Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

Study on Methotrexate and Dexamethasone for Infants Under One Year with Acute Lymphoblastic Leukemia

Verified siteFast replyRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search on your own, or looking for clinical trials for a rare disease?

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on treating infants under one year old who have been diagnosed with a specific type of blood cancer called acute lymphoblastic leukemia (ALL) or a similar condition known as mixed phenotype acute leukemia (MPAL). The study aims to improve the outcomes for these young patients by using a treatment protocol called Interfant-21. This protocol includes a combination of medications that are commonly used in cancer treatment. These medications include methotrexate, dexamethasone, tioguanine, daunorubicin, vincristine sulfate, mitoxantrone, asparaginase, cyclophosphamide monohydrate, etoposide, mercaptopurine, prednisone, and blinatumomab. Some of these medications are given as injections, while others are taken orally as tablets or suspensions.

The purpose of the study is to see if this new treatment approach can help improve the chances of survival without the cancer coming back. The study will follow the infants through different phases of treatment, which may include receiving some of these medications in a hospital setting. The treatment plan is designed to be as effective as possible while minimizing side effects. The study will also compare the results to previous treatment protocols to see if there is an improvement in outcomes.

Throughout the study, the infants will be closely monitored by healthcare professionals to ensure their safety and to track their progress. The study will collect information on how well the treatment works, any side effects experienced, and the overall health of the infants. This information will help doctors understand more about how to treat this type of leukemia in very young children and may lead to better treatment options in the future.

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 clinical trial, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a series of tests to ensure the patient meets the criteria for participation.

  2. Step 2

    Induction therapy

    The first phase of treatment is called induction therapy. During this phase, several medications are administered to reduce the number of cancer cells. These medications include methotrexate (injection and tablet form), dexamethasone (tablet), daunorubicin (injection), vincristine sulfate (injection), and asparaginase (injection).

    The specific dosage and frequency of these medications are determined by the medical team based on individual patient needs. This phase aims to achieve remission, where no cancer cells are detectable.

  3. Step 3

    Consolidation therapy

    Following induction therapy, consolidation therapy is administered to eliminate any remaining cancer cells. Medications used in this phase include mitoxantrone (infusion), etoposide (infusion), cyclophosphamide (injection), and cytarabine (injection).

    This phase is crucial for preventing the cancer from returning and typically lasts several weeks.

  4. Step 4

    Maintenance therapy

    The final phase is maintenance therapy, which helps to keep the cancer in remission. This involves taking medications such as mercaptopurine (oral suspension), methotrexate (tablet), and prednisone (tablet) over a longer period.

    The goal is to maintain remission and monitor for any signs of cancer returning. Regular follow-up appointments are scheduled to assess progress and adjust treatment as necessary.

  5. Step 5

    Follow-up and monitoring

    Throughout the trial, regular follow-up appointments are necessary to monitor health and response to treatment. These appointments may include physical exams, blood tests, and other assessments.

    The medical team will provide guidance on managing any side effects and ensure the treatment plan is effective.

Who can join the trial?

4 criteria

  • Patients must have a new diagnosis of B-precursor acute lymphoblastic leukemia (ALL) or B-cell mixed phenotype acute leukemia (MPAL). These are specific types of blood cancer.
  • The leukemia must have a specific genetic change called KMT2A-rearrangement.
  • Patients must be 365 days old or younger at the time of diagnosis.
  • Parents or legal guardians must provide written informed consent. This means they agree to let their child participate in the study after being fully informed about it.

Who cannot join the trial?

4 criteria

  • Patients who have a different type of leukemia other than acute lymphoblastic leukemia cannot participate. Acute lymphoblastic leukemia is a type of cancer that affects the blood and bone marrow.
  • Patients who have not been newly diagnosed with KMT2A-rearranged (KMT2A-r) infant acute lymphoblastic leukemia are excluded. This is a specific genetic change found in some leukemia cases.
  • Patients who are not infants are excluded. Infants are typically children under one year of age.
  • Patients who are not part of the vulnerable population selected for this study cannot participate. Vulnerable populations may include groups like infants or those with specific health conditions.
Clinical Trials Concierge

Prefer not to search on your own, or looking for clinical trials for a rare disease?

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

RecruitingNot yet recruitingNot recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Prednisone

    is a medication that helps reduce inflammation and suppresses the immune system. In this trial, it is used to help control the leukemia by reducing the number of cancer cells in the body.

  • Vincristine

    is a type of chemotherapy drug that works by stopping the growth of cancer cells. It is used in this trial to help kill the leukemia cells and prevent them from spreading.

  • Daunorubicin

    is another chemotherapy drug that targets and kills cancer cells. It is used in this trial to help reduce the number of leukemia cells in the body.

  • Cytarabine

    is a chemotherapy medication that interferes with the DNA of cancer cells, preventing them from growing and multiplying. It is used in this trial to help eliminate leukemia cells.

  • Asparaginase

    is a medication that breaks down a nutrient called asparagine, which leukemia cells need to survive. By reducing the availability of asparagine, this drug helps to kill the leukemia cells.

  • 6-Mercaptopurine

    is a chemotherapy drug that interferes with the growth of cancer cells. It is used in this trial to help control the leukemia by reducing the number of cancer cells.

What is already known about the treatment

  • Vincristine sulfate

    Given as an intravenous injection, vincristine is a long‑established chemotherapy drug used to treat various leukemias including acute lymphoblastic leukemia (ALL). It works by binding to tiny structures inside cells called microtubules, stopping them from dividing and spreading. It belongs to the class of Vinca alkaloids, which are plant‑derived anticancer agents. Clinical studies have shown it to be effective as part of combination therapy for ALL.

  • Methotrexate

    This medication can be taken as oral tablets or given by intravenous infusion and is a standard treatment for ALL and other cancers. It blocks an enzyme needed for making DNA building blocks, which slows the growth of rapidly dividing cancer cells. Methotrexate is classified as an antimetabolite chemotherapy drug. It has been widely used for decades and is well documented in medical literature.

  • Blinatumomab

    Administered by continuous intravenous infusion, blinatumomab is a newer, FDA‑approved therapy for certain types of ALL and is also designated as an orphan drug for rare pediatric leukemia. It is a bispecific antibody that connects a T‑cell (immune cell) to a cancer cell, helping the immune system kill the leukemia cells. It belongs to the class of immune‑engaging bi‑specific antibodies. Ongoing trials continue to expand its use in infant ALL.

  • Dexamethasone

    Taken as oral tablets, dexamethasone is a synthetic steroid used widely in ALL protocols to reduce inflammation and help kill cancer cells. It works by turning off genes that protect cancer cells and by dampening the body’s immune response, which can enhance chemotherapy effects. It is classified as a glucocorticoid corticosteroid. Dexamethasone has a long history of safe use in pediatric oncology.

  • Asparaginase

    Given as an intravenous infusion, asparaginase is an enzyme therapy that removes the amino acid asparagine, which leukemia cells cannot make themselves. Without asparagine, the cancer cells starve and die. It is categorized as an enzyme‑based anticancer agent. Asparaginase has been a cornerstone of ALL treatment for many years.

  • Mitoxantrone

    Delivered by intravenous infusion, mitoxantrone is a chemotherapy drug used for ALL and other blood cancers. It inserts itself between DNA strands, preventing the DNA from copying correctly and leading to cell death. It belongs to the class of anthracenedione anticancer agents. It is an established drug with extensive clinical experience.

Investigated diseases

Acute lymphoblastic leukemia – Acute lymphoblastic leukemia 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 begins in the bone marrow, where blood cells are produced, and can quickly spread to the blood and other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. The disease progresses rapidly, leading to symptoms such as fatigue, fever, frequent infections, and easy bruising or bleeding due to the lack of normal blood cells. As the number of lymphoblasts increases, they crowd out normal cells, impairing the body's ability to fight infections and carry oxygen. The progression of the disease can vary, but it typically requires prompt medical attention to manage the rapid growth of abnormal cells.
Trial detailsLast updated 30 Oct 2026
Age0-17PhasePhase IIITrial ID2022-502503-30-00Protocol codeSP-MH20INTEstimated enrolment264 patientsSponsorPrinses Maxima Centrum voor Kinderoncologie B.V.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search on your own, or looking for clinical trials for a rare disease?

Tell us about your condition – we search every trial in Europe and connect you with the right site.