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Study on Ponatinib and Chemotherapy for Adults with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL)

Fast replyRegistered 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 Philadelphia chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL). The study aims to evaluate the effectiveness of a medication called Ponatinib, which may be used alone or in combination with other chemotherapy drugs. The chemotherapy drugs involved in this study include Cytarabine, Methylprednisolone, Methotrexate, Vincristine, and Prednisone. These medications are used to help manage the disease by reducing the number of cancer cells in the body.

The purpose of the study is to see how well Ponatinib works in helping patients achieve remission, which means reducing or eliminating signs of cancer. The study will look at patients who have persistent or relapsed disease, as well as those who have not achieved remission after previous treatments. Participants will receive treatment over a period of time, and their response to the medication will be monitored. Some patients may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, the safety and side effects of the treatments will be closely monitored. The study will also explore how the treatment affects the presence of specific genetic changes in the cancer cells, such as mutations in the BCR-ABL1 gene. The goal is to understand how these treatments can help patients achieve better outcomes and improve their quality of life. The study is expected to continue until 2026, providing valuable information on the management of Ph+ ALL.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the presence of Ph+ acute lymphoblastic leukemia and minimal residual disease (MRD) or relapse.

    Basic health parameters such as liver and pancreatic function are evaluated. A pregnancy test is required for females of childbearing potential.

  2. Step 2

    Treatment initiation

    Treatment begins with the administration of ponatinib, which is taken orally in the form of 15 mg film-coated tablets. The frequency and duration of this medication are determined by the study protocol.

    In some cases, ponatinib is combined with chemotherapy drugs such as cytarabine, methylprednisolone, methotrexate, vincristine, and prednisone. These drugs are administered through various routes, including intrathecal (directly into the spinal canal) and intravenous (into a vein).

  3. Step 3

    Monitoring and evaluation

    Throughout the treatment, regular monitoring is conducted to assess the response to therapy. This includes checking for MRD negativity or reduction after three months.

    The safety profile is evaluated by monitoring for any side effects or toxicities. Adjustments to the treatment plan may be made based on these evaluations.

  4. Step 4

    Follow-up assessments

    Follow-up assessments are conducted to determine the duration of complete molecular remission (CMR) and to evaluate the best molecular response achieved.

    Additional analyses may include mutational analysis and correlation studies to understand the impact of treatment on different genetic profiles.

  5. Step 5

    Long-term outcomes

    The study aims to evaluate long-term outcomes such as disease-free survival (DFS) and overall survival (OS) at 24 months.

    The role of clinical and biological assessments at baseline is also analyzed to understand their impact on treatment outcomes.

Who can join the trial?

9 criteria

  • Patients must have **Ph+ Acute Lymphoblastic Leukemia (ALL)**, which is a type of blood cancer.
  • Patients should show evidence of **MRD disease** (Minimal Residual Disease) or be in **relapse/refractoriness**, meaning the disease has returned or is not responding to treatment.
  • Patients must be **18 years or older**. There is no upper age limit.
  • Patients need to have **adequate liver function**, which means:
    • Total serum bilirubin should be no more than 1.5 times the normal upper limit, unless they have **Gilbert's syndrome**, a mild liver disorder.
    • **ALT (alanine aminotransferase)** and **AST (aspartate aminotransferase)** levels should be no more than 2.5 times the normal upper limit. These are enzymes that help assess liver health.
    • Patients need to have **adequate pancreatic function**, meaning:
      • **Serum lipase and amylase** levels should be no more than 1.5 times the normal upper limit. These are enzymes that help assess pancreas health.
      • Females who can have children must have a **negative pregnancy test** before joining the study.
      • Both female and male patients who can have children must agree to use an **effective form of birth control** with their partners from the start of the study until 4 months after treatment ends.
      • Patients must provide **signed written informed consent**, which means they agree to participate in the study after understanding all the details, according to international and local laws.

Who cannot join the trial?

7 criteria

  • Patients who have a different type of leukemia than Ph+ Acute Lymphoblastic Leukemia cannot participate. This is a specific type of blood cancer.
  • Patients who are not within the specified age range for the study cannot participate. The study is only for certain age groups.
  • Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible to participate.
  • Patients who have allergies or reactions to the study medication or similar medications cannot participate.
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Investigated drugs

Ponatinib is a medication used in this clinical trial to manage minimal residual disease (MRD) and hematologic relapse in adult patients with a specific type of leukemia known as Ph+ acute lymphoblastic leukemia (Ph+ ALL). It is being tested to see how well it can reduce or eliminate the remaining cancer cells after initial treatment. The study is evaluating the effects of using ponatinib alone or in combination with other chemotherapy treatments over a period of three months.

What is already known about the treatment

Ponatinib – Ponatinib is administered orally in tablet form. It is currently being studied for its effectiveness in treating minimal residual disease and hematologic relapse in adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The medication works by inhibiting specific proteins that promote cancer cell growth, particularly targeting the BCR-ABL protein, which is a result of the Philadelphia chromosome mutation. Ponatinib is classified as a tyrosine kinase inhibitor, a type of targeted cancer therapy.

Investigated diseases

Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia – This is a type of cancer that affects the blood and bone marrow, characterized by the presence of the Philadelphia chromosome. It is a subtype of acute lymphoblastic leukemia (ALL) where the bone marrow produces too many immature white blood cells, known as lymphoblasts. The disease progresses rapidly, leading to an accumulation of these abnormal cells in the blood and bone marrow, which can interfere with the production of normal blood cells. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. The presence of the Philadelphia chromosome is due to a specific genetic abnormality that results in the production of an abnormal protein that promotes the growth of cancer cells. This condition is considered rare and requires specialized medical attention.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-516868-28-00Protocol codeALL2620Estimated enrolment67 patientsSponsorFondazione Gimema Franco Mandelli Onlus

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