Who can enter
- Children and young adults with a large-cell anaplastic lymphoma (ALCL), in whom the disease has returned (recurrence) or who have responded insufficiently to treatment (refractory)
- Children and young adults with difficult to operate inflammatory myofibroblastic tumor (IMT)
- Children and young adults with another tumor in which the ALK gene is found to be abnormal
- Age: 5 to 25 years
The goal of this study is to determine a safe dose of the drug brigatinib in children. Furthermore, we want to investigate how children react to the treatment and what side effects may occur as a result of the treatment.
This study is carried out in children and young adults with a cancer in which the gene called ALK (Anaplastic Lymphoma Kinase) has been found to be abnormal. ALK changes cause normal cells to turn into tumor cells and grow extra fast.
Brigatinib is not a chemotherapy, but a so-called tyrosine kinase inhibitor, also known as targeted therapy. The drug brigatinib specifically blocks the effects of ALK changes in the tumor cell. Laboratory research and studies in adults with tumors with ALK alterations suggest that brigatinib may help to inhibit tumor growth.
Brigatinib has been approved in Europe for the treatment of lung cancer in adults with an ALK gene change, but not yet for children and young adults.
In children with IMT, the standard treatment is surgery to remove the tumor. Sometimes it is not possible to remove the tumor completely, because of its location, its relation to other important structures, or because the tumor is too big. In these children, we want to see whether treatment with brigatinib reduces the size of the tumor, to facilitate a complete removal of the tumor. In children with a different type of tumor where an ALK defect has been identified, we hope that brigatinib will help to combat the disease.
To better understand how the disease responds to brigatinib, we want to store body material (blood, a piece of tumour tissue, and in the case of ALCL also bone marrow) at the Princess Máxima Center for further research. This involves additional samples for which separate permission is asked and residual material that remains after a diagnostic and/or treatment assessment. We request permission to store and use this extra material for scientific research related to the BrigaPED protocol. The aim is to gain a better understanding of how and why an ALK abnormality occurs, and of the effects and side effects of brigatinib. In this way we may be able to further improve the treatment and reduce side effects in the future.
This scientific study was set up and is being carried out in partnership with universities and children's hospitals in Europe. Three European scientific groups on lymphoma (EICNHL), sarcoma (EpSSG), and early phase clinical trials (ITCC) are closely involved in the study design.