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Testing Tuvusertib in Patients with Returning IDH and ATRX Mutant Astrocytoma Brain Tumor

Verified siteInvestigationalNo placebo
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What is this trial about?

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

This study involves patients with a specific type of brain tumor called astrocytoma that has certain genetic changes, including IDH mutation, ATRX mutation, and p53 mutation. Astrocytoma is a type of brain tumor that develops from star-shaped cells in the brain. The study will use a medicine called tuvusertib, which is given as a tablet by mouth. The purpose of the study is to evaluate how well tuvusertib works in patients whose astrocytoma has come back after previous treatment with radiation therapy and chemotherapy.

Patients in this study will take tuvusertib tablets daily for up to 480 days. During the study, patients will have regular visits where doctors will check how the tumor is responding to treatment using magnetic resonance imaging, which is a type of scan that creates detailed pictures of the brain. The doctors will follow specific guidelines called RANO criteria to determine if the tumor is growing, shrinking, or staying the same. Patients will also have blood tests to check their overall health and will be asked questions about their thinking abilities, daily activities, and any symptoms they may be experiencing.

The study will measure how long patients live without their tumor getting worse, which is the main goal of the research. Other measurements include how many patients see their tumor shrink, overall survival time, how long before another treatment is needed, and any side effects from the medicine. Patients will also complete questionnaires about their quality of life and how they are functioning in their daily activities. Regular brain scans and medical examinations will be performed throughout the study to monitor the tumor and the patient's condition.

The research process

The trial runs in 10 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Starting treatment with tuvusertib

    Treatment begins with tuvusertib, which is an oral medication in the form of a film-coated tablet.

    The medication is taken by mouth.

    The corticosteroid dose must remain stable for 2 weeks before starting tuvusertib. The maximum allowed dose is 4 mg of dexamethasone per day or an equivalent dose of another corticosteroid.

  2. Step 2

    Regular monitoring with imaging scans

    Disease progression is monitored using magnetic resonance imaging (MRI), which is a type of scan that creates detailed images of the brain.

    The scans are evaluated according to RANO 2.0 criteria, which are standardized guidelines for assessing brain tumor response to treatment.

    These imaging assessments occur at regular intervals throughout the treatment period.

  3. Step 3

    Assessment at 6 months

    At 6 months after the first dose of tuvusertib, an evaluation is performed to determine if the disease has progressed.

    This assessment measures progression-free survival, which means the length of time during and after treatment that the disease does not get worse.

    MRI scans are used to evaluate the status of the disease according to RANO 2.0 criteria.

  4. Step 4

    Ongoing monitoring of treatment response

    Throughout the treatment period, the response to tuvusertib is continuously evaluated.

    This includes monitoring for objective response, which measures whether the tumor shrinks, stays the same, or grows.

    Assessments continue to track progression-free survival and overall survival.

  5. Step 5

    Neurological and functional assessments

    Neurocognitive function is assessed using several tests: HVLT (memory test), TMT-A and TMT-B (tests measuring attention and mental flexibility), and COWA (verbal fluency test).

    Neurologic status is evaluated using the NANO scale and MMSE test, which assess brain function and cognitive abilities.

    Functional status is measured using the Barthel index (ability to perform daily activities) and Karnofsky index (overall functional ability).

  6. Step 6

    Patient-reported outcomes

    Information about symptoms and quality of life is collected through questionnaires.

    The NCI-PRO-CTCAE Custom Survey is used to report any side effects or symptoms experienced.

    The PGIC Questionnaire (Patient Global Impression of Change) is used to assess overall improvement or worsening of condition.

  7. Step 7

    Safety monitoring

    Any adverse events, which are unwanted or harmful effects that occur during treatment, are monitored and recorded.

    Particular attention is given to treatment-related adverse events, which are side effects directly linked to tuvusertib.

    Treatment compliance is tracked, including any dose reductions or interruptions that may be necessary.

  8. Step 8

    Blood tests for safety monitoring

    Regular blood tests are performed to monitor blood cell counts, liver function, and kidney function.

    Blood tests check platelet count (cells that help blood clot), hemoglobin (protein that carries oxygen in blood), and absolute neutrophil count (white blood cells that fight infection).

    Liver function is assessed by measuring bilirubin (substance produced by the liver), AST and ALT (liver enzymes).

    Kidney function is monitored by measuring serum creatinine and calculating creatinine clearance, which indicates how well the kidneys are filtering waste from the blood.

  9. Step 9

    Long-term follow-up

    Follow-up continues to monitor overall survival, which is the length of time from the start of treatment until death from any cause.

    Time to next intervention is tracked, which measures how long before another treatment becomes necessary.

    Scheduled visits and examinations continue according to the protocol requirements.

  10. Step 10

    Contraception requirements during and after treatment

    Male participants must use a condom during sexual activity throughout the treatment period and for at least 3 months after the last dose of tuvusertib. Sperm donation is not permitted during this time.

    Female participants of childbearing potential must use highly effective contraception (with less than 1% failure rate per year) during treatment and for at least 6 months after the last dose of tuvusertib. Egg donation is not permitted during this time.

    Pregnancy tests are required within 72 hours before the first dose of tuvusertib for women of childbearing potential.

Who can join the trial?

13 criteria

  • You must provide written informed consent, which is a document showing you agree to join the study, before any study activities begin.
  • If you are a male participant, you must agree not to donate sperm during the study and for at least 3 months after your last dose of the study medicine. You must also either avoid any sexual activity or use a condom during sexual intercourse. If your partner can become pregnant, she must also use highly effective birth control, which is a method to prevent pregnancy. You must use a condom with pregnant partners as well.
  • If you are a female participant, you must not be pregnant or breastfeeding. If you are able to become pregnant, you must use highly effective birth control before starting the study medicine, during the study, and for at least 6 months after your last dose. You must also agree not to donate eggs during this time. You will need a negative pregnancy test within 72 hours before receiving your first dose of study medicine.
  • You must be able to swallow and take medications by mouth.
  • You must be willing and able to follow the study plan, attend all scheduled visits, and complete all required examinations and follow-up appointments.
  • You must be 18 years of age or older when you sign the consent form.
  • Your Karnofsky performance status must be greater than 60 percent. This is a score that measures how well you can perform daily activities, with higher numbers meaning you function better.
  • You must have been diagnosed with Grade 2 to 4 astrocytoma, which is a type of brain tumor, with a specific change in a gene called IDH.
  • Your tumor must have confirmed changes in two other genes called ATRX and p53, which will be checked through laboratory tests. A test for another gene called CDKN2A is also required.
  • Your disease must be getting worse and able to be measured according to specific brain tumor assessment guidelines called RANO 2.0 criteria. Your MRI scan, which is an imaging test of your brain, must show disease that lights up with contrast dye.
  • You must have already received standard treatment including radiotherapy, which is radiation treatment, and chemotherapy, which is drug treatment to kill cancer cells, using specific medicines.
  • Your dose of corticosteroids, which are medicines used to reduce swelling in the brain, must have been stable for 2 weeks before starting the study medicine. The maximum allowed dose is 4 milligrams per day of dexamethasone or an equivalent amount of a similar medicine.
  • Your blood tests must show adequate function of your blood cells, liver, and kidneys. This includes having enough platelets, which help your blood clot, at least 100,000 per cubic millimeter; enough hemoglobin, which carries oxygen in your blood, at least 9.0 grams per deciliter; enough neutrophils, which are white blood cells that fight infection, at least 1,500 per microliter without recent growth factor treatment; bilirubin level, which shows liver function, not more than 1.5 times the normal upper limit; liver enzyme levels called AST and ALT not more than 3 times the normal upper limit; and creatinine level, which shows kidney function, not more than 1.5 times the normal upper limit, or if higher, your kidney filtering ability must be at least 50 milliliters per minute.

Who cannot join the trial?

5 criteria

  • The study does not list specific exclusion criteria in the provided information. However, patients should discuss with their doctor whether they meet all requirements to participate in this clinical trial.
  • Patients who do not have the specific type of brain tumor being studied cannot participate. This study is only for people with a type of astrocytoma (a brain tumor) that has specific changes in genes called IDH1/2, ATRX, and p53, and where the tumor has come back for the first time after initial treatment.
  • Patients whose tumor has come back more than once after treatment may not be eligible, as this study focuses on the first recurrence (first time the tumor returns after treatment).
  • Patients whose tumor does not have all three specific genetic changes mentioned above cannot participate in this study.
  • Your doctor will need to confirm your tumor type and genetic characteristics through testing before you can be considered for this study.
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Investigated drugs

Tuvusertib is an investigational medication being tested in this clinical trial. It is designed to treat a specific type of brain tumor called astrocytoma that has certain genetic changes (mutations in IDH1/2, ATRX, and p53 genes) and has come back after previous treatment. This medication works by targeting specific processes in cancer cells to help stop or slow down tumor growth. In this study, patients will receive tuvusertib alone to see how well it prevents the cancer from getting worse, as measured by brain scans.

What is already known about the treatment

Tuvusertib – Tuvusertib is an experimental medication currently being studied in clinical trials for the treatment of a specific type of brain tumor called astrocytoma that has certain genetic changes (mutations in IDH1/2, ATRX, and p53 genes). This drug is being tested in patients whose brain tumor has come back after initial treatment, and researchers are evaluating how well it works by measuring how long patients live without their disease getting worse. The medication is taken as a single treatment (monotherapy), and doctors monitor its effects using brain scans to see if the tumor stops growing or spreading.

Investigated diseases

IDH1/2-mutated, ATRX-mutated and p53-mutated astrocytoma – This is a specific type of brain tumor that develops from star-shaped brain cells called astrocytes. The disease is characterized by three particular genetic changes: mutations in the IDH1 or IDH2 genes, mutation in the ATRX gene, and mutation in the p53 gene. These tumors grow abnormally in the brain tissue and can affect normal brain function. The disease can progress over time, and in some cases, it may return after initial treatment, which is called recurrence. When the tumor grows or spreads, it is considered to be progressing. The genetic mutations that define this type of astrocytoma distinguish it from other forms of brain tumors.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-521843-19-00Protocol codeGEINO-2401Estimated enrolment56 patientsSponsorGrupo Espanol De Investigacion En Neurooncologia

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