Status:
COMPLETED
Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
Lead Sponsor:
Immedica Pharma AB
Conditions:
Arginase 1 Deficiency
Eligibility:
All Genders
1-24 years
Phase:
PHASE3
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....
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 i...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
August 30 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 17 2025
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT06582524
Start Date
August 30 2024
End Date
June 17 2025
Last Update
November 28 2025
Active Locations (3)
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1
Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität
Graz, Austria, A-8036
2
Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria
Lisbon, Portugal
3
Bradford Royal Infirmary Duckworth Lane
Bradford, United Kingdom, BD9 6RJ