New hope for babies with rare enzyme disorder: weekly shot shows promise
NCT ID NCT06582524
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a weekly injection of pegzilarginase (Loargys) in 3 infants under 24 months old with arginase 1 deficiency, a rare genetic disorder that causes harmful buildup of arginine. The goal was to see if the drug is safe and can lower arginine levels in the blood. The treatment was given for 12 weeks, followed by 8 weeks of monitoring.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
pegzilarginase (Loargys)
What this could lead to
If successful, this could provide a treatment option for infants with arginase 1 deficiency to help control their condition from an early age.
What could go wrong
This is a very small study (only 3 participants) and results may not apply to all patients. The treatment requires ongoing weekly injections and long-term safety is not yet established.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Bradford Royal Infirmary Duckworth Lane
Bradford, BD9 6RJ, United Kingdom
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Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria
Lisbon, Portugal
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Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität
Graz, A-8036, Austria