Targeted therapies for children with refractory or relapsing cancer

E-SMART is an ongoing clinical study with an adaptative design for early-phase study of novel drugs in children:

  • Adaptative design means the study allows for the testing of several drugs simultaneously and that it can be amended to include new drugs without going through the administrative burden of opening a new trial.
  • The tested novel drugs are drugs which are currently in development for the adults and which show promises for paediatric cancers.

This clinical trial is aimed at patients with a cancer that is either refractory or in relapse who have undergone a prior deep molecular analysis (whole exome sequencing and RNA sequencing) through a specific national program (such as iNFORM in Belgium). This prior test allows to identify whether and which targeted therapy is relevant for the patient.

In short, the study will allow all children with whatever type of cancer (solid tumours, leukaemia’s or lymphomas), to access novel targeted therapies rapidly.

This study initially opened in France in 2016 and has now been extended to the Netherlands, UK, Spain, Italy and soon Denmark. Since its opening, 14 treatment arms have been opened, which allowed to test 19 drugs in total (single agent or combination), including 7 for the first time in children. Three new treatment arms are currently in preparation. As of 20 May 2021, 169 patients have been enrolled in the study.

Our financial support will allow the opening of 3 new arms; it covers the equivalent of one year of the structural costs for the coordination and implementation of the study. Each new arm will then require additional financing, which will be cared for by the pharmaceutical company that will have its agent tested in the framework of E-SMART.

Every new drug will be tested with an intent to file, i.e., the plan to submit a registration application for the paediatric indication of the agent. This will ensure that drugs are registered for use in children and used on label.

KickCancer will finance the equivalent of one year of costs of this study or 413.571 €. Our contribution will be spread over four years. Simultaneously, Imagine for Margo (France) and Fondatioun Kriibskrank Kanner (Luxembourg) will also finance the study. If we want to accelerate research for children with cancer, collaboration at a European level is as important for financers, researchers as it is crucial for children with cancer.

In addition, KickCancer will dedicate funds to assist the Belgian patients who need to travel to France or the Netherlands to participate in the study in order to facilitate their participation.

The study is coordinated through the ITCC network (Innovative Therapies for Children with Cancer).

Quality control: this project has been selected for financing in the framework of competitive processes by the INCA (Institut National du Cancer, France) and CRUK (Cancer Research UK), two major well-known organisations in the field of cancer research in Europe.

Targeted therapies for children with refractory or relapsing cancer

  • Financed: 413 571 euros
    More than 85% of your donations go to financing research and supporting our advocacy work, less than 15% go to administration.
  • Duration: 4 years
  • Countries: France, Netherlands, UK, Spain, Italy & Denmark
  • Disease: All paediatric cancers
  • Status: Ongoing

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