Who can enter
- Children and adolescents with B-cell acute lymphoblastic leukemia (B-ALL) with a high risk of recurrence
- Age: 1 up to 25 years
The goal of this study is to find out how safe and effective the new tisagenlecleucel treatment is for children and adolescents aged 1 up to 25 years with a high-risk B-ALL.
Children and adolescents who are diagnosed with B-ALL are treated according to the ALLTogether or ALL-11 treatment protocol. At the end of the first blocks of chemotherapy, sometimes minimal residual disease is still present (i.e., some leukemic cells can still be detected in the bone marrow). These children have a high risk of recurrence. In this study we test a new way of treating children with high-risk B-ALL: using CAR T-cells (a form of immunotherapy).
To do so, T cells (certain white blood cells) will first be collected from the child's blood. This process is called apheresis. The genetic characteristics of these T-cells are then changed in the laboratory so that they can recognize and destroy the leukemic cells. The genetically altered cells, called CAR T-cells, are returned to the child via an infusion. CAR T cells from the Novartis company are called tisagenlecleucel.
Tisagenlecleucel has been approved (“registered”) by the Dutch authorities as a drug for the treatment of advanced B-ALL in children and adolescents up to 25 years old and for a certain type of lymphoma in adults. When the medication is given to children for whom no other treatment options are available, more than 80 percent enter remission and approximately 65 percent of the children are still free of leukemia after two years. In children with an earlier stage of the disease doctors are not yet allowed to prescribe this medication. That is why this study is needed.