Chordoma Foundation


Afatinib in locally advanced and metastatic chordoma


This is a Phase 2 trial studying the effectiveness of a targeted cancer drug called afatinib. Afatinib inhibits the EGFR protein, which is believed to be involved in driving the growth of chordoma tumors. This study is designed specifically for chordoma patients 18 years or older with recurrent or metastatic tumors. It is enrolling patients at Leiden University Medical Center (LUMC), University College London Hospital (UCLH), and Istituto Nazionale dei Tumori (INT) in Milan in the coming months. The principal investigators for this study are Dr. Hans Gelderblom at LUMC, Dr. Silvia Stacchiotti at INT, and Dr. Sandra Strauss at UCLH.

Why this trial is being done

Epidermal Growth Factor Receptor (EGFR) is a protein found on the surface of certain cells throughout the body. Ordinarily, EGFR helps to regulate cell growth and plays a role in wound healing. In certain cancers, including most chordomas, EGFR becomes overactive, leading the cancer cells to multiply out of control.

Drugs that block EGFR called “EGFR inhibitors” are approved to treat several different types of cancer. Afatinib is an EGFR inhibitor that is currently approved to treat non-small-cell lung cancer and is being tested in other tumor types.

Several EGFR inhibitors have been shown to slow or stop the growth of chordoma cells and chordoma tumors in mice. Of all the EGFR inhibitors tested, afatinib was the most effective in mouse models of chordoma. In some mouse models, it slowed the growth of the tumors, while in others it caused the tumors to shrink considerably.

This trial aims to determine whether afatinib can shrink or stop the growth of chordoma tumors in patients with recurrent or metastatic disease.

Who can participate

This trial is designed specifically for chordoma patients whose tumors are not operable, have come back in the same area after previous treatment, or have spread to other parts of the body. 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.

  • Confirmed diagnosis of a chordoma tumor that expresses the EGFR protein
    (Note: Expression of EGFR protein is different than mutations in the EGFR gene. The EGFR protein is often highly expressed in chordoma tumors, whereas EGFR gene mutations are almost never found in chordomas. This means that patients can be eligible for this trial even if EGFR mutations are not found in molecular profiling tests such as FoundationOne. Expression of the EGFR protein will be determined by a pathologist at one of the trial sites.)
  • Recurrent, non-operable, or metastatic disease confirmed by imaging
  • Progression of disease documented in last 6 months
  • No major surgery less than 4 weeks before beginning treatment
  • Age 18 years or older
  • 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 will take a 40 mg oral dose of afatinib daily. This will continue in 4-week cycles until there is tumor growth or the participant leaves the trial.
  • Check-up exams will be performed every 4 weeks and imaging done every 12 weeks.
  • All exams, treatments, and evaluations are provided at the trial site where the patient enrolled in the study. Participants not local to the trial sites MUST be willing and able to travel to their trial site for all visits required by the trial.

How to enroll in this trial

The trial team will see all potential patients in a clinic visit before beginning the screening process. See the FAQ below, “How do I find out if I’m eligible to participate?”, for more specific information on enrolling.



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.

Contact a CF Patient Navigator

Learn more about targeted therapy


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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