A Phase 3 Open-Label Study of Safety, Pharmacokinetics and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency
This study is exploring a new medicine named Pegzilarginase for babies and very young children (under 2 years old) who have a rare genetic condition called Arginase 1 Deficiency (ARG1-D). In ARG1-D, the body can't properly break down a substance called arginine, leading to harmful levels in the blood. The main goal is to find out if weekly injections of Pegzilarginase can help lower these high arginine levels after 12 weeks of treatment. Researchers will also be checking how safe the medicine is, looking for any side effects, and seeing how the body handles the medicine. They'll also observe if there are any improvements in the children's physical development.
At a glance
What is this study about?
This study is focused on a genetic condition called Arginase 1 Deficiency (ARG1-D), which affects babies and very young children. In simple terms, children with ARG1-D are missing an important enzyme (a type of protein) that helps their body get rid of a substance called arginine. When arginine builds up to high levels, it can be harmful to their health and development.
The new medicine being tested is called Pegzilarginase, which is given as an injection under the skin every week. The study's main aim is to see if this medicine can help lower the high levels of arginine in the blood of these young patients. They will specifically measure changes in arginine levels after 12 weeks of treatment.
Beyond just arginine levels, the doctors will carefully monitor the children's overall health throughout the study. This includes looking for any side effects, checking how the medicine moves through their body, and observing their growth and physical abilities. The hope is that Pegzilarginase could offer a new way to manage ARG1-D and improve the lives of affected children.
Key takeaways
- This study is for babies and very young children under 2 with Arginase 1 Deficiency (ARG1-D).
- It's testing a new weekly injection called Pegzilarginase to lower harmful arginine levels.
- The main focus is to check safety and how well the medicine works after 12 weeks.
- Doctors will closely monitor development, side effects, and how the body uses the medicine.
- Participation offers a chance to try a new treatment but also involves regular clinic visits and health checks.
Who may be eligible?
To join this study, the child must be under two years old and have been diagnosed with Arginase 1 Deficiency (ARG1-D).
Both boys and girls can take part. The research team will carefully check other specific medical requirements to make sure the study is safe and suitable for your child. These details will be discussed fully with you by the study doctor.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is my child under 2 years old?
- Has my child been diagnosed with Arginase 1 Deficiency (ARG1-D)?
- Is my child generally healthy enough to participate in a study?
- Am I able to commit to regular clinic visits with my child?
What does participation involve?
If your child takes part in this study, they will receive weekly injections of the study medicine, Pegzilarginase, under the skin. Over a period of 12 weeks, your child will have regular visits to the clinic. During these visits, doctors and nurses will:
* Take blood samples to check arginine levels, how the body handles the medicine, and for any signs of side effects. * Perform general health checks, including measuring their height, weight, and taking their blood pressure. * Do simple tests like an ECG (heart tracing) to check their heart. * Carefully watch for any changes in their physical development, using specific assessment tools for children.
The research team will explain the full schedule of visits and what each involves, ensuring you are comfortable every step of the way. The total length of your child's participation in the treatment part of the study will be at least 12 weeks, with follow-up appointments after that.
Potential risks and benefits
Locations (2)
- —UnverifiedPortugal
- —UnverifiedAustria
Common questions
What is Arginase 1 Deficiency (ARG1-D)?
It's a rare genetic condition where the body can't properly break down a substance called arginine, leading to harmful build-up in the blood.
What is Pegzilarginase?
It's the new medicine being tested in this study. It aims to help lower the high levels of arginine in children with ARG1-D.
How is the medicine given?
It's given as an injection just under the skin (subcutaneous injection) once a week.
What is the main goal of the study?
The main goal is to see if Pegzilarginase can safely reduce high arginine levels in the blood of very young children with ARG1-D after 12 weeks.
Can my child choose to leave the study at any time?
Yes, you can choose to withdraw your child from the study at any point, and it won't affect their regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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