, in the opinion of the Investigator, would interfere with study compliance or data interpretation

2. 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

3. Active infection requiring anti-infective therapy within < 2 weeks before first dose of study drug

4. Known active infection with human immunodeficiency virus, hepatitis B, or hepatitis C

5. 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

6. 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

7. Previous liver or haematopoietic stem cell transplant

8. Use of botulinum toxin within 16 weeks prior to first dose

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 24 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Immedica Pharma AB

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mattias Rudebeck, PhD MSc BMedSc

Role: STUDY_DIRECTOR

Immedica Pharma AB

Locations

Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität

Graz, , Austria

Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria

Lisbon, , Portugal

Bradford Royal Infirmary Duckworth Lane

Bradford, , United Kingdom

Countries

Austria; Portugal; United Kingdom

Other Identifiers

CAEB1102-301A (Europe)

Identifier Type: -

Identifier Source: org_study_id