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Study of tovorafenib versus standard chemotherapy as first treatment for children with low-grade glioma with RAF gene changes

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What is this trial about?

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

This study focuses on pediatric low-grade glioma, a type of brain tumor that occurs in children and young people under 25 years of age. The study specifically looks at cases where the tumor has certain genetic changes called RAF alterations and requires treatment for the first time. The purpose is to compare two different treatment approaches: a new medication called tovorafenib versus standard chemotherapy treatments that include vincristine, vinblastine, and carboplatin.

The study will determine which treatment is more effective at shrinking or controlling the tumor. Patients will be randomly assigned to receive either tovorafenib tablets or oral suspension taken by mouth, or standard chemotherapy medications given through an intravenous line (a small tube inserted into a vein). The study will measure how well tumors respond to treatment and how long patients remain free from tumor growth.

Doctors will regularly monitor patients using brain scans and medical examinations to track the tumor's response to treatment. The study will continue for several years to gather long-term information about how well each treatment works and how it affects patients' health over time. This research aims to help determine the most effective treatment option for children and young people with this specific type of brain tumor.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial evaluation and assignment

    Your eligibility for the trial will be confirmed based on being under 25 years of age and having a low-grade glioma (a type of brain tumor) with specific genetic characteristics.

    You will need to have at least one tumor that can be measured.

    You will be randomly assigned to receive either the new medication or standard treatment.

  2. Step 2

    Treatment path A - New medication

    If assigned to this group, you will receive tovorafenib.

    This medication will be taken by mouth, either as tablets or as a powder mixed with liquid.

  3. Step 3

    Treatment path B - Standard treatment

    If assigned to this group, you will receive standard chemotherapy medications:

    Vincristine, vinblastine, and carboplatin will be given through an IV line.

    These medications will be administered at regular intervals according to the standard treatment schedule.

  4. Step 3

    Monitoring during treatment

    Regular imaging scans will be performed to measure how your tumor responds to treatment.

    Your response to treatment will be evaluated using specific criteria that look for any changes in tumor size.

    Your overall health status will be monitored throughout the study.

  5. Step 4

    Long-term follow-up

    The study will continue until March 2030.

    You will be monitored for any changes in your condition.

    The medical team will track how long the treatment remains effective and your overall health status.

Who can join the trial?

6 criteria

  • Must be under 25 years old and have a low-grade glioma (a slow-growing brain tumor) with specific genetic changes in the RAF gene
  • Must have a confirmed diagnosis of glioma or glioneuronal tumor (types of brain tumors) through tissue examination
  • Must have at least one tumor that can be measured by imaging tests
  • Must be starting treatment for the first time with medication that goes throughout the body (systemic therapy)
  • Both males and females can participate in the study
  • Children and young people from birth to 24 years old are eligible

Who cannot join the trial?

12 criteria

  • Patients who do not have a confirmed diagnosis of pediatric low-grade glioma (a type of slow-growing brain tumor)
  • Patients whose tumor does not have an activating RAF alteration (a specific genetic change in the tumor)
  • Patients who have already received systemic therapy (medicine that travels through the bloodstream)
  • Patients younger than 6 months or older than 25 years of age
  • Patients who have received previous treatment with tovorafenib or similar medications
  • Patients with severe heart, liver, or kidney problems
  • Patients who are currently participating in other clinical trials
  • Patients who are pregnant or breastfeeding
  • Patients who cannot undergo required medical tests or procedures
  • Patients with known allergies to the study medications
  • Patients who have had major surgery within 4 weeks before starting the study
  • Patients who cannot swallow oral medications
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Investigated drugs

  • DAY101 (tovorafenib)

    This medication is designed to target specific genetic changes (RAF alterations) in pediatric low-grade brain tumors called gliomas. It is taken as a monotherapy, meaning it is used alone without other cancer medications. It works by blocking certain proteins that contribute to tumor growth.

  • Standard of care chemotherapy

    This refers to the currently accepted and commonly used chemotherapy treatments for pediatric low-grade gliomas. These are established cancer-fighting medications that have been proven effective and are considered the usual treatment approach for this type of brain tumor.

What is already known about the treatment

Tovorafenib – A novel targeted therapy medication administered orally that specifically inhibits RAF proteins in cancer cells. This investigational drug is currently being studied in clinical trials as a first-line treatment for pediatric low-grade gliomas (brain tumors) that have RAF gene alterations. The medication works by blocking specific molecular pathways involved in tumor growth, particularly targeting the RAF signaling pathway that is abnormally activated in these types of brain tumors. Tovorafenib belongs to the class of kinase inhibitors and represents a potential alternative to standard chemotherapy, with ongoing Phase 3 clinical trials evaluating its effectiveness compared to conventional treatment methods.

Investigated diseases

Pediatric Low-Grade Glioma - A slow-growing brain tumor that typically develops in children and young adults, primarily affecting the brain or spinal cord tissue. It forms from glial cells, which are supportive cells in the central nervous system. When caused by RAF gene alterations, these tumors show abnormal cell growth patterns. The tumor can develop in various parts of the brain or spinal cord, leading to different symptoms depending on its location. These tumors tend to grow slowly over time, and their behavior can vary significantly between patients.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-510742-13-00Protocol codeDAY101-002Estimated enrolment400 patientsSponsorDay One Biopharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).