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A study of mirdametinib for infants and toddlers under 24 months of age with neurofibromatosis type 1 associated plexiform neurofibromas

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves infants and toddlers under 24 months of age who have Neurofibromatosis Type 1 associated with Plexiform Neurofibromas. Neurofibromatosis Type 1 is a genetic condition that causes tumors to grow along nerves in the body. Plexiform Neurofibromas are a type of tumor that can grow in multiple areas and may cause problems depending on where they are located, such as near important blood vessels, airways, or nerves. These tumors can cause symptoms like pain, loss of function, or visible changes in appearance, or they may not cause symptoms but are in locations where they could lead to serious problems. The treatment being tested in this study is Mirdametinib, also known by its code name PD-0325901, which is given as a dispersible tablet taken by mouth.

The purpose of this study is to evaluate the safety, how well the body handles the medication, and how the medication works in very young children with these tumors. The study will look at whether the medication causes any side effects, changes in laboratory test results, vital signs, heart function measured by tests called electrocardiograms and echocardiogram, and eye examinations. The study will also measure the amount of medication in the blood to understand how it is processed by the body. Additionally, the study will examine whether the tumors shrink by at least 20 percent compared to their size at the start, using imaging scans analyzed by independent reviewers.

During the study, children will receive Mirdametinib for up to 36 months, and they will be monitored regularly through physical examinations, blood tests, imaging scans using MRI, and assessments of quality of life and pain levels using age-appropriate scales. The study will also track how long any tumor shrinkage lasts and whether there are improvements in the child's comfort and daily functioning. The medication dose can be up to 8 milligrams per day, with a maximum total dose of 288 milligrams during the treatment period.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment period

    Your child will receive mirdametinib as a dispersible tablet that is taken by mouth.

    The study doctor will determine the appropriate dose based on your child's age and weight.

    Your child will take the medication daily throughout the study period.

    The treatment is expected to continue for an extended period as the study runs until February 2032.

  2. Step 2

    Safety monitoring visits

    Your child will undergo regular safety assessments throughout the study.

    These assessments will include blood tests to check laboratory parameters.

    The study doctor will measure your child's vital signs, including blood pressure, heart rate, and temperature.

    Your child will have regular physical examinations to monitor their overall health.

  3. Step 3

    Heart monitoring

    Your child will have electrocardiograms, which are tests that record the electrical activity of the heart.

    Your child will also have echocardiograms, which are ultrasound examinations of the heart to check how it is functioning.

  4. Step 4

    Eye examinations

    Your child will have regular eye examinations performed by an eye specialist to monitor eye health.

  5. Step 5

    Tumor imaging assessments

    Your child will have magnetic resonance imaging scans, which are detailed imaging tests that allow doctors to see the size of the tumor.

    These scans will be performed at regular intervals to measure changes in the size of the plexiform neurofibroma, which is the type of tumor being treated.

    The scans will be reviewed by independent doctors who do not know which treatment your child is receiving to ensure objective assessment.

    Scans showing a decrease of 20% or more in tumor size compared to the start of the study, confirmed on consecutive scans taken 2 to 6 months apart, will be considered a positive response.

  6. Step 6

    Quality of life assessments

    Your child's quality of life will be assessed using the Pediatric Quality of Life Inventory Infant Scales, which is a questionnaire designed for infants and toddlers.

    These assessments will help the study doctor understand how your child is feeling and functioning during the study.

  7. Step 7

    Pain assessments

    Your child's pain level will be assessed using an age-appropriate pain scale called faces, legs, activity, cry, consolability.

    This scale helps the study doctor evaluate whether your child is experiencing any discomfort or pain.

  8. Step 8

    Blood sample collection for medication level testing

    Blood samples will be collected from your child at specific times to measure the levels of mirdametinib in the blood.

    This testing, called pharmacokinetics, helps the study doctor understand how the body processes the medication.

    The measurements will include the lowest level of medication in the blood, the highest level, and the total amount of medication in the blood over time.

  9. Step 9

    Ongoing monitoring

    Throughout the study, any side effects or health changes your child experiences will be recorded and graded according to a standard system used in cancer research.

    The study doctor will continue to monitor your child's response to treatment and safety throughout the entire study period.

Who can join the trial?

5 criteria

  • The child must be between 0 and 21 months old at the time of receiving the first dose of the study medicine. If the child was born early, the age will be calculated from the expected delivery date, not from the actual birth date.
  • The child must have been diagnosed with Neurofibromatosis Type 1, which is a genetic condition that causes tumors to grow along nerves. The diagnosis must follow specific medical guidelines from 2021 and include the presence of a plexiform neurofibroma (a type of nerve tumor) plus at least one other sign of this condition.
  • The child must have a target plexiform neurofibroma, which is the most important tumor that doctors will focus on during the study. This tumor must be visible on at least 3 slices of an MRI scan (a special imaging test that takes detailed pictures inside the body), and the entire tumor must fit within the scan image. The tumor must be at least 3 milliliters in size, which can be measured using a special method.
  • The child must have one of the following situations with their tumor: either the tumor is in a dangerous location where it could cause serious health problems even if it is not currently causing symptoms, or the tumor is already causing symptoms. Dangerous locations include the head or neck area (except scalp only), near major nerve bundles in the shoulder or lower back area, or next to important body structures such as major blood vessels, airways, organs like the heart or lungs, or the spinal cord. Symptoms that the tumor might cause include breathing difficulties, nerve compression, loss of body function, major changes in body shape, painful areas, or problems with arm or leg function.
  • Both male and female children can participate in this study.

Who cannot join the trial?

3 criteria

  • The source data does not provide specific exclusion criteria for this clinical trial
  • Exclusion criteria are reasons why a patient cannot participate in a study, but these details are not available in the provided information
  • To learn about specific reasons that would prevent participation in this study, you would need to review the complete study documentation
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Investigated drugs

Mirdametinib is an investigational medication being studied for the treatment of plexiform neurofibromas in infants and toddlers with neurofibromatosis type 1. Plexiform neurofibromas are non-cancerous tumors that grow along nerves. This medication is being tested to see if it is safe and well-tolerated in very young children, and to understand how the body processes the drug. The study aims to help children who have symptoms from these tumors or who have tumors in locations that could cause serious health problems.

What is already known about the treatment

Mirdametinib – Mirdametinib is an experimental medication currently being studied in clinical trials for the treatment of plexiform neurofibromas, which are nerve tumors that occur in patients with Neurofibromatosis Type 1, a genetic condition. This drug works by blocking specific proteins called MEK enzymes that help cancer and tumor cells grow and multiply, thereby potentially slowing down or stopping the growth of these tumors. The medication is being tested specifically in infants and toddlers under 2 years of age to evaluate how safe it is, how well the body processes it, and whether it can help manage these tumors that can cause serious health problems depending on where they grow in the body.

Investigated diseases

Neurofibromatosis Type 1 – Neurofibromatosis Type 1 is a genetic disorder that affects the nervous system and causes tumors to form on nerve tissue throughout the body. The condition is present from birth and is caused by changes in a specific gene that normally helps control cell growth. People with this disorder develop various types of tumors, including plexiform neurofibromas, which are growths that involve multiple nerves and can appear anywhere in the body. These tumors grow along the length of nerves and can affect surrounding tissues, potentially causing pain, disfigurement, or functional problems depending on their location. Plexiform neurofibromas may be present at birth or develop during early childhood, and they tend to grow larger over time, particularly during periods of rapid body growth. The condition can affect infants and toddlers, with some experiencing symptoms while others may have tumors in high-risk locations that could lead to complications as they develop.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase ITrial ID2025-522000-24-00Protocol codeMEK-NF1-104Estimated enrolment8 patientsSponsorSpringworks Therapeutics Inc.

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