Trial Outcomes & Findings for Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency (NCT NCT06582524)
NCT ID: NCT06582524
Last Updated: 2026-05-22
Results Overview
To evaluate the effect of pegzilarginase on plasma arginine concentrations in subjects \<24 months of age with arginase 1 deficiency (ARG1-D).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
3 participants
Primary outcome timeframe
From baseline up to 12 weeks.
Results posted on
2026-05-22
Participant Flow
Participants were enrolled at 3 different study sites between 30 August 2024 and 17 June 2025.
All 3 participants were included in the study.
Participant milestones
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
All participants received a once weekly (QW) SC dose of pegzilarginase for 12 weeks.
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
Baseline characteristics by cohort
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Age, Continuous
|
20.3 Months
STANDARD_DEVIATION 4.6 • n=2 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=2 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=2 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=2 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=2 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
|
Baseline plasma arginine levels
|
228.67 μM
STANDARD_DEVIATION 133.67 • n=2 Participants
|
PRIMARY outcome
Timeframe: From baseline up to 12 weeks.To evaluate the effect of pegzilarginase on plasma arginine concentrations in subjects \<24 months of age with arginase 1 deficiency (ARG1-D).
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Change From Baseline in Plasma Arginine Concentrations in Subjects <24 Months of Age With Arginase 1 Deficiency (ARG1-D).
|
-221.53 μM
Standard Deviation 81.26
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.PK parameters with evaluation of half-life (T½).
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacokinetic (PK) Profile of Pegzilarginase: Half-life (T½).
|
52.0 hours
Geometric Coefficient of Variation 0.836
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.PK parameters with evaluation on time to maximum observed concentration (Tmax).
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Tmax).
|
31.0 hours
Geometric Coefficient of Variation 5.69
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.PK parameters with evaluation of maximum observed concentration (Cmax).
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Cmax).
|
0.646 μg/mL
Geometric Coefficient of Variation 24.6
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.PK parameters with evaluation on area under the plasma drug concentration-time curve.
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacokinetic (PK) Profile of Pegzilarginase: Area Under the Plasma Drug Concentration-time Curve.
|
64.6 hours x µg/mL
Geometric Coefficient of Variation 23.4
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Samples taken on visit 1, 2, 4, 8 and 13 (pre-dose if on a dosing day).PD response evaluation, anti-drug antibodies (ADAs).
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacodynamic (PD) Response of Pegzilarginase: Anti-drug Antibodies (ADAs).
|
0 Participants
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks. Samples taken on visit 1, 2, 4, 8 and 13 (pre-dose if on a dosing day).PD response evaluation, levels of plasma arginine. Arginine within guidance level.
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Pharmacodynamic (PD) Response of Pegzilarginase: Levels of Plasma Arginine.
|
3 Participants
|
SECONDARY outcome
Timeframe: From baseline up to 12 weeks.Changes in physical function after 12 weeks of pegzilarginase treatment as measured by Gross Motor Function Measure (GMFM)-66 Parts A through E (total score). The Gross Motor Function Measure (GMFM) utilize a 4-point scoring system for each item across dimensions A-E. The minimum score is 0; the maximum score is 198, with a higher score representing better gross motor function.
Outcome measures
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 Participants
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Changes From Baseline in Physical Function: GMFM-66.
|
21.0 Points
Standard Deviation 13.9
|
Adverse Events
Weekly Subcutaneous (SC) Administration of Pegzilarginase
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Weekly Subcutaneous (SC) Administration of Pegzilarginase
n=3 participants at risk
All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Investigations
Coagulation time prolonged
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Nervous system disorders
Muscle spasticity
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Blood and lymphatic system disorders
Anemia
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
General disorders
Condition aggravated
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
General disorders
Injection site erythema
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
General disorders
Pyrexia
|
66.7%
2/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Infections and infestations
Febrile infection
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Infections and infestations
Respiratory syncytial virus infection
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Infections and infestations
Respiratory tract infection
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
|
Infections and infestations
Respiratory tract infection bacterial
|
33.3%
1/3 • From first dose until last safety follow up, week 20 (±7days).
|
Additional Information
VP Head of Global Integrated Evidence Generation
Immedica Pharma AB
Phone: 0046 8 533 39 500
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60