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A study of tovorafenib for children and young adults with newly diagnosed or recurrent craniopharyngioma

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What is this trial about?

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

This study investigates the use of tovorafenib for the treatment of craniopharyngioma. This is a rare type of tumor that grows near the pituitary gland, which is a small area at the base of the brain that controls many important body functions. The study focuses on children and young adults who have either been newly diagnosed with this condition or are experiencing a recurrence, which means the tumor has returned after previous treatment.

The purpose of this study is to see how well the medication works in preventing the disease from getting worse and how it affects a person's daily well-being. Participants will take tovorafenib in the form of either a tablet or a powder for oral suspension, which is a liquid mixture made by dissolving the medicine in water. During the course of the study, the health and daily activities of the participants will be monitored over time.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Medication administration

    The treatment involves taking tovorafenib. this medication is available as either a tablet or a powder for oral suspension (a liquid made by mixing powder with water).

    The dose for this medication is 600 mg taken by mouth.

  2. Step 2

    Monitoring and evaluation

    Regular assessments are conducted to measure progression free survival, which tracks how long the craniopharyngioma (a type of tumor near the brain) remains stable without growing.

    The quality of life is monitored, focusing on physical function over a 12-month period.

    visual acuity, which is the clarity or sharpness of vision, is checked to see if there is improvement, stability, or worsening.

    visual field assessments are performed using a humphrey visual field test to check the entire area that can be seen while the eyes are fixed in one position.

    Medical checks are performed to monitor for changes in hormone levels or related conditions, such as hypothalamic obesity (weight gain caused by issues with the hypothalamus part of the brain), diabetes insipidus (a condition affecting how the body manages fluids), growth hormone deficiency (not enough growth hormone), or adrenal insufficiency (when the adrenal glands do not produce enough hormones).

Who can join the trial?

18 criteria

  • Participants must be between 1 and 39 years of age.
  • For those newly diagnosed, the diagnosis of craniopharyngioma (a type of tumor near the pituitary gland) must be confirmed by medical imaging or a doctor's review.
  • For those with a returning tumor, there must be a previous medical record confirming the original craniopharyngioma diagnosis.
  • Participants must be able to perform daily activities at a certain level, measured by the Karnofsky or Lansky scale, which are tools doctors use to rate a patient's ability to care for themselves. People who use a wheelchair but can sit up are included.
  • If taking dexamethasone (a type of steroid used to reduce swelling), the dose must have been stable or decreasing for at least one week and must not exceed a specific limit.
  • Participants must have healthy bone marrow, which is the part of the body that makes blood cells. This is checked by ensuring there are enough neutrophils (white blood cells that fight infection) and platelets (cells that help blood clot).
  • Participants must have healthy kidney function, checked by measuring creatinine (a waste product in the blood).
  • Participants must have healthy liver function, checked by measuring levels of bilirubin (a yellow substance in the blood), SGPT/ALT (an enzyme found in the liver), and albumin (a protein made by the liver).
  • Participants must have healthy lung function, meaning they should not have dyspnea (shortness of breath) while resting and should have sufficient oxygen levels in their blood.
  • Participants must have healthy neurologic function (brain and nerve health). People with seizures (uncontrolled electrical activity in the brain) may join if their condition is well controlled.
  • Blood clotting tests, such as PT/PTT/INR, must be within normal ranges.
  • Men and women of child-bearing age must agree to use effective contraception (methods to prevent pregnancy), such as barrier methods, because the study drug may make hormonal birth control ineffective.
  • A parent, guardian, or the patient must be willing and able to sign informed consent documents, which are forms that explain the study details.
  • Newly diagnosed patients must be candidates for biopsy (taking a small tissue sample) or resection (surgical removal of the tumor).
  • Patients with a returning tumor must have had enough time to recover from previous treatments, such as chemotherapy (drugs to kill cancer cells), radiation therapy (using high-energy beams to treat cancer), or monoclonal antibodies (specialized proteins used in treatment).
  • Participants with a returning tumor must be willing to provide archival tissue, which are stored samples of their previous tumor for testing.
  • Patients continuing maintenance therapy must have measurable disease, meaning the tumor is large enough to be accurately measured by doctors.
  • Participants must be able to complete the PedsQL Core Module, which is a questionnaire used to measure a child's quality of life.

Who cannot join the trial?

20 criteria

  • People who have recently been diagnosed and have not yet had any tumor-directed therapy, which is any medical treatment meant to target or shrink a tumor.
  • People who have previously received treatment for the RAS-pathway (a specific biological signaling process in cells) but have not taken tovorafenib.
  • People experiencing symptoms that are worsening very quickly and require urgent surgery or radiation therapy.
  • People with uncontrolled neuroendocrine dysfunction, which means hormone imbalances caused by issues with the glands that control body functions, such as diabetes insipidus (a condition affecting water balance), hypothyroidism (low thyroid hormone), or panhypopituitarism (deficiency of all hormones produced by the pituitary gland).
  • People with uncontrolled hormone issues who have not been on a stable dose of replacement medication for at least 2 weeks.
  • People with serious cardiovascular disease, meaning heart or blood vessel problems, or a history of myocardial infarction (a heart attack).
  • People who have had deep vein thrombosis (blood clots in a deep vein, usually in the leg) or a pulmonary embolism (a blood clot in the lung) within the last 6 months.
  • People with ongoing cardiomyopathy, which is a disease of the heart muscle that makes it harder to pump blood.
  • People with a prolonged QT interval, which is a measurement of the time it takes for the heart's electrical system to recharge between beats, specifically a QTcF (a specific way to measure this interval) greater than 440 ms.
  • People with a history of allergic reactions to medications that are chemically similar to tovorafenib or other drugs used in this study.
  • People experiencing moderate to severe nausea or vomiting (specifically Grade 2 or higher) or malabsorption, which is a condition where the body cannot properly absorb nutrients from food.
  • People who have had significant bowel or stomach surgery that would prevent the body from absorbing study medications correctly.
  • People with any uncontrolled illness, such as an active or ongoing infection.
  • People who are currently taking any other investigational agents, which are experimental drugs being tested in other studies.
  • Women who are pregnant or breast-feeding.
  • People taking strong CYP2C8 inhibitors or inducers, which are medications that speed up or slow down how the body processes certain drugs through a specific liver enzyme.
  • People who have received chemotherapy or radiotherapy within the last 3 weeks (or 6 weeks for specific drugs like nitrosoureas or mitomycin C).
  • People who have not yet recovered from acute adverse events (unexpected or harmful side effects) from treatments given more than 4 weeks ago.
  • People who cannot return for follow-up visits or medical tests needed to monitor toxicity, which refers to the harmful effects of a medication.
  • People who have not been off biologic or small molecule agents (types of targeted medications) for at least 1 week, or for a longer period if those drugs are known to cause side effects that last longer than a week.
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Investigated drugs

Tovorafenib is a medication taken by mouth that is being studied to see if it can help treat children and young adults who have been newly diagnosed with or have a recurrence of a type of brain tumor called craniopharyngioma.

What is already known about the treatment

Tovorafenib - This medication is taken by mouth in the form of either a tablet or a powder that is mixed with liquid. Currently being studied in clinical trials, it is designed to treat specific types of brain tumors like craniopharyngioma in children and young adults. It works by blocking specific proteins in cells that signal them to grow and divide uncontrollably. It is classified as a targeted therapy that focuses on stopping the growth of cancer cells.

Investigated diseases

Craniopharyngioma - This is a type of tumor that grows near the pituitary gland and the hypothalamus in the brain. As the tumor grows, it can press on nearby structures, such as the optic nerves, which may affect vision. It can also impact the hypothalamus, potentially leading to changes in weight, hormone levels, and fluid balance in the body. The tumor typically develops slowly over time. Its progression can cause various hormonal imbalances and changes in physical functions.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511510-20-00Estimated enrolment57 patientsSponsorPrincess Maxima Center For Pediatric Oncology

sourced from the EU Clinical Trials Register and site verification

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