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Asciminib in Pediatric Patients With Philadelphia Positive Chronic Myeloid Leukemia in Chronic Phase

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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 studying chronic myeloid leukemia in children and adolescents, including cases that are newly diagnosed and cases that were treated before but did not respond well or were not tolerated. Some participants may have a change called the T315I mutation, which can make the disease harder to treat. The treatment being tested is asciminib, given by mouth as film-coated granules. The purpose of the study is to see whether asciminib is safe and helpful in these pediatric participants.

Participants will take asciminib during the study and will have regular study visits over time. The study will follow how the disease responds, how long the response lasts, and whether the treatment causes side effects. Growth and development will also be checked, including height or length, weight, bone growth, and puberty changes. The study is planned as a single-group trial, so all participants receive the same study treatment.

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

    Study start

    From the moment you join the study, you begin treatment with asciminib, which is taken by mouth as film-coated granules.

    The study uses asciminib as the only study drug. The dose is 520 mg or 510 mg, taken once daily. The exact dose you receive is the one assigned in the study.

    Your leukemia is one of the following: newly diagnosed ph+ cml-cp, resistant or intolerant to previous tkis (tyrosine kinase inhibitors, a type of cancer medicine), or ph+ cml-cp with the t315i mutation.

    cml-cp means chronic myelogenous leukemia in chronic phase, which is a slower stage of the disease.

  2. Step 2

    Treatment period

    You continue taking asciminib once daily by mouth throughout the study period.

    The study follows your response to treatment over time, including blood-based checks for major molecular response (mmr). mmr means a very strong decrease in the amount of leukemia measured in the blood.

    The study also checks for hematologic response, cytogenetic response, and other molecular responses. These are measures of how the leukemia is responding to treatment in the blood and bone marrow.

    The study records the time to response, how long the response lasts, time to treatment failure, time to disease progression, event-free survival, and overall survival.

  3. Step 3

    Scheduled assessments during the study

    At scheduled timepoints, your response to treatment is checked. The main planned timepoint is week 48, when the study measures major molecular response (mmr).

    The study also measures mmr at week 96 and at other scheduled timepoints.

    Your safety is monitored during the study by recording treatment-emergent adverse events. These are unwanted health problems that start or get worse after treatment begins.

    The study may also check your height or length, weight, bone age by x-ray, and tanner staging. tanner staging is a way to describe body changes during growth and puberty.

    The study measures how your body handles asciminib by checking pk parameters. pk means pharmacokinetics, which describes how the medicine is absorbed, moved through the body, and removed from it. The measured values include auclast, auctau, cmax, tmax, and ctrough.

  4. Step 4

    Study end

    The study continues until the planned end of follow-up for the trial.

    The estimated study end date is 2033-02-23.

Who can join the trial?

11 criteria

  • Written informed consent must be signed before joining the study. This means the parent, guardian, or participant gives formal permission after reading the study information.
  • The participant must be a male or female who is 1 year old and younger than 18 years old at the time of joining the study.
  • The participant must have chronic myeloid leukemia in chronic phase (CML-CP), confirmed by testing of the cells, with a Philadelphia-positive (Ph+) chromosome. This means the leukemia cells must show the specific chromosome change called Philadelphia positive.
  • If the participant is newly diagnosed, the diagnosis must have been made within 3 months before screening. Screening is the set of tests done to see if someone can join the study.
  • If the participant has had previous treatment with a tyrosine kinase inhibitor (TKI), the disease must show resistance or intolerance to that treatment. A TKI is a type of cancer medicine. Resistance means the medicine is not working well enough. Intolerance means the medicine causes side effects that the patient cannot safely continue.
  • For participants with a poor response to a previous TKI, the study requires one or more of the following: BCR::ABL1 level above 10% at 3 months, above 10% at 6 months, above 1% at 12 months, loss of a previous response at any time, or new resistant BCR::ABL1 changes or high-risk additional chromosome changes found on local testing. BCR::ABL1 is a gene marker used to measure the leukemia. Additional chromosome changes are extra changes in the cells that may show higher risk.
  • For participants with TKI intolerance, the study requires either severe non-blood side effects or severe blood-related side effects. Non-blood side effects are side effects not related to blood counts. Blood-related side effects involve low neutrophils or platelets; neutrophils are white blood cells that help fight infection, and platelets help blood clot.
  • For non-blood side effects, the participant must have had grade 3 or grade 4 toxicity while taking the medicine, or ongoing grade 2 toxicity that did not improve with the best possible treatment, including dose changes when appropriate. Toxicity means harmful side effects. Grade 3 or 4 means severe or very severe side effects.
  • For blood-related intolerance, the participant must have had grade 3 or grade 4 low neutrophils or low platelets that came back even after the dose was lowered to the lowest allowed TKI dose.
  • The leukemia cells must show a typical BCR::ABL1 transcript at screening, specifically e14a2 and/or e13a2, and this must be suitable for RQ-PCR testing. RQ-PCR is a lab test that measures the amount of this gene marker very precisely.
  • The participant must have a performance status of at least 50%. This means the participant must be able to do at least half of normal daily activities. For participants 16 years or older, the Karnofsky score must be 50% or higher. For participants younger than 16 years, the Lansky score must be 50 or higher. These are scales that measure how well a person can carry out daily activities.

Who cannot join the trial?

4 criteria

  • Having a known second chronic phase of chronic myeloid leukemia after the disease had already changed to a more advanced stage called accelerated phase or blast phase.
  • Having had a previous hematopoietic stem-cell transplantation, which is a transplant of blood-forming stem cells.
  • Being planned to have an allogeneic hematopoietic stem cell transplantation, which means a stem-cell transplant from another person.
  • Having a known BCR::ABL mutation that is known to resist the study treatment, according to current international treatment guidelines, at any time before joining the study.
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Investigated drugs

  • Asciminib

    is the study medicine being tested in this trial. It is taken by mouth and is being given to children and adolescents with Philadelphia chromosome-positive chronic myelogenous leukemia in the chronic phase. The goal of the study is to see how well it works and how safe it is for patients who are newly diagnosed or who have already been treated before. It is also being studied in patients with or without a known T315I mutation, which can make some treatments less effective.

  • Asciminib hydrochloride

    is the active form of the study medicine used in the trial. It is the same treatment as asciminib and is given by mouth as part of the research to help control the leukemia. The study looks at whether this medicine can help lower or control the disease in pediatric patients, including those whose leukemia has not responded well to earlier treatments.

What is already known about the treatment

  • Asciminib

    Asciminib is an oral medicine taken by mouth, and in this study it is given as film-coated granules for swallowing. It is an approved and well-described medicine in medical literature, mainly used for chronic myeloid leukemia, especially when the disease is newly diagnosed or when other similar treatments have not worked well or caused side effects. It works by blocking a specific switch on the BCR-ABL1 protein that helps leukemia cells grow, so it slows or stops the cancer cells from multiplying. Asciminib is classified as a targeted cancer medicine and a type of tyrosine kinase inhibitor, but it acts in a more selective way than many older drugs in this group.

  • Asciminib Hydrochloride

    Asciminib hydrochloride is the salt form of asciminib and is given by mouth, which makes it a convenient oral treatment. It is a modern, targeted medicine that is already recognized in medical practice and research for treating chronic myeloid leukemia, including cases that are resistant or intolerant to earlier treatments and some cases with the T315I mutation. It works by attaching to a special site on the BCR-ABL1 protein and stopping the abnormal signal that tells leukemia cells to grow and survive. This medicine is classified as a targeted anti-cancer drug and a selective BCR-ABL1 inhibitor.

Investigated diseases

Philadelphia-positive chronic myeloid leukemia, chronic phase - A type of blood cancer that starts in the bone marrow and leads to too many abnormal white blood cells. It usually develops slowly in the chronic phase, where the disease is often more stable and symptoms may be mild or absent at first. Over time, it can become more active and progress to more advanced phases with increasing numbers of abnormal cells. In some cases, specific gene changes such as the T315I mutation are present and may be associated with resistance to previous therapy.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2025-522138-29-00Protocol codeCABL001I12202Estimated enrolment50 patientsSponsorNovartis Pharma AG

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