Active not recruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

This research study is looking at a new medicine called selumetinib for adults living with Neurofibromatosis Type 1 (NF1). NF1 can cause growths called plexiform neurofibromas (PNs). This study is for people whose PNs are causing symptoms like pain and can't be removed with surgery. We want to find out if selumetinib is effective in making these PNs smaller and reducing symptoms, and if it's safe to use. Some participants will receive selumetinib and others will receive a placebo (a dummy drug with no active medicine) to compare their effects. This is a worldwide study involving many people, and it aims to provide clear answers about whether this medicine could be a good treatment option for patients with NF1 and these specific growths.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

AstraZeneca

Enrolment target

145

Start

19 Nov 2021

Estimated completion

15 Feb 2029

Neurofibromatosis 1 Plexiform Neurofibroma (PN)

What is this study about?

This study is about a medical condition called Neurofibromatosis Type 1, often shortened to NF1. People with NF1 can develop different types of growths. This research focuses on specific growths known as plexiform neurofibromas (PNs). For some people, these PNs can grow large, cause pain, and affect daily life, and surgery might not be an option to remove them.

The main goal of this study is to test a new medication called selumetinib. We want to see if this medicine can help adults with NF1 whose PNs are causing noticeable problems and can't be surgically removed. The study will look at whether selumetinib can make these PNs smaller and reduce the symptoms they cause. It will also carefully check for any side effects to make sure the medicine is safe.

The study compares selumetinib to a 'placebo', which looks like the real medicine but contains no active drug. This helps researchers understand the true effect of selumetinib. By taking part, you would be helping doctors and scientists learn more about NF1 and potentially find new ways to treat it, improving the lives of many people with this condition.

Key takeaways

This study is for adults with NF1 who have painful, non-removable plexiform neurofibromas.
It tests a new medicine called selumetinib against a placebo (dummy pill).
The main goals are to see if the medicine shrinks growths and reduces symptoms like pain.
Your health would be closely monitored with regular check-ups and scans.
Participation is voluntary, and you can withdraw at any time.
This research helps improve understanding and treatment of NF1.

Who may be eligible?

To be considered for this study, you need to be an adult, aged 18 or older, and have a confirmed diagnosis of Neurofibromatosis Type 1 (NF1). A key part of qualifying is having plexiform neurofibromas (PNs) that are causing symptoms, such as pain, and can't be removed through surgery. We'll also need to be able to measure at least one of these PNs using special scans.

There are also some health requirements. Your body's organs, like your heart, liver, and kidneys, need to be working well, along with your bone marrow. We would also check if you have had stable pain medication for your PNs and are generally healthy enough to take part in a study like this.

However, some health conditions would mean you couldn't join. For example, if you have certain types of cancer (unless it was completely treated a long time ago), serious heart problems, or certain eye conditions. If you've previously taken similar types of medication called MEK inhibitors, you also wouldn't be able to participate.

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.

Are you 18 years old or older?
Have you been diagnosed with Neurofibromatosis Type 1 (NF1)?
Do you have plexiform neurofibromas (PNs) that cause symptoms and can't be removed by surgery?
Are your heart, liver, kidneys, and bone marrow generally healthy?
Do you have stable pain medication use for your PNs?
Have you NOT had certain serious medical conditions, like some cancers or severe heart issues?

Answer every question to see your result.

What does participation involve?

If you join this study, you would be randomly assigned to either receive the study medicine, selumetinib, or a placebo (a dummy pill). Neither you nor your study doctor would know which you are receiving, which helps us get accurate results. You would take this medicine or placebo regularly.

Throughout the study, you would have regular visits to the clinic. These visits would involve medical check-ups, blood tests, and imaging scans (like MRI) to see how your plexiform neurofibromas are responding. We would also closely monitor any symptoms you're experiencing, especially pain, and any side effects. The total duration of your participation in the study would involve taking the medication and attending these follow-up appointments.

Potential risks and benefits

Taking part in a study like this has potential benefits and potential risks. You might benefit if selumetinib helps to shrink your plexiform neurofibromas or reduces your symptoms, like pain. However, there's also a chance you could receive the placebo and not get the active medicine, or you might experience side effects from selumetinib. We would carefully monitor you for any side effects, and you would be fully informed about these before you decide to join. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (34)

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

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Gainesville, United States
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Rockville, United States
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St Louis, United States
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Commack, United States
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Melbourne, Australia
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St Leonards, Australia
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Porto Alegre, Brazil
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Ribeirão Preto, Brazil
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São Paulo, Brazil
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Toronto, Canada
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Montreal, Canada
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Beijing, China

Common questions

What is Neurofibromatosis Type 1 (NF1)?

NF1 is a genetic condition that causes growths (tumours) to form on nerve tissue. These growths are usually not cancerous but can cause various issues.

What are plexiform neurofibromas (PNs)?

PNs are a specific type of growth that can happen in people with NF1. They can sometimes be large, grow along nerves, and cause pain or other problems.

What is a placebo?

A placebo is a 'dummy' treatment that looks just like the study medicine but doesn't contain any active drug. It helps ensure we can fairly compare the real medicine's effects.

Will I know if I'm getting the real medicine or the placebo?

No, during the main part of the study, neither you nor your doctor will know whether you are receiving selumetinib or the placebo. This is called 'double-blind' and makes the study results more reliable.

Can I stop participating in the study if I want to?

Yes, absolutely. You have the right to withdraw from the study at any time, for any reason, and this will not affect your ongoing medical care.