AuthorisedTherapeutic exploratory (Phase II)Interventional

Trametinib in neurofibromatosis type 1 related symptomatic plexiform neurofibromas

This research study is investigating a medication called Mekinist. Mekinist comes as a tablet, and the study is for adults who have Neurofibromatosis Type 1 (NF1) and are experiencing bothersome or painful nerve tumours, called plexiform neurofibromas. The main goal is to find out if Mekinist can make these tumours smaller. Researchers will also look at how the treatment might affect pain levels, general well-being, and overall health. This is an important step to see if Mekinist could be a helpful treatment option for people living with NF1.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Enrolment target

Start

06 Sep 2024

Neurofibromatosis type 1

What is this study about?

This study is looking at a new way to treat a condition called Neurofibromatosis Type 1, often shortened to NF1. People with NF1 can develop various growths, including something called plexiform neurofibromas. These are non-cancerous tumours that grow along nerves and can sometimes cause pain or other problems.

The study is using a tablet medication called Mekinist (with the active ingredient trametinib). The main aim is to see if taking Mekinist can shrink these nerve tumours. Doctors will use special MRI scans to measure the size of the tumours before and during the study to see if they get smaller.

Apart from tumour size, the researchers also want to understand how this treatment might affect a person's daily life. They'll be looking at things like how much pain a person experiences, their overall quality of life, and if the treatment causes any side effects. This type of study (a Phase II trial) helps us learn more about how well a new treatment works and if it's safe for people.

Key takeaways

This study is testing a new tablet medicine (Mekinist) for adults with NF1.
It aims to see if Mekinist can shrink painful or problematic nerve tumours.
Researchers will also track improvements in pain and overall quality of life.
Participants will have regular MRI scans and health checks.
Joining is voluntary, and you can stop at any time.

Who may be eligible?

To join this study, you need to be an adult, aged 18 or older. There is no upper age limit, so older adults can also take part.

Both men and women are welcome to participate in this research. The study is specifically for individuals who have been diagnosed with Neurofibromatosis Type 1 (NF1).

If you have NF1 and are experiencing symptoms from plexiform neurofibromas, such as pain or other difficulties, this study might be for you. The research team will check other specific medical details to make sure the study is a safe and good fit for your individual health.

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)?
Are you experiencing symptoms from plexiform neurofibromas (nerve tumours)?
Are you able to take tablets as prescribed?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be asked to take the Mekinist tablets as prescribed. You would have regular appointments for follow-up. These appointments would likely include MRI scans to measure your tumours, as well as discussions about any pain you might be experiencing and your general well-being. You would also fill out questionnaires about how you are feeling and have photos taken of certain areas. Doctors would carefully check for any side effects you might have. The total duration of your participation would depend on how the treatment works for you and the study's design.

Potential risks and benefits

Taking part in this study could offer some potential benefits, such as receiving a new investigational treatment that might shrink your tumours and reduce symptoms like pain. However, there's no guarantee the treatment will work for everyone, and it might not improve your condition. All medications have potential risks and side effects, which the study team will explain in detail. You will be closely monitored for any adverse effects. Remember, joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Netherlands

Common questions

What is Neurofibromatosis Type 1 (NF1)?

NF1 is a genetic condition that causes tumours to grow on nerves, usually non-cancerous, which can affect many parts of the body.

What are plexiform neurofibromas?

These are specific types of nerve tumours that can occur in people with NF1 and can sometimes cause pain or other problems.

What is Mekinist?

Mekinist is a tablet medication being studied to see if it can help shrink plexiform neurofibromas in people with NF1.

Will I get paid to be in this study?

Clinical trials usually don't pay you to participate, but often cover travel costs or other study-related expenses. You should ask the research team about this.

How long will the study last?

The exact duration will vary for each person, depending on their response to treatment and the study plan. The study team can give you more details.