JCAR017-BCM-004

Active, not recruiting

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)

Goal

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.

Background

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.

This study is now closed for inclusion. 

Last reviewed

March 23, 2026

The above information is intended as a brief summary only and may not reflect the most up-to-date information. For full details and the current status of a protocol, physicians can contact the Princess Máxima Center directly.

Summary

Treatment with liso-cel, a form of CAR T-cell therapy, did not work well enough in children with relapsed leukemia. This is evident from international research in which the Princess Máxima Center participated. The study was therefore discontinued. The study did, however, show that treatment with liso-cel is safe in children. The researchers continue to follow the children who participated in this study for long-term effects.

The goal of the JCAR017 study was to find a safe and effective treatment for children and adolescents with B-cell acute lymphoblastic leukemia (B-ALL) or B-cell non-Hodgkin lymphoma (B-NHL). The researchers wanted to see if liso-cel could help children and adolescents with B-ALL or B-NHL in whom the disease had returned after other treatments. They wanted to determine how safe the new treatment is and whether it works well.

Liso-cel

Liso-cel is made from a particular type of white blood cells called T cells. Healthy T cells are collected from the child's blood. In the laboratory, a new gene is placed in the T cells. This new gene allows the child's T cells (now called CAR T cells) to bind to a special protein called CD19. The CAR T cells can now recognize and attack leukemia and lymphoma cells. This type of treatment is called immunotherapy with CAR T cells or CAR T cell therapy. After chemotherapy treatment, the CAR T cells are returned to the child by infusion.

Almost half showed a response

Twenty-one children with B-ALL from different European countries participated in the study. Fourteen of them eventually received liso-cel. In eleven children, researchers were able to determine how well the treatment worked. Almost half (45.5%) of them showed a response to the treatment. There were no leukemia cells visible anymore in any of them, but normal blood cells had not (yet) fully recovered.

Liso-cel also caused side effects. The most common side effects were anemia and the so-called “cytokine-release syndrome” (CRS), an overreaction of the immune system. In CRS, substances are released that can cause fever, low blood pressure and breathing problems. CRS is a common side effect of CAR T cell therapy. Other side effects that were seen were deficiencies in platelets and white blood cells. No side effects made it necessary to stop treatment.

Study discontinued

Through this study, much has been learned about the safety and efficacy of liso-cel in children and adolescents with relapsed B-ALL. However, liso-cel does not seem to work better than existing treatments. Therefore, the study was discontinued earlier than planned. As a result, the best dosage of liso-cel could not be determined. Six of the children who received liso-cel are now being followed for long-term effects.

More information:

Clinicaltrials.gov: NCT03743246 
EU clinical trials registry: 2018-001246-34