EZH2 inhibitor in recurrent or refractory INI-1-negative tumors

"A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma"

This study is aimed at pediatric patients (6 months - 11 years) with INI1-negative rhabdoid tumors, for whom standard therapy has proven to be ineffective (i.e. recurrent or refractory) or incompatible and for which there are no standard curative treatment options. The safety of tazemetostat at different doses is investigated and the so-called "maximum tolerated dose" is determined (expansion phase). It also determines the cancer response to the treatment with tazemetostat and the compatibility of the drug for the patients. The investigational drug tazemetostat inhibits the functioning of the histone methyl transferase EZH2. It has been demonstrated preclinically that the investigational drug slows down or eliminates the growth of cancer cells lacking the INI1 gene. Tazemetostat is taken continuously as a tablet or as a suspension. Tumor response is assessed by MRI every 8 weeks. The study lasts up to 2 years.

The inclusion of patients is possible.

Inclusion criteria for the expansion phase:

  • Age between 6 months and 21 years
  • Rhabdoide tumors of the soft tissue (MRT) or kidneys (RTK)
  • Lansky performance status ≥ 50%

For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (ClinicalTrials.gov).
See NCT02601937 

EudraCT-number: 2015-002468-18 (see EU clinical trial registry)