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Study on Tisagenlecleucel for High-Risk B-Cell Acute Lymphoblastic Leukemia in Pediatric and Young Adult Patients with Minimal Residual Disease

Verified siteRegistered 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 B-cell acute lymphoblastic leukemia (B-ALL), specifically in children and young adults who are considered high-risk. The treatment being tested is called tisagenlecleucel, which is a form of cell therapy. This therapy involves using a patient's own immune cells, which are modified to better fight cancer cells. The purpose of the study is to evaluate how effective and safe tisagenlecleucel is for patients with high-risk B-ALL who still have some cancer cells remaining after initial treatment.

Participants in the study will receive tisagenlecleucel through an infusion, which means the treatment is given directly into the bloodstream. The study will monitor patients over a period of time to see how well they respond to the treatment and to check for any side effects. The study will also compare the results of those receiving tisagenlecleucel with those who do not, to better understand the treatment's impact on survival and disease progression.

In addition to tisagenlecleucel, the study involves other medications that are commonly used in cancer treatment, such as tocilizumab, cyclophosphamide, cytarabine, etoposide, mercaptopurine, methotrexate, vincristine sulfate, and fludarabine phosphate. These medications may be used to manage symptoms or as part of the treatment process. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatment. The study aims to provide valuable information on the potential benefits and risks of using tisagenlecleucel for treating high-risk B-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

    Enrollment

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, health status, and previous treatments.

    A process called leukapheresis is performed to collect cells from the blood. This is necessary for the manufacturing of the treatment product.

  2. Step 2

    Preparation for treatment

    Before receiving the main treatment, certain medications are administered to prepare the body. These include cyclophosphamide and fludarabine phosphate, both given through an intravenous route.

    This preparation phase helps to create a suitable environment in the body for the main treatment to work effectively.

  3. Step 3

    Main treatment

    The main treatment involves an infusion of tisagenlecleucel, a specialized therapy designed to target specific cells in the body.

    This infusion is administered intravenously and is a critical part of the treatment process.

  4. Step 4

    Monitoring and follow-up

    After the infusion, regular monitoring is conducted to assess the treatment's effectiveness and to check for any side effects.

    This includes various tests and assessments over a period of time to ensure the patient's safety and to evaluate the treatment's impact.

  5. Step 5

    Long-term follow-up

    The study includes a long-term follow-up phase to monitor overall survival and disease-free survival over several years.

    This phase helps to gather important data on the treatment's long-term effects and benefits.

Who can join the trial?

13 criteria

  • The patient must have a type of cancer called CD19 expressing B-cell Acute Lymphoblastic Leukemia, which is found in the blood or bone marrow.
  • The patient should have been diagnosed with de novo NCI HR B-ALL and received first-line treatment, with a specific level of cancer cells remaining, known as MRD ≥ 0.01%, at the end of the first treatment phase.
  • The patient must be between the ages of 1 and 25 years at the time of screening.
  • The patient should have a performance status of at least 60% on a scale that measures their ability to perform daily activities. This is measured by the Lansky scale for those under 16 years and the Karnofsky scale for those 16 years and older.
  • The patient must have adequate organ function, which includes:
  • Renal function: The kidneys should be working well, with specific limits on a substance called serum creatinine based on age and gender.
  • Liver function: The liver should be functioning properly, with specific limits on substances called ALT, AST, and total bilirubin.
  • Pulmonary function: The lungs should be working well, with no or mild breathing difficulty and oxygen levels above 90% in the air.
  • Cardiac function: The heart should be functioning properly, with specific measurements confirmed by an echocardiogram or MUGA scan.
  • The patient may have had certain types of chemotherapy before, but not more than three blocks of standard chemotherapy for first-line B-ALL.
  • The patient must provide signed written consent to participate in the study, and if applicable, assent forms must also be signed.
  • The patient must meet the criteria to undergo a procedure called leukapheresis, which involves collecting certain blood cells.
  • Once all other criteria are confirmed, the patient must have a leukapheresis product of non-mobilized cells that is accepted by the manufacturing site.
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Investigated drugs

Tisagenlecleucel is a type of therapy used in this clinical trial. It is a form of treatment known as CAR-T cell therapy, which involves modifying a patient's own immune cells to better recognize and attack cancer cells. In this trial, it is being tested for its effectiveness in treating pediatric and young adult patients with a specific type of leukemia called B-cell acute lymphoblastic leukemia (B-ALL). The goal is to see if this therapy can improve overall survival and disease-free survival in patients who still have signs of the disease after initial treatment.

What is already known about the treatment

Tisagenlecleucel – This medication is administered as an intravenous infusion and is currently being studied in clinical trials for its effectiveness in treating high-risk B-cell acute lymphoblastic leukemia, particularly in pediatric and young adult patients. It is a form of immunotherapy known as CAR-T cell therapy, which involves modifying a patient's own T-cells to better recognize and attack cancer cells. Tisagenlecleucel is classified as a biologic therapy and works by targeting a specific protein on the surface of cancer cells, leading to their destruction. The medication is still under investigation, but it shows promise in improving overall survival and disease-free survival in patients with minimal residual disease after initial treatment.

Investigated diseases

High-risk B-cell 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 is considered high-risk due to factors such as age, genetic abnormalities, or response to initial treatment, which can influence the disease's progression. The disease typically progresses rapidly, leading to symptoms like fatigue, fever, frequent infections, and easy bruising or bleeding. As the abnormal cells multiply, they crowd out normal blood cells, impairing the body's ability to fight infections and carry oxygen. The disease can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-508081-15-00Protocol codeCCTL019G2201JEstimated enrolment106 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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