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Study of Palbociclib with Drug Combinations for Children and Young Adults with Recurrent or Refractory Neuroblastoma and Ewing Sarcoma

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 treatments for children, adolescents, and young adults with certain types of cancer that have returned or are not responding to treatment. The cancers being studied include neuroblastoma and Ewing sarcoma. The trial will test the effectiveness of a medication called Palbociclib (also known as Ibrance®) when used in combination with other cancer treatments. These combinations include Irinotecan and Temozolomide, or Topotecan and Cyclophosphamide. The purpose of the study is to evaluate how well these combinations work in treating these cancers.

Participants in the study will receive one of the treatment combinations, and some may receive a placebo. The study will monitor the safety and effectiveness of these treatments over time. The trial will also look at how the body processes these medications and their impact on the participants' quality of life. The study aims to find the best dose of these medications that can be safely given to patients.

The trial will involve regular check-ups and tests, such as MRI or CT scans, to track the progress of the treatment. Participants will be closely monitored for any side effects or changes in their condition. The study is expected to continue until November 2025, with the goal of improving treatment options for young patients with these challenging cancers.

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, the patient will be informed about the trial's purpose and procedures. Consent will be obtained to ensure understanding and agreement to participate.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm eligibility. This includes a review of medical history, physical examination, and necessary laboratory tests to ensure adequate bone marrow, liver, and kidney function.

  3. Step 3

    Treatment phase 1

    The patient will receive a combination of medications. The specific combination depends on the group assigned.

    One group will receive palbociclib (oral tablet), irinotecan (intravenous), and temozolomide (oral capsule).

    Another group will receive palbociclib (oral tablet), topotecan (intravenous), and cyclophosphamide (intravenous).

    The dosage and frequency will be determined by the study protocol and adjusted based on the patient's response and tolerance.

  4. Step 4

    Monitoring and evaluation

    Regular monitoring will occur throughout the treatment phase. This includes blood tests, imaging studies, and assessments to evaluate the response to treatment and any side effects.

    The patient's health and any adverse effects will be closely monitored to ensure safety.

  5. Step 5

    Treatment phase 2

    If the patient continues to meet the criteria, they will proceed to the second phase of treatment. This phase may involve adjustments to the medication regimen based on the initial response.

    The same medications will be used, with potential changes in dosage or frequency as needed.

  6. Step 6

    Follow-up

    After completing the treatment phases, follow-up visits will be scheduled to monitor long-term effects and overall health.

    These visits will include physical exams, laboratory tests, and imaging studies to assess the patient's condition.

Who can join the trial?

9 criteria

  • The patient must have a solid tumor that has come back or is not responding to treatment. This includes certain types of brain tumors but not lymphomas. For some specific tumors, a scan showing the tumor is enough to join the study.
  • The patient must be between 2 and 21 years old at the time of joining the study.
  • The patient must be able to perform daily activities to a certain level. For those 16 years or younger, they should be able to do at least 50% of normal activities. For those older than 16, they should be able to do most activities with some limitations.
  • The patient must have enough healthy blood cells. This means having a certain number of white blood cells, platelets, and hemoglobin without needing recent blood transfusions.
  • The patient must have healthy kidneys, which is checked by measuring a substance in the blood called creatinine.
  • The patient must have a healthy liver. This is checked by measuring certain enzymes and a substance called bilirubin in the blood.
  • The patient must have a tumor that can be measured or evaluated using specific medical criteria. For neuroblastoma, the tumor must be visible on certain scans or found in the bone marrow.
  • The patient must have recovered from any side effects of previous treatments, except for hair loss.
  • If the patient is a girl aged 8 or older, she must have a negative pregnancy test before starting the study.

Who cannot join the trial?

7 criteria

  • Patients who have a different type of cancer than the ones being studied, such as neuroblastoma or Ewing sarcoma, cannot participate.
  • Patients who are not within the specified age range for the study, which includes children, adolescents, and young adults, cannot participate.
  • Patients who are not able to safely receive the study medications due to other health conditions cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients who have certain allergies or reactions to the study medications cannot participate.
  • Patients who are unable to follow the study procedures or attend required visits cannot participate.
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Investigated drugs

  • Palbociclib (Ibrance®)

    is a medication used in this trial to help treat children, adolescents, and young adults with certain types of solid tumors that have come back or are difficult to treat. It works by blocking specific proteins that help cancer cells grow, which may slow down or stop the growth of the tumor.

  • Irinotecan

    is a chemotherapy drug used in this study to treat solid tumors. It works by interfering with the DNA of cancer cells, preventing them from dividing and growing. This can help reduce the size of the tumor or slow its growth.

  • Temozolomide

    is another chemotherapy medication included in the trial. It is used to treat certain types of cancer by damaging the DNA of cancer cells, which can lead to their death. This helps in controlling the spread of the tumor.

  • Topotecan

    is a chemotherapy drug that is part of the study. It works by inhibiting an enzyme needed for cancer cell replication, which can help stop the growth of the tumor.

  • Cyclophosphamide

    is a chemotherapy medication used in this trial. It works by damaging the DNA of cancer cells, which can prevent them from multiplying and lead to their destruction. This helps in managing the tumor's growth.

What is already known about the treatment

Palbociclib – This medication is administered orally and is currently being studied in combination with other drugs for treating recurrent or refractory solid tumors in pediatric patients. It is in the clinical trial phase, with ongoing research to determine its effectiveness and safety. The main therapeutic indications include recurrent or refractory neuroblastoma and Ewing sarcoma. Palbociclib works by inhibiting certain proteins called CDK4 and CDK6, which are involved in cell division, thereby slowing down the growth of cancer cells. It is classified as a CDK4/6 inhibitor.

Investigated diseases

  • Recurrent or Refractory Neuroblastoma

    Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body, most commonly affecting children. In recurrent or refractory cases, the disease either returns after treatment or does not respond to standard therapies. It often begins in the adrenal glands but can also develop in the neck, chest, abdomen, or spine. The progression of the disease can vary, with some tumors growing slowly and others rapidly. Symptoms may include abdominal pain, a lump in the abdomen, or changes in bowel habits. As the disease progresses, it can spread to other parts of the body, such as the bones or bone marrow.

  • Recurrent/Refractory Ewing Sarcoma

    Ewing sarcoma is a type of cancer that occurs in bones or in the soft tissue around the bones, primarily affecting children and young adults. In recurrent or refractory cases, the cancer returns after treatment or does not respond to standard therapies. It typically begins in the long bones of the arms and legs, pelvis, or chest wall. The disease can progress by spreading to other parts of the body, such as the lungs or other bones. Symptoms often include pain and swelling at the tumor site, fever, and fatigue. As the disease advances, it may lead to more severe symptoms and complications.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-511975-14-00Protocol codeA5481092Estimated enrolment94 patientsSponsorPfizer Inc.

sourced from the EU Clinical Trials Register and site verification

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