Phase 3
N=3
Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
Arginase 1 Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT06582524 ↗Enrolled (actual)
3
Serious AEs
0.0%
Results posted
Apr 2026
Primary outcome: Primary: Change From Baseline in Plasma Arginine Concentrations in Subjects <24 Months of Age With Arginase 1 Deficiency (ARG1-D). — -221.53 μM
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Pegzilarginase (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Immedica Pharma AB
- Primary completion
- Jun 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Plasma Arginine Concentrations in Subjects <24 Months of Age With Arginase 1 Deficiency (ARG1-D). |
-221.53 | — |
| SECONDARY Pharmacokinetic (PK) Profile of Pegzilarginase: Half-life (T½). |
52.0 | — |
| SECONDARY Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Tmax). |
31.0 | — |
| SECONDARY Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Cmax). |
0.646 | — |
| SECONDARY Pharmacokinetic (PK) Profile of Pegzilarginase: Area Under the Plasma Drug Concentration-time Curve. |
64.6 | — |
| SECONDARY Pharmacodynamic (PD) Response of Pegzilarginase: Anti-drug Antibodies (ADAs). |
— | — |
| SECONDARY Pharmacodynamic (PD) Response of Pegzilarginase: Levels of Plasma Arginine. |
3 | — |
| SECONDARY Changes From Baseline in Physical Function: GMFM-66. |
21.0 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Subjects must be 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT06582524). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.