NF1-Plexiform Neurofibroma (NF1-PN) Trial (ReNeu)

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We are conducting a Phase 2b clinical trial designed to evaluate the safety, effectiveness, and tolerability of mirdametinib in children 2 years or older and adults with an inoperable NF1-PN.

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a clinical trial

Clinical trials are essential research practices for advancing science, enhancing medical knowledge, and improving patient care. They are carefully designed to maximize safety for patients and are closely monitored at every step, but there are risks as we explore new science.

Scientists make the discoveries, but clinical trials can’t happen without patients. If you are considering joining a trial, we thank you. And we want to make sure you completely understand your role, your rights and what is expected of you.

Before volunteering for a clinical trial, you should talk to your doctor and read about the clinical trial process to ensure you understand the risks – and your rights. You will be asked to sign an Informed Consent form before joining the trial. Learn more by exploring our brochure, Understanding Clinical Trials.

What is the ReNeu trial?

The ReNeu trial is a Phase 2b clinical trial designed to evaluate the safety, effectiveness, and tolerability of an investigational medicine called mirdametinib in children two years or older and adults with an inoperable NF1-PN. The ReNeu trial is an open-label study, meaning that both study participants and doctors are aware of the study treatment being given.

How often do you need to be seen as part of the trial?

Following an initial evaluation, participants will begin taking the study medication, which is available in a oral capsule or a pediatric formulation for children who cannot swallow a capsule. Participants will be asked to return to the clinic for study visits on a monthly basis for the first seven months and then every other month until the end of the study, which is two years after the first dose of study treatment.

How long does the study lasts?

Participation in the ReNeu study will last approximately two years.

What will happen during the study?

During participation in the study, participants can expect the following:

• Complete the Screening assessments to confirm they can participate
• Take the study drug by mouth in four week cycles (twice per day for three weeks, then stop for one week and repeat)
• Record each dose of the study drug in an electronic diary called an ePRO device
• Attend study visits and complete health assessments once per month for seven months, then every other month
• Participate in a monthly wellness check by phone or email (this check may be completed during study visits)
• Complete a follow-up visit 30 days after the last dose of the study drug

Participants may also have the option to continue taking the study drug for additional cycles as part of a Long-term Follow-up Phase.

How do I get involved in the ReNeu trial?

  • The ReNeu trial is fully enrolled and is not accepting new patients.
  • For medical professionals seeking additional information about the ReNeu trial, please email
  • The ReNeu trial is being conducted at approximately 50 sites in the United States. Details of the trial locations can be found here.