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A study of ulixertinib, tovorafenib, and vinblastine sulfate for children with progressive, relapsed, or refractory low-grade glioma

Verified siteRegistered drugNo placebo
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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 with pediatric low-grade glioma, which is a type of slow-growing brain tumor. The research specifically looks at cases where the tumor has continued to grow despite treatment, has returned after a period of stability, or has not responded to standard medical therapies. The purpose of this study is to find the most effective way to use specific medications, either alone or in combination, to treat this condition.

The treatments being investigated include vinblastine sulfate, which is given as an intravenous injection, and two oral medications called ulixertinib and tovorafenib. Participants may receive different combinations of these drugs depending on the specific part of the study they are assigned to. This approach allows for the testing of various treatment plans to see which ones work best and are safest.

During the study, the amount of medication provided may be adjusted to find the highest dose that can be taken safely. Regular monitoring occurs throughout the treatment period to observe how the body responds to the drugs and to check for any side effects. The study will evaluate how well the medications control the tumor over time.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>treatment assignment</b>

    After joining the study, a specific treatment regimen is assigned. this regimen may involve a single medication or a combination of different medications to address the pediatric low-grade glioma, which is a type of brain tumor.

  2. Step 2

    <b>medication administration</b>

    The treatment may include the administration of vinblastine sulfate, which is given as an injection directly into a vein. other medications, such as ulixertinib or tovorafenib, are taken as tablets by mouth.

    The dose of these medications may be adjusted over time. this dose escalation process is used to find the most effective amount that can be taken safely.

  3. Step 3

    <b>monitoring and assessment</b>

    The response to the treatment is assessed every 8 weeks. this evaluation continues for a maximum of 12 cycles of treatment.

    Assessments are conducted to determine the best response, which refers to how much the tumor shrinks or stabilizes, and to monitor for any adverse events, which are side effects or unexpected medical occurrences.

Who can join the trial?

22 criteria

  • The patient must be between 6 and 21 years old at the time of signing the consent forms.
  • The patient must have pediatric low-grade glioma (pLGG), which is a type of slow-growing brain tumor, that is continuing to grow, has returned, or has not responded to previous treatments.
  • The tumor must be a Grade 1 or 2 glioma or glioneuronal tumor, which refers to the severity or growth speed of the brain tumor based on official medical classifications.
  • The tumor must show specific genetic alterations, such as a RAF alteration (a change in the DNA that can cause cells to grow abnormally), including KIAA1549::BRAF fusion, BRAF V600E mutation, or RAF1-fusions.
  • The patient must have a confirmed DNA methylation classification, which is a laboratory test used to identify the specific type of tumor by looking at how DNA is modified.
  • The disease must be measurable according to specific medical rules called RANO-LGG criteria, which are used to track if a tumor is getting larger or smaller.
  • If the patient is taking steroids (medicines used to reduce swelling or inflammation) for symptoms caused by the tumor, the dose must have stayed the same for at least 14 days before starting the study.
  • The patient must have a life expectancy of more than 3 months and be in a sufficient general condition, meaning they are healthy enough to participate based on standard medical scales like the Lansky or Karnofsky scales.
  • The patient must be able to swallow and keep down the medicine taken by mouth.
  • The patient must have adequate blood counts, including enough granulocytes (a type of white blood cell that fights infection), platelets (cells that help blood clot), and hemoglobin (a protein in red blood cells that carries oxygen).
  • The patient must have healthy organ function, specifically regarding the liver (measured by bilirubin, AST, and ALT levels) and the kidneys (measured by serum creatinine levels).
  • The patient must have a normal ECG (an electrical test of the heart), specifically a normal QTc interval, which measures the timing of the heart's electrical cycle.
  • The patient's Body Surface Area (BSA), which is a calculation of the total surface area of the body, must be at least 0.9 square meters.
  • Females of childbearing age must have a negative pregnancy test within 7 days of starting the study.
  • Women of childbearing age who are sexually active must use two forms of contraception (birth control), including one barrier method like a condom, during the study and for 180 days after.
  • Men who are sexually active with a female partner must use a condom plus another highly effective form of birth control for at least 120 days after the last treatment.
  • The patient and/or their legal representative must be able to understand the study and its consequences.
  • The patient must be willing to complete all study assessments, including electronic patient-reported outcomes, which are digital forms where patients report how they are feeling.
  • There must be no psychological, family, or social conditions that would make it difficult to follow the study rules and schedule.
  • Written informed consent, which is a legal document where the patient or guardian agrees to participate after being fully informed, must be signed.
  • For those in the "Treat-Biopsy" group, the patient must require a biopsy (taking a small sample of tissue) or partial resection (removing part of the tumor) as part of their standard medical care.
  • If a patient joins the study shortly after brain surgery, an MRI (a detailed medical image of the brain) must be completed within 72 hours after the procedure.

Who cannot join the trial?

18 criteria

  • Patients with high-grade gliomas, which are more aggressive and faster-growing brain tumors, or tumors where it is not known if they are cancerous.
  • Patients with specific genetic mutations, such as histone H3, isocitrate dehydrogenase (IDH) 1/2, or cyclin-dependent kinase inhibitor (CDKN)2A/B deletion, which are changes in the DNA that affect how cells grow.
  • Patients taking certain medications that affect cytochrome P450 enzymes (proteins in the liver that help break down drugs) or medications that interact with the breast cancer resistance protein (BCRP).
  • Use of traditional herbal medicines, as these can cause unexpected reactions when mixed with the study drug.
  • Patients with a history of hypersensitivity, which means an allergy or an extreme physical reaction, to the study drug, similar drugs, or any of the excipients (inactive ingredients used to make the medicine).
  • Females who are pregnant or lactating (breastfeeding).
  • Patients who are being held in an institution by a legal or official order.
  • Patients who are financially dependent on the person or group running the study.
  • Patients who are already participating in other ongoing clinical trials.
  • Patients who have previously participated in this specific clinical trial.
  • Patients diagnosed with or suspected of having NF-1, which is a genetic condition that can cause tumors to grow.
  • Patients with central nervous system (CNS) tumors (tumors in the brain or spinal cord) or metastases (cancer that has spread to other parts of the body) who have neurologically unstable symptoms, such as convulsions (seizures), despite receiving treatment.
  • Patients who cannot take medicine by mouth due to contraindications (medical reasons to avoid a treatment), such as severe nausea and vomiting, malabsorption (the inability to properly soak up nutrients or medicine), or having had part of the small bowel removed.
  • Patients with clinically significant, uncontrolled heart disease.
  • Patients who have received any anticancer therapy, such as chemotherapy or immunotherapy, within a specific timeframe determined by the drug's half-life (the time it takes for the amount of drug in the body to reduce by half).
  • Patients who have received radiotherapy (the use of radiation to kill cancer cells) within 12 weeks of starting the study drug.
  • Patients who have been treated with ulixertinib in the past.
  • Patients who have had a major surgical procedure not related to their brain tumor within 21 days, or those who will likely need major surgery during the study.
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Investigated drugs

  • Vinblastine

    is a medication given through a vein that works as a chemotherapy drug to help stop the growth of cancer cells.

  • Ulixertinib

    is a medication taken by mouth that is designed to target specific proteins in cells to help stop cancer from growing or spreading.

  • Tovorafenib

    is a medication taken by mouth that works by blocking certain signals in cells that tell them to grow, which is used to treat cancer.

What is already known about the treatment

  • Vinblastine sulfate

    This medication is given as an injection directly into a vein to treat certain types of cancer. It is a well-established chemotherapy drug used to slow the growth of cancer cells. At a molecular level, it works by stopping cells from dividing properly, which prevents the tumor from spreading. It is classified as a vinca alkaloid.

  • Ulixertinib

    This medicine is taken orally in tablet form and is currently being studied for its potential to treat specific types of cancer. It belongs to a group of drugs called kinase inhibitors. It works by blocking specific proteins within cells that act like "on" switches for tumor growth, thereby helping to stop the cancer from progressing.

  • Tovorafenib

    This is an oral tablet that is being investigated in clinical research for treating pediatric brain tumors. It is classified as a kinase inhibitor designed to target specific molecular signals. By blocking these signals, the drug prevents the cancer cells from receiving the instructions they need to grow and multiply.

Investigated diseases

Pediatric low-grade glioma - This is a type of slow-growing tumor that occurs in the brain of children. These tumors develop from cells within the nervous system and typically grow at a gradual pace. In some cases, the disease may progress by continuing to expand in size or by recurring after an initial period of stability. It can also become resistant to standard management approaches over time.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-516896-34-00Protocol codeKiTZ-EPILOGUE-2024Estimated enrolment56 patientsSponsorUniversitaetsklinikum Heidelberg AöR

sourced from the EU Clinical Trials Register and site verification

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