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ESMART (different tumor types)

Clinical study in children with cancer into the safety and efficacy of drugs matching a specific DNA alteration in the tumor.
Who can enter
  • Children and teenagers with cancer in whom the disease has returned (relapse) or responded insufficiently to treatment (refractory).
  • Age: up to 18 years.


The goal of this study is to assess how safe and effective a drug (or a combination of drugs) is that matches a molecular alteration in the tumor of children with cancer.


Cancer arises by the accumulation of multiple changes in the DNA, also called molecular alterations. These changes can for example result in uncontrolled growth and division of tumor cells, or insufficient sensitivity to chemotherapy. Therefore, more and more drugs are being developed that are specifically aimed at certain molecular alterations in the tumor.

Previously, the iTHER-study determined which alterations are present in the tumor (the so-called ‘molecular profile’) of children with relapsed or refractory disease. When such an alteration is present, treatment with a drug directly targeting this alteration may be possible in certain cases. This is also called ‘precision medicine’ or ‘targeted therapy’. These treatments are part of the clinical study ESMART.

The ESMART study is divided into different treatment options (so-called ‘arms’). In doing so, we try to choose a drug that matches the molecular alteration in the tumor.

The following ‘arms’ have started:

This study was initiated by ITCC, an international collaboration that promotes drug research for children with cancer in Europe.

In order to participate in a study please refer to your/your child’s doctor.

Last reviewed

February 21, 2022