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Study of tovorafenib (DAY101) in children and young adults aged 6 months to 25 years with brain tumors and other solid tumors that have 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 children and young adults with low-grade glioma (a type of brain tumor) and other advanced solid tumors that have specific genetic changes called RAF alterations. The study will test a new medication called tovorafenib (also known as DAY101), which is designed to target these specific genetic changes in tumors. This medicine comes in two forms - as a powder that can be made into a liquid for drinking, and as tablets.

The purpose of this research is to determine how well tovorafenib works in treating these types of tumors and to understand its safety in patients aged 6 months to 25 years. The medication is specifically designed to reach the central nervous system (brain and spinal cord) and works by blocking certain proteins that can cause tumor growth.

During the study, participants will receive tovorafenib daily by mouth. The treatment may continue for up to 72 months, depending on how well it works for each patient. Doctors will regularly check the size of tumors using imaging tests and monitor patients for any side effects that may occur during treatment.

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

    Your eligibility for the study will be assessed based on your age (between 6 months and 25 years) and medical condition.

    A confirmation of your brain tumor (low-grade glioma) or advanced solid tumor diagnosis is required.

    Your previous treatments and disease progression will be reviewed.

    Your physical condition will be evaluated using a performance score test.

  2. Step 2

    Treatment assignment

    You will be assigned to one of three treatment groups:

    Group 1: For patients with low-grade glioma with specific genetic changes

    Group 2: For patients with low-grade glioma with certain genetic alterations

    Group 3: For patients with advanced solid tumors with specific genetic changes

  3. Step 3

    Medication administration

    You will receive tovorafenib (also known as DAY101) in one of two forms:

    Either as a powder that will be mixed into a liquid for drinking

    Or as tablets that you will take by mouth

    Your doctor will provide specific instructions about how and when to take the medication

  4. Step 4

    Monitoring period

    Regular check-ups will track how your body responds to the treatment

    Your tumor size will be measured regularly using imaging scans

    Blood tests will be performed to monitor how your body processes the medication

    Any side effects or health changes will be recorded

  5. Step 5

    Study completion

    The study will continue until April 2027

    Your participation may end earlier based on how your condition responds to the treatment

    A final evaluation will be performed to assess the overall results of your treatment

Who can join the trial?

9 criteria

  • Patient must be between 6 months and 25 years old
  • Patient must have one of these conditions:
    • Returning or worsening low-grade glioma (a slow-growing brain tumor) with a confirmed BRAF alteration (a specific genetic change)
    • Returning or worsening low-grade glioma with a confirmed or expected BRAF mutation or RAF fusion (specific types of genetic changes)
    • Advanced or spreading solid tumor with a confirmed or expected RAF fusion
    • Patient must have had their cancer confirmed through tissue examination (biopsy) either when first diagnosed or when the cancer returned
    • Patient must have received at least one previous treatment and show evidence that the cancer has grown on imaging scans (for low-grade glioma patients only)
    • Patient must have at least one tumor that can be measured or evaluated by medical imaging
    • Patient must have a performance score of at least 50 on either:
      • The Karnofsky scale (for patients 16 years and older) - a scale that measures a person's ability to perform daily activities
      • The Lansky scale (for patients under 16 years) - a similar scale used for children

Who cannot join the trial?

11 criteria

  • Age below 6 months or above 25 years
  • Absence of RAF gene alterations (changes in specific genes that affect cell growth)
  • No evidence of tumor progression or recurrence
  • Previous treatment with tovorafenib (the study medication)
  • Patients who have not experienced a relapse or progression of their low-grade glioma (slow-growing brain tumor) or advanced solid tumor
  • Patients who cannot undergo required medical imaging to monitor their condition
  • Inability to take oral medications
  • Presence of other serious medical conditions that could interfere with study participation
  • Current participation in other clinical trials
  • Pregnancy or breastfeeding
  • Known allergies or hypersensitivity to the study medication or its components
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Investigated drugs

Tovorafenib (also known as DAY101) is an oral medication that works as a pan-RAF inhibitor. It targets and blocks specific proteins called RAF that are involved in cancer cell growth. This medication is designed to treat pediatric patients who have certain types of brain tumors called low-grade gliomas and other advanced solid tumors where the RAF genes have been altered. The medication works by interfering with abnormal cell signaling that causes tumor growth, particularly in cases where there are BRAF alterations or RAF fusions.

What is already known about the treatment

Tovorafenib (DAY101) – An oral pan-RAF inhibitor medication specifically designed for pediatric patients with RAF-altered tumors. This drug is administered orally and is currently being investigated in clinical trials for treating recurrent or progressive low-grade glioma and advanced solid tumors in patients aged 6 months to 25 years. The medication works by targeting and inhibiting RAF proteins, which are involved in cell growth signaling pathways that can become abnormally activated in certain cancers. As a targeted therapy in the kinase inhibitor class, tovorafenib represents a promising treatment approach for pediatric cancers with specific genetic alterations in the RAF pathway.

Investigated diseases

  • Low-Grade Glioma

    A type of brain tumor that develops from glial cells and grows slowly in the brain or spinal cord. It begins in supportive brain tissue and typically has a less aggressive growth pattern compared to high-grade variants. These tumors can affect various parts of the brain, leading to different symptoms depending on their location. Low-grade gliomas can gradually increase in size over time, potentially causing pressure on surrounding brain structures.

  • Advanced Solid Tumor

    A solid mass of abnormal cells that can occur in various body tissues and organs, which has spread beyond its original location. These tumors form distinct masses or lumps that can be detected in tissue. Solid tumors can develop in different parts of the body, including organs, muscles, or bones. They are characterized by the growth of abnormal cells that multiply uncontrollably and can expand into nearby tissues.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-510691-20-00Protocol codeDAY101-001Estimated enrolment140 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).