The Clinical Cooperation Unit Pediatric Oncology together with the Department of Pediatric Oncology, Hematology and Immunology of the University Hospital Heidelberg is preparing several clinical trials for the treatment of children and adolescents with malignant diseases. The concepts of the studies are based on the current knowledge of novel molecular targets in pediatric oncology and neuro-oncology.




Phase I/II vorinostat

© dkfz.de

A phase I/II trial for the treatment of children with recurrent malignant diseases with the oral histone deacetylase (HDAC) inhibitor vorinostat is open and recruiting patients since May 2012 (http://clinicaltrials.gov/ct2/show/NCT01422499). We and other groups have shown that HDACs propagate growth and survival of many pediatric cancer cells, while inhibiting diffentiation and programmed cell death. The targeted inhibition of HDACs could therefore be a novel therapeutic strategy in the treatment of pediatric tumors. Children and adolescents (3-18 years of age) who did not respond to standard first or second line therapy, including patients with recurrent solid tumors, lymphomas or leukemias, can be treated in this trial. The patients are treated according to an intra-individual dose escalation concept in 10 centers throughout Germany. The trial is funded by the German Children´s Cancer Foundation (Deutsche Kinderkrebsstiftung), the drug vorinostat is being supplied by MSD Sharp & Dohme GmbH. Small children will receive individually prepared oral drug suspensions. A synopsis can be downloaded here.
For inquiries, please contact Professor Olaf Witt (o.witt@dkfz.de).

Phase III Everolimus for the treatment of subependymal giant cell astrocytoma (EXIST1)

© dkfz.de

The phase III, placebo-controlled, randomized study of Everolimus (RAD001) for the treatment of patients with tuberous sclerosis (TSC) and subependymal giant cell astrocytoma (SEGA) has closed its enrollment. After positive evaluation of efficacy and safety of everolimus, the study has now entered its 4 year extension phase since March2, 2011. Patients with tuberous sclerosis have a mutation of the TSC1 or the TSC2 gene. This leads to permanent activation of the PI3K/AKT/mTOR-signalling pathway in the cells, in turn promoting uncontrolled proliferation of different cell types in the patient´s body. Everolimus is a specific inhibitor of mTOR, and may constitute a novel therapy for the treatment of SEGAs, as well as for other symptoms of TSC. The study has recruited patients with a diagnosed TSC showing a progression of a SEGA as documented by MRI. This study is being conducted worldwide and sponsored by Novartis. The principal investigator for Germany is Professor Olaf Witt.
For inquiries, please contact Professor Olaf Witt (o.witt@dkfz.de).

EFFECTS-study

This phase III expanded access study recruits patients with tuberous sclerosis (TSC) and subependymal giant cell astrocytoma (SEGA) has now closed recruitment.  For background and rationale see EXIST1- trial. The study is sponsored by Novartis. The principal investigator for Germany is Professor Olaf Witt.
For inquiries, please contact Professor Olaf Witt (o.witt@dkfz.de)

to top