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Study of Ruxolitinib and Chemotherapy Drug Combination for Children with Acute Lymphoblastic Leukemia and JAK/STAT Pathway Activation

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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 cancer called Acute Lymphoblastic Leukemia (ALL) in children. The study involves a treatment that includes a medication called Ruxolitinib and a specific chemotherapy regimen known as AIEOP-BFM 2017 Poland. The purpose of the study is to explore the effectiveness of this treatment combination in children with ALL who have a specific genetic activation known as the JAK/STAT pathway.

Participants in the study will receive Ruxolitinib along with chemotherapy. The chemotherapy includes several medications such as Ifosfamide, Doxorubicin Hydrochloride, Daunorubicin, Asparaginase, Vindesine Sulfate, Methotrexate, Cyclophosphamide, Cytarabine, Dexamethasone, Vincristine Sulfate, Pegaspargase, Mercaptopurine, Etoposide, and Tioguanine. These medications are used to target and kill cancer cells. Some of these drugs are given through an injection into a vein (intravenous), while others are taken by mouth (oral).

The study will follow participants over a period to monitor their response to the treatment. The main goal is to see how many patients achieve a state where no cancer cells are detectable in their blood, known as MRD-negative status. The study will also look at the time it takes for any cancer-related events to occur, such as relapse or resistance to treatment, and will monitor any side effects experienced by the participants. The trial is expected to continue until 2029.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after meeting specific criteria, including a new diagnosis of acute lymphoblastic leukemia (ALL) and confirmed activation of the JAK/STAT pathway.
Eligibility is determined based on genetic markers and risk stratification.
Step 2
Initial treatment phase
Treatment follows the AIEOP-BFM 2017 Poland protocol, which includes a combination of chemotherapy drugs.
Medications such as ifosfamide, doxorubicin hydrochloride, and ruxolitinib are administered through various routes, including intravenous and oral.
Step 3
Consolidation phase
The consolidation phase involves additional chemotherapy to eliminate remaining cancer cells.
Drugs like cytarabine, methotrexate, and vincristine sulfate are used, with administration methods including intravenous and oral.
Step 4
Maintenance phase
The maintenance phase aims to prevent cancer recurrence and involves lower doses of chemotherapy over a longer period.
Medications such as mercaptopurine and methotrexate are taken orally.
Step 5
Monitoring and follow-up
Regular monitoring is conducted to assess the response to treatment and detect any adverse effects.
Follow-up includes measuring minimal residual disease (MRD) to evaluate treatment effectiveness.
Step 6
End of trial
The trial is expected to conclude by August 31, 2029.
Final assessments will determine the overall success of the treatment regimen and any long-term effects.

Who can join the trial?

13 criteria

The patient must have been newly diagnosed with **acute lymphoblastic leukemia (ALL)**, which is a type of cancer affecting the blood and bone marrow.
The patient should be treated according to the **AIEOP-BFM 2017 Poland protocol**, which is a specific treatment plan for ALL.
There must be a confirmed genetic change that causes activation of the **JAK-STAT pathway**. This includes specific genetic markers like **CRLF2, JAK2, EPOR**, or **CRLF2 expression** on the surface of leukemia cells.
The patient should be classified as **early high risk** according to the AIEOP-BFM 2017 Poland criteria. This includes:
- No complete remission by day 33 of treatment.
- Presence of specific genetic markers like **KMT2A-AFF1** or **TCF3-HLF**.
- Having fewer than 45 chromosomes, known as **hypodiploidy**.
- Having a high level of leukemia cells in the bone marrow on day 15, measured as **FCM-MRD** ≥ 10%, and not having certain other genetic markers.
- Presence of **IKZF1plus** and a positive or inconclusive **PCR-MRD** test at a specific time point, without certain other genetic markers.
- A **PCR-MRD** test result of ≥ 5x10^-4 at a specific time point.
- Being younger than 1 year old with any **KMT2A rearrangement**.
- The patient must be between the ages of 2 and 18 years old.
- Both male and female patients are eligible to participate.
- The study includes vulnerable populations, which means it considers patients who might need special protection or care.

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Verified sites

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Kliniczny Szpital Wojewodzki Nr 2 Im. Sw. Jadwigi Krolowej W Rzeszowie
Verified
Rzeszow, Poland
Pomeranian Medical University
Verified
Szczecin, Poland
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
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Lodz, Poland

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Investigated drugs

Ruxolitinib
is a medication used in this trial to treat children with acute lymphoblastic leukemia (ALL) who have a specific activation of the JAK/STAT pathway. It works by blocking certain proteins that are involved in the growth and survival of cancer cells, potentially helping to control the disease.
AIEOP-BFM 2017 Poland Chemotherapy
is a standard chemotherapy regimen used in this trial for treating children with acute lymphoblastic leukemia. This therapy involves a combination of drugs designed to kill cancer cells or stop them from growing and dividing, aiming to achieve remission in patients.

What is already known about the treatment

Ruxolitinib – Ruxolitinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating acute lymphoblastic leukemia (ALL) in children, particularly those with confirmed activation of the JAK/STAT pathway. The main therapeutic indication for ruxolitinib in this context is to improve treatment outcomes in ALL by targeting specific molecular pathways involved in the disease. At the molecular level, ruxolitinib works by inhibiting the Janus kinase (JAK) enzymes, which play a crucial role in the signaling pathways that regulate immune function and cell growth. It is classified pharmacologically as a JAK inhibitor.

Investigated diseases

Acute Lymphoblastic Leukemia – This is a type of cancer that affects the blood and bone marrow, characterized by the overproduction of immature white blood cells, known as lymphoblasts. It typically progresses rapidly, leading to an accumulation of these cells in the bone marrow, which interferes with the production of normal blood cells. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. The rapid progression of the disease requires prompt medical attention to manage its effects on the body.