Active not recruitingOBSERVATIONAL

PASS of Paediatric Patients Initiating Selumetinib

This study, called a Post-authorisation Safety Study (PASS), is designed to check the safety of a medicine called selumetinib in children aged 3 to 17. These children have Neurofibromatosis Type 1 (NF1) and specific nerve tumours called plexiform neurofibromas that cause symptoms and can't be removed by surgery. Selumetinib has already been approved, and this study is a way to gather more information about its effects, especially potential long-term risks, as it's used in day-to-day healthcare across Europe and Israel. It aims to fill in gaps in our current understanding of the medicine's safety profile.

At a glance

Status

Active not recruiting

Sponsor

AstraZeneca

Enrolment target

124

Start

23 May 2022

Estimated completion

23 May 2028

Neurofibromatosis Type 1

What is this study about?

This study is about a condition called Neurofibromatosis Type 1, or NF1. NF1 is a rare genetic condition that can cause non-cancerous growths on nerves, known as plexiform neurofibromas. Sometimes these growths can cause problems and can't be removed with surgery. A medicine called selumetinib has been approved for use in children with NF1 who have these difficult-to-treat nerve tumours.

After a medicine is approved, regulators like the European Medicines Agency (EMA) sometimes ask for more information about its safety when it's used in everyday situations. This particular study is called a Post-authorisation Safety Study (PASS), and its main purpose is to carefully monitor the safety of selumetinib in children and young people (aged 3 to 17) who are taking it for their NF1-related nerve tumours. It's especially focused on understanding potential long-term effects.

The study will gather information from a large number of patients across different healthcare systems. This will help doctors and researchers get a clearer picture of any side effects or important safety information about selumetinib that might only become apparent with wider use, especially regarding children's growth and development. This helps ensure the medicine is as safe as possible for patients.

Key takeaways

This study focuses on the long-term safety of selumetinib in children with NF1.
It's a 'real-world' study, gathering data from routine hospital visits.
Participation helps improve understanding of the medicine for future patients.
There are no extra tests or treatments required for this study.
Consent from a parent or legal guardian is essential for participation.

Who may be eligible?

To be considered for this study, children and young people must have a diagnosis of Neurofibromatosis Type 1 (NF1) and have nerve tumours (plexiform neurofibromas) that are causing symptoms and cannot be surgically removed.

They should be aged between 3 and under 18 years old and have started selumetinib treatment up to six months before joining the study. A parent or legal guardian will need to give their informed consent for their child to participate.

Children will not be able to join if they have previously taken a similar type of medication (a 'mitogen-activated protein kinase inhibitor') or are already taking part in another clinical trial where a new treatment is being tested.

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.

Does your child have Neurofibromatosis Type 1 (NF1)?
Does your child have problematic nerve tumours that can't be removed by surgery?
Is your child currently taking selumetinib and started it within the last 6 months?
Is your child aged between 3 and under 18 years old?
Is your child NOT currently in another study testing a new medicine?

Answer every question to see your result.

What does participation involve?

This study is a 'non-interventional' study, which means you won't be given specific treatments or tests just for the study. Instead, researchers will collect information from your child's routine medical care. This involves looking at medical records to understand their health, how selumetinib is being used, and any effects it might have. There aren't any extra visits, tests, or medication specifically required for this study beyond what your child would normally receive as part of their treatment. The study aims to follow patients for a longer period to understand the long-term safety of selumetinib.

Potential risks and benefits

The main benefit of participating in this study is that it contributes to a better understanding of selumetinib's long-term safety in children with NF1, which can help future patients. There are no direct medical benefits to your child from taking part, as they will continue to receive their standard medical care. As this is an observational study, it does not involve any additional medical procedures or changes to your child's treatment plan beyond what their doctor recommends. Information collected will be anonymised where possible to protect privacy. You have the right to withdraw your child from the study at any time without affecting their medical care.

Locations (46)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Vienna, Austria
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Amiens, France
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Angers, France
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Bordeaux, France
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Lille, France
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Lyon, France
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Marseille, France
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Paris, France
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Rennes, France
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Strasbourg, France
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Toulouse, France
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Tours, France

Common questions

What is Neurofibromatosis Type 1 (NF1)?

NF1 is a rare genetic condition that causes non-cancerous tumours to grow on nerves, called plexiform neurofibromas.

What is selumetinib?

Selumetinib is a medicine approved to treat children with NF1 who have certain symptomatic, inoperable plexiform neurofibromas.

What does 'non-interventional' mean?

It means the study observes what happens during your child's routine medical care and doesn't involve any extra treatments, tests, or procedures specifically for the study.

Why is this study being done?

Even though selumetinib is approved, this study helps gather more real-world information about its safety, especially long-term effects on children, to ensure it's as safe as possible.

Will my child receive extra care by participating?

No, your child will continue to receive their usual medical care from their doctors. The study only collects information from this routine care.