Arginase deficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:14:59 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Arginase deficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Title: Safety and effectiveness study of pegzilarginase weekly injections in children under 2 years old with Arginase 1 Deficiency https://clinicaltrials.eu/trial/study-on-the-safety-and-activity-of-pegzilarginase-for-children-under-2-years-with-arginase-1-deficiency/ Wed, 29 Apr 2026 15:03:42 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-activity-of-pegzilarginase-for-children-under-2-years-with-arginase-1-deficiency/ This study focuses on children under two years of age who have Arginase 1 Deficiency (ARG1-D), a rare genetic condition where the body cannot properly break down an amino acid called arginine. The study will test a medication called pegzilarginase, which is given as an injection under the skin once per week. This medicine is designed to help reduce the high levels of arginine in the blood that occur in people with this condition.

The purpose of this research is to evaluate how well pegzilarginase works in lowering arginine levels in the blood of young children with Arginase 1 Deficiency. The study will also look at how safe the medication is and how the body processes it. The treatment will continue for 12 weeks, during which children will receive weekly injections of pegzilarginase.

Throughout the study, doctors will monitor the children’s health through regular check-ups and blood tests. They will track various aspects of the children’s development, including their physical abilities and growth. The study will also check how the children’s bodies respond to the medication and whether they develop any reactions to the treatment.

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