Who can enter
- Children and adolescents with advanced or metastatic infantile fibrosarcoma, a brain tumor, or another solid tumor in whom the disease has returned (relapsed) or responded insufficiently to treatment (refractory). They must have a documented NTRK gene abnormality in the tumor
- Age: up to 21 years
Goal
The goal of this study is to evaluate the safety and efficacy of larotrectinib in children and adolescents. We will assess which side effects occur due to the treatment, and to what extent and how long the tumor responds to the treatment.
Background
NTRK gene abnormalities are rare, and occur in different tumor types, both in children and adults. Among others, this abnormality has been found in children with infantile fibrosarcoma, some brain tumors, thyroid carcinoma, and certain forms of melanoma.
The NTRK gene abnormality results in the production of an aberrant TRK protein, a so-called tyrosine kinase. This enables tumor cells to grow and proliferate. The drug larotrectinib selectively inhibits the TRK protein. This type of drug is called a tyrosine kinase inhibitor (TKI).
In previous clinical studies larotrectinib was found to be safe, and resulted in a good response in many patients. Based on these results, larotrectinib has been granted accelerated approval for the treatment of adults and children with solid tumors carrying an NTRK gene abnormality, who have no other suitable treatment options. However, additional data are needed to be able to properly assess the efficacy and safety.