Exploring the effectiveness and safety of Tisagenlecleucel in B-Cell Acute Lymphoblastic Leukemia treatment

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    What is this study about?

    This study aims to investigate the efficacy of a new treatment called tisagenlecleucel in helping children and young adults combat B-cell Acute Lymphoblastic Leukemia (B-ALL), a high-risk form of blood cancer. The research is conducted across multiple hospitals and involves several stages, including eligibility assessments, treatment preparation, treatment administration, regular check-ups to monitor progress, and long-term follow-up. After receiving tisagenlecleucel, patients will have more frequent hospital visits in the initial month, followed by regular visits every few months for the first two years, and then annually until the study concludes, approximately eight years after the first patient undergoes treatment.

    Learn more about this Trial

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      Locations

      Where does the clinical trial take place?

      Gent , Belgium

      Copenhagen , Denmark

      Helsinki , Finland

      Frankfurt , Germany

      Hamburg , Germany

      Muenchen , Germany

      Monza , Italy

      Roma , Italy

      Utrecht , Netherlands

      Oslo , Norway

      Esplugues De Llobregat , Spain

      Goteborg , Sweden

      Stockholm , Sweden

      London , United Kingdom

      London , United Kingdom

      You can join this study in:

      Denmark: Copenhagen.

      Spain: Esplugues de Llobregat.

      Germany: Frankfurt, Hamburg.

      Finland: Helsinki.

      Italy: Monza.

      Norway: Oslo.

      Sweden: Stockholm.

      Netherlands: Utrecht.


      Study Steps

      How Complex Will My Study Be and What Phases Will I Go Through?

      In this clinical trial, the patient will go through the following sequential phases:

      1. Screening: Initial phase where eligibility is determined based on inclusion/exclusion criteria.
      2. Pre-treatment: Preparatory phase before the actual treatment begins.
      3. Treatment & Follow-up: Administration of the investigational treatment, followed by a series of assessments. Patients will be assessed more frequently in the first month after tisagenlecleucel infusion, then at Day 29, every 3 months for the first year, every 6 months for the second year, and then yearly until the end of the study.
      4. Survival: Monitoring patient survival after the completion of the treatment and follow-up phase.

      Specifically, for treatment, based on the patient’s weight, one of two possible dose ranges of CTL019 (Tisagenlecleucel) will be prepared:

      • For subjects ≤ 50 kg: 0.2 to 5.0 x 10^6 CAR-positive viable T cells per kg body weight.
      • For subjects > 50 kg: 0.1 to 2.5 x 10^8 CAR-positive viable T cells.

      Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study.


      Diseases Under Investigation

      What Conditions Qualify Me for This Study?

      To join the study, you need to have CD19 expressing B-cell Acute Lymphoblastic Leukemia.


      Terms and conditions

      What Requirements I Have to Meet To Join This Study?

      When Am I Eligible to Join the Study?

      The conditions you must meet to join the study are:

      Inclusion Criteria:

      1. CD19 expressing B-cell Acute Lymphoblastic Leukemia: You must have a diagnosis of B-cell Acute Lymphoblastic Leukemia (ALL) that is CD19 positive.
      2. De novo NCI HR B-ALL and MRD ≥ 0.01% at EOC: You should be a newly diagnosed patient with National Cancer Institute High-Risk B-ALL (B-cell Acute Lymphoblastic Leukemia) who has received first-line treatment and has a Minimal Residual Disease (MRD) of equal to or greater than 0.01% at the End of Course (EOC) of treatment. MRD is a measurement of the number of cancer cells that remain in the body during or after treatment, which can be as few as one cancer cell in a million normal cells.
      3. Age 1 to 25 years at the time of screening: Your age should be between 1 to 25 years when you are screened for the study.
      4. Lansky or Karnofsky performance status ≥ 60%:
        • If under 16 years old, a Lansky score of 60% or more — The Lansky Play-Performance Scale measures the functional status of children up to 16 years old. A score of 60% means that you need occasional assistance, but are able to care for most of your needs.
        • If 16 years or older, a Karnofsky score of 60% or more — The Karnofsky Performance Scale measures the ability of cancer patients to perform ordinary tasks. A score of 60% indicates that you require occasional assistance but are able to care for most personal needs.
      5. Adequate organ function:
        • Renal function based on age/gender: Your kidney function should be adequate according to standards adjusted for your age and gender.
        • ALT ≤ 5 times ULN for age: Alanine aminotransferase (ALT), a liver enzyme, should not exceed 5 times the upper limit of normal for your age.
        • AST ≤ 5 times ULN for age: Aspartate aminotransferase (AST), another liver enzyme, should not exceed 5 times the upper limit of normal for your age.
        • Total bilirubin < 2 mg/dL: Your total bilirubin levels in the blood should be below 2 mg/dL, or below 4 mg/dL if you have Gilbert's Syndrome, a condition that affects the way bilirubin is processed.
        • Adequate pulmonary function: You should have no or mild shortness of breath (Grade 1 or lower) and an oxygen saturation level above 90% on room air.
        • Adequate cardiac function: Your left ventricular shortening fraction (LVSF) should be equal to or greater than 28% as confirmed by echocardiogram, or your left ventricular ejection fraction (LVEF) should be 45% or higher as confirmed by echocardiogram or MUGA within 6 weeks of screening.
      6. Prior chemotherapy: You are allowed to have had up to 3 blocks of standard chemotherapy for first-line treatment of B-ALL. This can include a 4-drug induction, BFM consolidation (Berlin-Frankfurt-Münster regimen), and interim maintenance with high-dose methotrexate.

      Please let me know if you need further explanations for any of the medical terms listed.

      What Reasons Could Exclude Me from the Study?

      Here is a list of conditions under which you cannot take part in the study, along with explanations of medical terms when necessary:

      1. M3 marrow at the completion of 1st line induction therapy: You cannot participate if, after the initial phase of leukemia treatment to induce remission, more than 25% of the cells in your bone marrow are leukemia cells.
      2. M2 or M3 marrow or persistent extramedullary disease after first-line consolidation therapy: You cannot participate if, following the first major treatment phase to reduce leukemia cells to undetectable levels, you have a significant number of leukemia cells (M2: 5-25%, M3: >25%) or evidence of leukemia outside the bone marrow.
      3. Philadelphia chromosome positive ALL: You cannot participate if your acute lymphoblastic leukemia (ALL) cells have a specific genetic abnormality called the Philadelphia chromosome.
      4. Hypodiploid: You cannot participate if your ALL cells have less than the normal number of chromosomes (less than 44) or a DNA index lower than 0.81.
      5. Prior tyrosine kinase inhibitor therapy: You cannot have received previous treatment with drugs that inhibit tyrosine kinases, which are enzymes involved in many cellular functions including growth and survival.
      6. Concomitant genetic syndromes associated with bone marrow failure: You cannot participate if you have genetic syndromes such as Fanconi anemia or Kostmann syndrome that affect bone marrow function. Down syndrome does not exclude participation.
      7. Burkitt’s lymphoma/leukemia: You cannot participate if you have Burkitt’s lymphoma/leukemia, which is characterized by certain types of mature B-cell leukemia with specific markers (sIg positive and kappa or lambda restricted positivity) and often associated with a MYC gene translocation.
      8. Prior anti-CD19 therapy: You cannot have been treated with any therapies targeting the CD19 molecule found on the surface of leukemia cells.
      9. Prior gene or engineered T cell therapy: You cannot have been treated with any genetic therapies or engineered T cells, which may interfere with study assessments or carry unknown risks.

      Other conditions defined in the study protocol may also apply.


      Investigational Medicinal Product

      What Products Are Being Used in This Study?

      The drug involved in the study is:

      1. Tisagenlecleucel (Kymriah): It is a chimeric antigen receptor (CAR) T-cell therapy. Essentially, Tisagenlecleucel is made by modifying the patient’s own T cells to include a new gene that codes for a protein called CAR. This protein directs the T cells to target and destroy leukemia cells that have a specific antigen, known as CD19, on their surface.

      Have the Medicinal Substances Used in the Trial Been Previously Studied in Medicine?

      The active substance participating in the clinical trial is known as tisagenlecleucel. Here is a description of this substance and its relevance to the clinical trial:

      1. Tisagenlecleucel: This is a transgene product used in the clinical trial that has been detected by quantitative polymerase chain reaction (qPCR) in target tissue. Tisagenlecleucel comprises CAR-positive viable T cells measured by flow cytometry in target tissue. Various parameters related to this substance are investigated in the trial such as its peak concentration in the blood (Cmax), the time to reach peak concentration (Tmax), the area under the curve (AUC) from time zero to days 29 and 84, and the half-life (T1/2) related to the elimination phase of a concentration-time curve in peripheral blood.

      Tisagenlecleucel is already known to medicine and the medical literature as a CAR T-cell therapy, a type of immunotherapy that has been approved for treating certain types of cancer, such as acute lymphoblastic leukemia (ALL) and diffuse large B-cell lymphoma (DLBCL). It involves genetically modifying a patient’s own T cells to express a chimeric antigen receptor (CAR) that targets cancer cells for destruction.


      Study ID

      CT-EU-00067474

      Recruitment status

      Recruting new patients

      Start of the trial

      5 years ago

      Study phase

      Phase
      II

      Medicinal Product