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Study to investigate the safety and activity of Pegzilarginase in children under two years of age with Arginase 1 Deficiency
EU CTIS ID: 2024-510797-25-00
What this study is testing
To evaluate the effect of pegzilarginase on plasma arginine concentrations in subjects < 24 months of age with arginase 1 deficiency (ARG1-D)
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join 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: a. elevated plasma arginine levels; b. a mutation analysis revealing a pathogenic variant; c. red blood cell (RBC) arginase activity
- 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: a. attempt to maintain a stable, age-appropriate level of protein consumption, including natural protein, and EAA supplementation within approximately ± 15 % of dietitian recommended diet; b. attempt to maintain current use of ammonia scavengers, if prescribed
- Subjects must weigh > 8 kg due to clinical trial related blood collection volumes required
You likely can't join 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
See the full eligibility criteria
- 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: a. elevated plasma arginine levels; b. a mutation analysis revealing a pathogenic variant; c. red blood cell (RBC) arginase activity
- 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: a. attempt to maintain a stable, age-appropriate level of protein consumption, including natural protein, and EAA supplementation within approximately ± 15 % of dietitian recommended diet; b. attempt to maintain current use of ammonia scavengers, if prescribed
- Subjects must weigh > 8 kg due to clinical trial related blood collection volumes required
- 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
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.