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Study of Ruxolitinib and Venetoclax for Children with Relapsed or Refractory Leukemia or Lymphoma with IL-7R/JAK-STAT Pathway Mutations

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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 with certain types of blood cancers, specifically Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma, which have either returned after treatment or have not responded to previous treatments. The study involves two medications: Venetoclax, also known by its code name ABT-199, and Ruxolitinib, which is sometimes referred to as INCB018424. These medications are being tested for their effectiveness in treating these cancers, particularly in cases where there are specific genetic changes in the IL-7R/JAK-STAT signaling pathway, which is a part of the body's cell communication system that can affect cancer growth.

The purpose of the study is to evaluate the safety and effectiveness of these medications in children. Participants in the study will receive either Venetoclax or Ruxolitinib, or a combination of both, in the form of oral tablets or suspensions. The study will be conducted in two phases. The first phase will focus on determining the safest dose of the medications, while the second phase will assess how well the medications work in treating the cancers. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the actual drugs.

Throughout the study, participants will be closely monitored by healthcare professionals to ensure their safety and to observe any changes in their condition. The study aims to provide valuable information on the potential benefits of Venetoclax and Ruxolitinib for children with these challenging types of blood cancers. The study is expected to continue until 2031, with recruitment starting in 2024.

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 assessed to ensure they meet the eligibility criteria. This includes age, ability to swallow tablets, and specific health conditions related to leukemia or lymphoma.

    The patient must have had prior molecular profiling of their disease to identify specific genetic alterations.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to evaluate the patient's overall health, including organ function and cardiac health.

    This assessment ensures the patient is fit to proceed with the trial.

  3. Step 3

    Medication administration

    The patient will receive two medications: venetoclax and ruxolitinib. Both are administered orally.

    The specific dosage and frequency will be determined based on the patient's condition and response to treatment.

  4. Step 4

    Monitoring and evaluation

    Throughout the trial, the patient's response to the medication will be closely monitored.

    Regular evaluations will be conducted to assess the effectiveness and any side effects of the treatment.

  5. Step 5

    Adjustments and continuation

    Based on the patient's response, adjustments to the medication dosage may be made to optimize treatment outcomes.

    The trial will continue with ongoing monitoring and adjustments as necessary.

  6. Step 6

    Completion of the trial

    The trial is expected to continue until 2031, with the patient's participation duration depending on individual response and health status.

    Upon completion, a final assessment will be conducted to evaluate the overall impact of the treatment.

Who can join the trial?

7 criteria

  • Children must be between 1 year (at least 12 months) and 18 years old at the time of first diagnosis and less than 21 years old at the time of joining the study. They must also be able to swallow tablets.
  • The child must have a certain level of activity or energy, measured by a score called the Karnofsky performance status (for those older than 12 years) or the Lansky Play score (for those 12 years or younger), which should be at least 50%.
  • Written permission must be given by the parents or legal representative, the patient, and the patient if they are old enough to understand, before any study-related tests are done. This must follow local, regional, or national rules.
  • The patient must have had detailed testing of their disease using advanced methods before joining the study. This includes looking at the disease at a molecular level and using a technique called flow-cytometric analysis. Additional testing is recommended but not required. If the patient had these tests at first diagnosis but not at relapse or when the disease returned, they might still join after discussing with the study organizers.
  • The patient's tumor must show specific changes in certain pathways, like the IL-7R and JAK-STAT signaling pathways. These changes include, but are not limited to, certain rearrangements, mutations, or overexpression in genes like CRLF2, EPOR, JAK1/2/3, IL-7R, SH2B3, USP9X, STAT5B, DNM2, and PTPN2.
  • The patient must have proper organ function, which includes:
    • Kidney and liver function: The levels of certain substances in the blood, like serum creatinine and bilirubin, must be within specific limits. For example, serum creatinine should be no more than 1.5 times the normal upper limit for their age, or their kidney function should be at least 60 mL/min/1.73 m². Bilirubin should be no more than 2 times the normal upper limit, or 3 times for those with Gilbert’s syndrome. Liver enzymes like ALT and AST should be no more than 5 times the normal upper limit. If liver problems are due to the disease, the patient might still be eligible after discussion with the study organizers.
    • Heart function: The heart's ability to pump blood, measured by shortening fraction (SF) or left ventricular ejection fraction (LVEF), should be above certain levels. SF should be more than 29% (or more than 35% for children under 3 years), and LVEF should be at least 50%. There should be no significant heart rhythm problems, like QTcF prolongation, which is a specific measure on an ECG test.

Who cannot join the trial?

10 criteria

  • Patients who have a different type of cancer than the ones being studied cannot participate.
  • Patients who are not in the age range specified for the study cannot participate.
  • Patients who are not able to follow the study procedures or instructions 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 had another cancer treatment recently that might affect the study cannot participate.
  • Patients who are allergic to the study drugs or their ingredients cannot participate.
  • Patients who have an infection that is not well controlled cannot participate.
  • Patients who have a history of certain heart problems cannot participate.
  • Patients who are taking medications that might interfere with the study drugs cannot participate.
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Investigated drugs

Investigational Agents are new drugs being tested in this trial to see if they are safe and how well they work for children with certain blood cancers that have come back or are not responding to treatment. These agents are specifically chosen because they might work well against certain changes in the cancer cells. The trial aims to find out the best dose that can be given safely and to see if these drugs can help treat the cancer effectively.

What is already known about the treatment

Investigational Agent – This medication is administered intravenously, allowing it to directly enter the bloodstream for rapid action. It is currently in clinical trials, specifically in Phase I and Phase II, to evaluate its safety, tolerability, and effectiveness in treating certain types of relapsed or refractory blood cancers in children. The main therapeutic indications include acute lymphoblastic leukemia and lymphoblastic lymphoma, both in recurrent and refractory forms. At the molecular level, the drug targets specific genetic alterations in cancer cells, disrupting their growth and survival. It is classified pharmacologically as an antineoplastic agent, which means it is designed to prevent the growth and spread of cancer cells.

Investigated diseases

  • Acute Lymphoblastic Leukemia, in Relapse

    This is a type of cancer that affects the blood and bone marrow, characterized by the overproduction of immature white blood cells called lymphoblasts. In relapse, the disease returns after a period of improvement or remission. The abnormal cells can crowd out normal cells, leading to symptoms like fatigue, fever, and increased risk of infections. The disease progresses as these cancerous cells multiply rapidly, affecting the body's ability to produce healthy blood cells. Over time, it can spread to other parts of the body, including the lymph nodes, liver, and spleen.

  • Lymphoblastic Lymphoma (Precursor B-Lymphoblastic Lymphoma/Leukemia) Recurrent

    This is a type of non-Hodgkin lymphoma that originates from immature B-lymphocytes. In its recurrent form, the disease returns after treatment. It typically presents as a rapidly growing mass, often in the chest area, and can spread to other organs. The disease progresses as the abnormal lymphocytes multiply uncontrollably, potentially leading to symptoms such as swelling of lymph nodes, fever, and weight loss. It can also affect the bone marrow and central nervous system.

  • Lymphoblastic Lymphoma (Precursor T-Lymphoblastic Lymphoma/Leukemia) Recurrent

    This is a type of aggressive non-Hodgkin lymphoma that arises from immature T-lymphocytes. When recurrent, the disease reappears after initial treatment. It often presents with a large mass in the chest and can spread to the bone marrow and central nervous system. The disease progresses as the cancerous T-cells proliferate rapidly, causing symptoms like chest pain, breathing difficulties, and swollen lymph nodes. It can also lead to complications due to the spread of cancerous cells.

  • Lymphoblastic Lymphoma (Precursor B-Lymphoblastic Lymphoma/Leukemia) Refractory

    This condition refers to a type of non-Hodgkin lymphoma that does not respond to standard treatments. It originates from immature B-lymphocytes and can present as a rapidly growing mass. The disease progresses as the abnormal cells continue to multiply despite treatment efforts, leading to symptoms such as swollen lymph nodes, fever, and fatigue. It can spread to other parts of the body, including the bone marrow and central nervous system, complicating the clinical picture.

  • Lymphoblastic Lymphoma (Precursor T-Lymphoblastic Lymphoma/Leukemia) Refractory

    This is a form of aggressive non-Hodgkin lymphoma that does not respond to conventional therapies. It arises from immature T-lymphocytes and often presents with a large mass in the chest. The disease progresses as the cancerous cells continue to grow and spread, causing symptoms like chest pain, difficulty breathing, and swollen lymph nodes. It can also affect the bone marrow and central nervous system, leading to further health complications.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2022-501867-42-01Protocol codeHEM-iSMART sub CEstimated enrolment25 patientsSponsorPrincess Maxima Center For Pediatric Oncology

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).