Chordoma Foundation


Investigational EZH2 inhibitor in chordoma


This is a Phase 2 trial evaluating the safety and efficacy of tazemetostat, a targeted cancer investigational agent. Tazemetostat inhibits the enhancer of zeste homolog 2 (EZH2) enzyme, which is believed to be involved in driving the growth of certain chordoma tumors. This trial is designed for patients ages 18 years or older whose tumor cells have lost expression of the protein INI1 and it includes a group, or “cohort”, of the trial that is specifically enrolling chordoma patients. The trial is enrolling patients at multiple locations around the world. See for a list of these locations.

Why this trial is being done

INI1, also called SMARCB1, is a protein that helps to suppress the growth of tumor cells. When INI1 protein expression is lost in tumor cells, dependency on the EZH2 enzyme increases, causing cancer cells to multiply out of control.

Tazemetostat is the first investigational agent of its kind, called an EZH2 inhibitor, that is designed to block the EZH2 enzyme. Tazemetostat is not yet approved to treat any type of cancer, but it is currently being studied in several clinical trials for different cancers.

This trial is potentially relevant to chordoma patients because one type of chordoma, called poorly differentiated chordoma, is generally associated with loss of INI1 protein expression. Loss of INI1 protein expression within the tumor may make this tumor type more susceptible to EZH2 inhibitors such as tazemetostat. Conventional and dedifferentiated chordomas may have INI1 loss as well.

This trial aims to determine the safety of tazemetostat and whether it may be able to shrink the growth of tumors in patients with INI1-negative tumor cells.

Who can participate

The chordoma-specific cohort of this Phase 2 trial is designed specifically for chordoma patients whose tumors have lost expression of the INI1 protein. Patients who meet the following criteria may be eligible to participate and should talk to their treating physician about whether this trial is right for them.

  • Age 18 years or older
  • Confirmed diagnosis of chordoma
  • Loss of INI1 confirmed by immunohistochemistry or molecular testing
  • Sufficient amount of time elapsed since the end of other treatments (chemotherapy, immunotherapy, radiation, etc.)
  • No major laboratory abnormalities
  • Willing and able to travel to the enrolling study site for all treatment and follow-up visits

Full eligibility criteria can be found at

How the trial will work

  • Participants take an 800 mg oral dose of tazemetostat (four 200 mg tablets) twice a day continuously for 28 days, which constitutes one cycle. Patients must check in with the trial site at the end of each cycle to receive their next 28-day supply of the drug.
  • Check-up exams will be performed every 2 weeks for the first 8 weeks of the trial and every 4 weeks after that.
  • All exams, treatments, and evaluations will be provided at the trial site where the patient is enrolled in the study. Participants not local to the trial sites MUST be willing and able to travel to their trial site at their own expense for all visits required by the trial.

How to enroll in this trial

If you are interested in enrolling, talk with your doctor to determine whether this clinical trial might be appropriate for you. The team at the trial site will need to review your medical history in order to determine if you are eligible to enroll. See the FAQ below, “How do I find out if I’m eligible to participate?”, for more specific information on the enrollment process.



If you have more questions about this trial or other clinical trials that may be options for chordoma patients, please contact a Chordoma Foundation Patient Navigator.

Further reading


Medical information provided by the Chordoma Foundation in any medium is for informational and educational purposes only. After reading content from the Chordoma Foundation, review the information carefully with your physician. Information from the Chordoma Foundation is not a substitute for consultations with qualified health care professionals who are familiar with your medical conditions and needs. The Chordoma Foundation strongly recommends that care and treatment decisions be made in consultation with your physician or other qualified health care professionals who are familiar with your specific health situation.

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