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Study on Ribociclib, Topotecan, and Temozolomide for Children and Young Adults (12 months - 21 years) with Relapsed or Refractory Neuroblastoma and Solid Tumors

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 clinical trial is focused on studying the treatment of relapsed or refractory neuroblastoma and other solid tumors in children and young adults aged 12 months to 21 years. Neuroblastoma is a type of cancer that forms in certain types of nerve tissue. The study will use a combination of medications, including ribociclib (also known by its code name LEE011), temozolomide, and topotecan hydrochloride. These medications are being tested together to see if they can effectively treat these types of cancers.

The purpose of the study is to assess the safety and effectiveness of this combination of drugs. The study is divided into two phases. In the first phase, researchers will determine the best dose of ribociclib to use with the other medications. In the second phase, the study will further evaluate how well this combination works in treating neuroblastoma compared to a placebo. Participants will receive the medications either orally or through intravenous infusion, depending on the specific drug.

Throughout the study, participants will be closely monitored for any side effects and to see how their tumors respond to the treatment. The study aims to find the most effective and safe way to use these medications together to help treat these challenging cancers in young patients. The trial is expected to continue until 2029, allowing researchers to gather comprehensive data on the treatment's impact.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, informed consent must be signed. This document confirms understanding and agreement to participate in the trial.

    Eligibility is based on age (12 months to 21 years) and diagnosis of specific solid tumors that have not responded to standard treatments.

  2. Step 2

    Phase I - part A: dose determination

    The goal is to find the maximum tolerated dose of ribociclib when combined with temozolomide and topotecan (referred to as TOTEM).

    Medications are administered as follows: temozolomide is taken orally, ribociclib is taken orally, and topotecan is given intravenously.

    The cycle lasts 28 days, during which the incidence of dose-limiting side effects is monitored.

  3. Step 3

    Phase I - part B: antitumor activity evaluation

    This phase evaluates the antitumor activity of the combination treatment.

    The overall response rate is assessed using specific criteria for different tumor types.

    Safety and tolerability are monitored, including any side effects and changes in health status.

  4. Step 4

    Phase II: treatment effect assessment

    The effectiveness of the treatment is compared to a placebo group.

    The primary focus is on the overall response rate, with additional assessments of progression-free survival and duration of response.

    Safety and tolerability continue to be evaluated, with regular monitoring of health indicators.

  5. Step 5

    Medication administration

    Temozolomide is administered as a powder for oral solution.

    Ribociclib is taken orally.

    Topotecan is administered intravenously, either as a slow bolus injection or through regular intravenous use.

  6. Step 6

    Monitoring and assessments

    Throughout the trial, regular assessments are conducted to monitor the patient's response to treatment.

    These include laboratory tests, imaging studies, and evaluations of overall health and quality of life.

Who can join the trial?

19 criteria

  • Participants must sign a document called an informed consent, which shows they understand the study and agree to take part. If the participant is a child, a guardian must sign for them.
  • Participants must be between the ages of 12 months and 21 years.
  • Participants must have certain types of solid tumors that have not responded to standard treatments or for which no effective standard treatment exists. These tumors include:
    • Neuroblastoma
    • Medulloblastoma
    • High-grade glioma
    • Malignant rhabdoid tumor
    • Rhabdomyosarcoma
    • Participants with central nervous system (CNS) disease who are taking medications called corticosteroids must have been on a stable dose for at least 7 days before starting the study medication.
    • Participants must have a measurable disease according to specific medical criteria used to assess tumors.
    • Participants must be able to sit upright in a wheelchair, even if they cannot walk due to paralysis.
    • Participants must have a life expectancy of at least 12 weeks.
    • Participants must have adequate bone marrow and organ function, which includes:
      • A certain level of neutrophils (a type of white blood cell) without needing extra support.
      • A certain level of platelets (cells that help with blood clotting) without needing extra support.
      • A certain level of hemoglobin (a protein in red blood cells) with or without transfusions.
      • Normal levels of bilirubin (a substance made by the liver) unless they have a condition called Gilbert's syndrome.
      • Normal liver function as shown by specific blood tests.
      • Normal kidney function as shown by specific blood tests.
      • Normal heart function as shown by specific heart tests.
      • Normal levels of potassium, magnesium, and calcium in the blood.

Who cannot join the trial?

10 criteria

  • Patients who have not recovered from previous treatments or surgeries.
  • Patients with other serious health conditions that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who are unable to follow the study procedures or attend scheduled visits.
  • Patients who have participated in another clinical trial recently.
  • Patients with allergies to the study medications.
  • Patients with certain infections that are not well controlled.
  • Patients with a history of certain heart problems.
  • Patients who are taking medications that could interfere with the study drugs.
  • Patients with a history of drug or alcohol abuse.
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Investigated drugs

  • Ribociclib

    is a medication used in this trial to help treat certain types of cancer. It works by blocking proteins that cancer cells need to grow and divide. In this study, it is being tested in combination with other drugs to see if it can help improve treatment outcomes for children with relapsed or refractory neuroblastoma and other solid tumors.

  • Topotecan

    is a chemotherapy drug that is used to treat cancer by interfering with the DNA of cancer cells, preventing them from growing and multiplying. In this trial, it is combined with other medications to assess its effectiveness in treating pediatric patients with specific types of cancer.

  • Temozolomide

    is another chemotherapy medication used in this study. It works by damaging the DNA of cancer cells, which can stop them from growing and spreading. The trial is testing its use in combination with other drugs to determine if it can improve treatment results for children with certain solid tumors.

What is already known about the treatment

  • Ribociclib

    This medication is administered orally and is currently being studied in combination with topotecan and temozolomide for its effectiveness and safety in treating pediatric patients with relapsed or refractory neuroblastoma and other solid tumors. Ribociclib is a cyclin-dependent kinase inhibitor, which works by blocking proteins that promote cell division, thereby slowing the growth of cancer cells. It is classified as an antineoplastic agent and is under investigation in clinical trials to determine its optimal dosing and therapeutic potential in combination therapies.

  • Topotecan

    Administered intravenously, topotecan is being evaluated in combination with ribociclib and temozolomide for its role in treating relapsed or refractory neuroblastoma and other solid tumors in children. It functions by inhibiting the enzyme topoisomerase I, which is essential for DNA replication, leading to the death of cancer cells. Topotecan is classified as a topoisomerase inhibitor and is part of ongoing research to assess its efficacy in combination treatments.

  • Temozolomide

    This medication is taken orally and is part of a combination therapy with ribociclib and topotecan in clinical trials aimed at treating pediatric patients with relapsed or refractory neuroblastoma and other solid tumors. Temozolomide works by damaging the DNA of cancer cells, which prevents them from dividing and growing. It is classified as an alkylating agent and is being studied to determine its effectiveness and safety in combination with other cancer treatments.

Investigated diseases

  • Neuroblastoma

    Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body, most commonly in and around the adrenal glands. It primarily affects children and can also occur in the abdomen, chest, neck, and near the spine. The disease often begins in the early stages of development and can spread to other parts of the body. Symptoms may include fatigue, loss of appetite, and a swollen abdomen. As it progresses, it may cause bone pain, fever, and weight loss.

  • Medulloblastoma

    Medulloblastoma is a type of brain tumor that originates in the cerebellum, the part of the brain that controls balance and coordination. It is most common in children but can also occur in adults. The tumor can grow rapidly and may spread to other parts of the brain and spinal cord. Symptoms often include headaches, nausea, vomiting, and problems with coordination. As the disease progresses, it may lead to changes in behavior and difficulty with movement.

  • High-Grade Glioma

    High-grade gliomas are aggressive brain tumors that arise from glial cells, which support nerve cells in the brain. These tumors grow quickly and can invade nearby brain tissue, making them difficult to treat. Symptoms may include headaches, seizures, and changes in personality or memory. As the tumor progresses, it can cause weakness, vision problems, and difficulty with speech.

  • Malignant Rhabdoid Tumor

    Malignant rhabdoid tumors are rare and aggressive cancers that typically occur in the kidneys but can also develop in other soft tissues. They are most commonly diagnosed in young children. The tumors grow rapidly and can spread to other parts of the body. Symptoms may include a noticeable lump, pain, and swelling in the affected area. As the disease advances, it may lead to weight loss and fatigue.

  • Rhabdomyosarcoma

    Rhabdomyosarcoma is a cancer that forms in the soft tissue, specifically in skeletal muscle tissue or sometimes in hollow organs such as the bladder or uterus. It is most common in children and adolescents. The tumor can occur in various parts of the body, including the head, neck, and limbs. Symptoms depend on the tumor's location and may include a visible lump, pain, and swelling. As it progresses, it can interfere with normal body functions and spread to other areas.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-512095-35-00Protocol codeCLEE011Q12101Estimated enrolment91 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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