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Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children

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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 effects of a medication called Ponatinib on certain types of cancers in children. The cancers being studied include recurrent or refractory leukemias, which are blood cancers that have returned or do not respond to treatment, and solid tumors, which are abnormal masses of tissue. The purpose of the study is to evaluate the safety and effectiveness of Ponatinib in treating these conditions. Ponatinib is a type of medication known as a kinase inhibitor, which works by blocking certain proteins that help cancer cells grow.

The study is divided into two phases. In the first phase, the goal is to find the best dose of Ponatinib for children with advanced blood cancers or solid tumors. In the second phase, the study will look at how well Ponatinib works in children with a specific type of leukemia called Chronic Myeloid Leukemia (CML) and other selected cancers. Participants will take Ponatinib orally, which means they will swallow it in the form of a tablet or capsule. The study will monitor the participants' health and any side effects they may experience while taking the medication.

Throughout the study, researchers will assess the participants' response to the treatment by using various medical tests and imaging techniques like MRI or CT scans. The study aims to provide valuable information on the potential benefits and risks of using Ponatinib in treating these challenging cancers in children. The study is expected to continue until 2025, allowing researchers to gather comprehensive data on the long-term effects of the treatment.

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 participant will be informed about the trial's objectives and procedures. Consent will be obtained from a parent or legal guardian, and assent from the participant if appropriate.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm the diagnosis of recurrent or refractory leukemias, lymphomas, or solid tumors. This may involve tests such as bone marrow aspirate or imaging studies.

  3. Step 3

    Medication administration

    The participant will receive ponatinib, administered orally. The dosage will be determined based on the phase of the study and the participant's specific condition.

    In Phase 1, the dosage will be adjusted to find the maximum tolerated dose. In Phase 2, the dosage will be based on the results from Phase 1.

  4. Step 4

    Treatment monitoring

    The participant's response to the treatment will be monitored regularly through clinical evaluations, blood tests, and imaging studies.

    The frequency and severity of any side effects will be recorded, and adjustments to the treatment may be made as necessary.

  5. Step 5

    Evaluation of treatment efficacy

    The effectiveness of the treatment will be assessed at specific intervals. This may include measuring the reduction in tumor size or changes in blood cell counts.

    For participants with chronic myeloid leukemia (CML), the response will be evaluated using specific criteria such as cytogenetic or molecular response.

  6. Step 6

    End of treatment

    The treatment will continue until the participant completes the study, experiences unacceptable side effects, or the disease progresses.

    After the treatment ends, follow-up assessments will be conducted to monitor the participant's health and any long-term effects of the treatment.

Who can join the trial?

7 criteria

  • Participants must have a confirmed diagnosis of certain types of cancer, such as **leukemias**, **lymphomas**, or **solid tumors**. This means that tests have shown they have one of these cancers.
  • Participants must have tried standard treatments for their cancer, but these treatments did not work or are not suitable for them.
  • Participants must be between 1 and 18 years old at the time of joining the study.
  • Participants must have a certain level of physical ability, measured by a test called the **Karnofsky performance status** for those 16 and older, or the **Lansky Play Scale** for those under 16. This means they need to be able to do some daily activities.
  • Participants must have recovered from any side effects of previous treatments to a certain level, except for hair loss.
  • Participants must agree to avoid pregnancy or fathering a child during the study and for a period after the study ends. This involves using effective birth control methods.
  • Participants must have a parent or legal guardian who can understand and sign a consent form, agreeing to the study's requirements and procedures.

Who cannot join the trial?

10 criteria

  • Patients who have a different type of cancer than the ones being studied, such as **recurrent or refractory leukemias, lymphomas, and solid tumors**. These are cancers that have come back after treatment or do not respond to treatment.
  • Patients who are not within the age range specified for the study. The study is for children, so adults cannot participate.
  • Patients who are not able to take the study medication by mouth, as the treatment is given orally.
  • Patients who have other serious health conditions that might interfere with the study treatment or make it unsafe for them to participate.
  • Patients who are pregnant or breastfeeding, as the effects of the study treatment on unborn babies or infants are not known.
  • Patients who are participating in another clinical trial at the same time, as this could affect the results of the study.
  • Patients who have had a recent major surgery or are recovering from a major surgery, as this could affect their ability to participate safely.
  • Patients who have an active infection that requires treatment, as this could interfere with the study treatment.
  • Patients who have a known allergy to the study medication or any of its ingredients.
  • Patients who have not recovered from the side effects of previous cancer treatments.
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Investigated drugs

Ponatinib is a medication being studied for its safety and effectiveness in treating certain types of leukemia and solid tumors in children. It is taken by mouth once a day. The study aims to find the best dose of Ponatinib for children with these conditions and to see how well it works for those who have not responded to other treatments or have specific genetic mutations.

What is already known about the treatment

Ponatinib – Ponatinib is administered orally, typically in the form of a tablet taken once daily. It is currently being studied in clinical trials for its safety and effectiveness in treating recurrent or refractory leukemias, lymphomas, and solid tumors, particularly in pediatric patients. The main therapeutic indications include chronic myeloid leukemia (CML) and other advanced hematologic malignancies. Ponatinib works by inhibiting the activity of specific proteins, such as BCR-ABL, which are involved in the growth and survival of cancer cells. It belongs to the pharmacological class of tyrosine kinase inhibitors, which are designed to block the action of enzymes that promote cancer cell proliferation.

Investigated diseases

  • Recurrent or Refractory Leukemias

    These are types of blood cancers that return after treatment or do not respond to standard therapies. Leukemias affect the blood and bone marrow, leading to an overproduction of abnormal white blood cells. As the disease progresses, it can interfere with the body's ability to produce healthy blood cells. Symptoms may include fatigue, frequent infections, and easy bruising or bleeding. The progression can vary depending on the specific type of leukemia and individual patient factors.

  • Lymphomas

    Lymphomas are cancers of the lymphatic system, which is part of the body's immune system. They typically begin in lymph nodes or other lymphatic tissues and can spread to other parts of the body. As lymphomas progress, they may cause swollen lymph nodes, fever, night sweats, and weight loss. The disease can be classified into Hodgkin and non-Hodgkin types, each with different progression patterns. The rate of progression can vary widely, with some lymphomas growing slowly and others more rapidly.

  • Solid Tumors

    Solid tumors are abnormal masses of tissue that can occur in various organs or tissues, excluding blood-related cancers. They can be benign (non-cancerous) or malignant (cancerous), with malignant tumors having the potential to invade nearby tissues and spread to other parts of the body. As solid tumors grow, they may cause symptoms related to the affected organ, such as pain, swelling, or dysfunction. The progression of solid tumors depends on the type, location, and aggressiveness of the cancer cells.

Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase ITrial ID2023-509699-41-00Protocol codeINCB 84344-102Estimated enrolment67 patientsSponsorIncyte Biosciences International S.a.r.l.

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