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Clinical study to evaluate the safety and efficacy of JCAR017 in children with a special type of leukemia (B-ALL) and non-Hodgkin’s lymphoma.
Who can enter
  • Children with B-cell acute lymphoblastic leukemia (B-ALL) or B-cell non-Hodgkin’s lymphoma (B-NHL), in whom the disease has returned (relapse) or did not respond to treatment (refractory)
  • Age: 0 up to 18 years old (in phase 2 possibly also young adults with B-ALL aged 18 up to and including 25 years)


The goal of this study is to define the appropriate dose of JCAR017, and to find out if the JCAR017 T cells are safe and could have beneficial effects for children and young people with leukemia or non-Hodgkin lymphoma after treatment with standard therapy for their disease.


The study drug JCAR017 is an experimental therapy that has not yet been approved for general use by patients. This type of treatment is called immunotherapy with CAR-T cells or ‘CAR-T-cell therapy’. It involves changing the child's own T cells so that they can recognize and attack the tumor cells.

In this study, healthy T cells (a certain type of white blood cells) are collected from the child’s blood. The collected cells are modified in a laboratory: a new gene is inserted into the T cells. This new gene enables the child’s T cells (now called JCAR017-T cells) to recognize a special protein called CD19. They can then bind to this protein. CD19 is present on the surface of leukemia and lymphoma cells of the B-cell type. This modification allows the T cells to go to the leukemia and lymphoma cells and potentially kill them. After a chemotherapy treatment, we return the JCAR017-T cells to the child via infusion.

CAR-T-cell therapy has been tested in a number of different clinical trials, including studies with children and young adults. The first clinical trial using the same JCAR017-T cells as in this study enrolled more than 300 adult patients with non-Hodgkin lymphoma. Results so far suggest significant activity against non-Hodgkin lymphoma with manageable side effects. But it is still too early to know whether there will be any longer-term benefit.

However, with CAR-T cells from another company, results have been published that show that in children with acute lymphoblastic leukemia, in a large number of cases all signs of disease have disappeared (complete remission). There are also children in whom this remission has persisted for several years.

In order to participate in a study please refer to your/your child’s doctor.
For international patients: please feel welcome to contact our International Office. 

Last reviewed

September 6, 2022