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Study of Ponatinib for Patients with Resistant Chronic Myeloid Leukemia

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 a type of blood cancer called Chronic Myeloid Leukemia (CML), specifically in its chronic phase. The study is investigating the effectiveness and safety of a medication called Ponatinib, which is also known by the brand name Iclusig. Ponatinib is a type of treatment known as a kinase inhibitor, which works by blocking certain proteins that help cancer cells grow. The trial involves patients who have a specific genetic marker, known as the T315I mutation, or those who have not responded well to previous treatments with other kinase inhibitors.

The purpose of the study is to evaluate how well Ponatinib works when given in different doses: 45 mg, 30 mg, and 15 mg daily. Participants will take the medication in the form of film-coated tablets, which are taken orally. The study will monitor the participants over a period to see how their condition responds to the treatment. The goal is to see if the medication can reduce the levels of a specific protein associated with CML, known as BCR-ABL1, to a very low level within 12 months. This protein is a marker used to measure the presence of cancer cells in the body.

Throughout the study, the safety of Ponatinib will also be assessed by observing any side effects or adverse reactions that participants may experience. The trial aims to provide valuable information on the best dosage of Ponatinib for treating patients with resistant CML, helping to improve treatment options for those affected by this condition.

The research process

The trial runs in 4 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 assigned to one of three starting doses of ponatinib: 45 mg, 30 mg, or 15 mg. This medication is taken orally in the form of film-coated tablets.

    The purpose of the study is to evaluate the effectiveness and safety of these doses in patients with chronic phase chronic myeloid leukemia (CP-CML) who are resistant to previous treatments or have a specific genetic mutation known as T315I.

  2. Step 2

    Medication administration

    The patient will take the assigned dose of ponatinib daily. The medication is administered orally, meaning it is taken by mouth.

    The study will monitor the patient's response to the medication over a period of 12 months, with the goal of achieving a specific reduction in the BCR-ABL1IS level, which is a marker used to measure the effectiveness of the treatment.

  3. Step 3

    Monitoring and assessments

    Throughout the study, the patient will attend scheduled visits for monitoring and assessments. These visits will include various tests to evaluate the patient's response to the treatment and to monitor for any side effects.

    The primary goal is to achieve a BCR-ABL1IS level of less than or equal to 1% at 12 months. Secondary goals include measuring molecular and cytogenetic response rates, as well as monitoring the safety and tolerability of the treatment.

  4. Step 4

    Completion of the study

    The study is expected to conclude by June 30, 2025. At the end of the study, the patient's overall response to the treatment will be evaluated, including any changes in their condition and any side effects experienced.

    The results will help determine the most effective and safe dose of ponatinib for patients with CP-CML who are resistant to previous treatments or have the T315I mutation.

Who can join the trial?

12 criteria

  • Have **Chronic Phase Chronic Myeloid Leukemia (CP-CML)** and have received at least two prior treatments with a type of medication called **TKI** (Tyrosine Kinase Inhibitor) and shown resistance to treatment, or have a documented history of a specific genetic change called **T315I mutation** after receiving any number of prior TKI treatments.
  • Be at least **18 years old**.
  • Have a performance status of **0, 1, or 2** on a scale used to assess how a disease affects a patient's daily living abilities, known as the **Eastern Cooperative Oncology Group (ECOG) performance status**.
  • Have adequate **kidney function**, which means certain blood tests show that the kidneys are working well enough.
  • Have adequate **liver function**, which means certain blood tests show that the liver is working well enough.
  • Have normal **pancreatic status**, which means certain blood tests show that the pancreas is working well enough.
  • Have a normal **QT interval** on an **electrocardiogram (ECG)**, which is a test that checks the heart's electrical activity.
  • For females who can have children, have a **negative pregnancy test** before joining the study.
  • Agree to use a highly effective form of **birth control** with sexual partners from the start of the study through at least 4 months after the end of treatment, for both male and female patients who can have children.
  • Provide **written informed consent**, which means agreeing to participate in the study after being fully informed about it.
  • Be willing and able to follow the **scheduled visits and study procedures**.
  • Have recovered from any side effects related to previous cancer treatments to a certain level, as defined by a standard called **NCI CTCAE v 4.0 grade 1** or lower.

Who cannot join the trial?

7 criteria

  • Patients who have a different type of leukemia other than Chronic Phase Chronic Myeloid Leukemia (CP-CML) cannot participate. CP-CML is a specific stage of a blood cancer called chronic myeloid leukemia.
  • Patients who are not resistant to previous TKI therapy cannot participate. TKI therapy refers to a treatment using drugs called tyrosine kinase inhibitors, which are used to treat certain types of cancer.
  • Patients who do not have the T315I mutation cannot participate. This mutation is a specific change in the cancer cells that can affect how the disease responds to treatment.
  • Patients who are not within the specified age range cannot participate. The age range is typically defined by the study and includes certain age groups.
  • Patients who are not part of the specified clinical trial group cannot participate. This refers to the specific group of patients the study is focusing on.
  • Patients who are not male or female cannot participate. The study includes both male and female participants.
  • Patients who are not considered part of a vulnerable population cannot participate. Vulnerable populations may include groups like children, pregnant women, or others who need special protection in research.
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Investigated drugs

Ponatinib is a medication used in this trial to treat patients with chronic phase chronic myeloid leukemia (CP-CML) who have not responded to previous treatments or have a specific genetic mutation known as T315I. The trial aims to understand how effective and safe ponatinib is when given in different starting doses. Ponatinib works by blocking certain proteins that help cancer cells grow, which may help to control the disease.

What is already known about the treatment

Ponatinib – Ponatinib is administered orally in tablet form and is currently being studied in clinical trials for its effectiveness in treating patients with chronic phase chronic myeloid leukemia (CP-CML) who are resistant to previous treatments or have a specific genetic mutation known as T315I. It is classified as a tyrosine kinase inhibitor, which works by blocking the activity of proteins that promote the growth of cancer cells. The medication is being evaluated for its ability to reduce the levels of a specific protein marker, BCR-ABL1, in the blood, which is associated with the progression of leukemia.

Investigated diseases

Chronic Phase Chronic Myeloid Leukemia – Chronic Phase Chronic Myeloid Leukemia (CP-CML) is a type of cancer that affects the blood and bone marrow. It is characterized by the overproduction of immature white blood cells, known as myeloid cells. In the chronic phase, the disease progresses slowly, and patients may not experience significant symptoms initially. Over time, the accumulation of these cells can lead to symptoms such as fatigue, weight loss, and an enlarged spleen. The disease is associated with a specific genetic abnormality known as the Philadelphia chromosome, which results in the production of an abnormal protein that promotes cancer cell growth. If left untreated, CP-CML can progress to more aggressive phases, known as the accelerated phase and blast phase.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514516-27-00Protocol codeAP24534-14-203Estimated enrolment251 patientsSponsorTakeda Development Center Americas Inc.

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