Chordoma Foundation

Nivolumab + stereotactic radiosurgery

Clinical trial of nivolumab with or without stereotactic radiosurgery in treating patients with recurrent, advanced, or metastatic chordoma


Doctors at Johns Hopkins Hospital are enrolling chordoma patients in a Phase 1 clinical trial studying the safety and initial effectiveness of the immunotherapy drug nivolumab with and without stereotactic radiosurgery (SRS). The goal of these therapies is to harness the body’s own immune system to more effectively fight chordoma.

This research study is designed specifically for chordoma patients 15 years or older with recurrent or metastatic tumors. The primary goal of this research study is to determine the safety of nivolumab alone or in combination with SRS for treating chordoma. The secondary goal is to determine whether these therapies can shrink or stop the growth of chordoma tumors. Dr. Lawrence Kleinberg is the Principal Investigator at Johns Hopkins Hospital.

Why this trial is being done

The immunotherapy drug nivolumab helps the immune system destroy tumor cells by blocking proteins called immune checkpoint molecules that cancer cells use to avoid attack by the immune system. Known as a “checkpoint inhibitor,” nivolumab is currently approved to treat several tumor types including melanoma, kidney cancer, lung cancer and bladder cancer. Recent laboratory research has shown that chordomas produce the same checkpoint molecules as these cancers, suggesting that a checkpoint inhibitor such as nivolumab might also be able to help the immune system more effectively fight chordoma. This research study is the first to test a checkpoint inhibitor for the treatment of chordoma.

One group of participants in this study will receive nivolumab alone, which is intended to stimulate the immune system to attack the tumor. A second group will receive nivolumab in combination with stereotactic radiosurgery (SRS). SRS, which delivers a high dose of focused radiation to a tumor over a short period of time, is already a standard treatment that is used to help control advanced or metastatic chordoma tumors (it is sometimes called CyberKnife or Gamma Knife, which are the names of machines used to deliver the radiation). Doctors believe SRS triggers an immune response by causing tumor cells to burst, exposing their abnormal contents to the immune system. It may also make tumor cells more sensitive to being killed by the immune system.

This understanding has provided rationale for testing the combination of checkpoint inhibitors with SRS. Clinical trials testing this combination in other tumor types have shown preliminary evidence of enhanced activity against the tumor compared with SRS alone or a checkpoint inhibitor alone. More recently, researchers at Johns Hopkins University showed that this combination reduces tumor growth and improves survival in a mouse model of chordoma. Taken together, this evidence provides strong rationale for a clinical trial testing nivolumab alone and in combination with SRS.

Who can participate

This study is designed specifically for chordoma patients whose tumors have come back in the same area or have spread to other parts of the body. Patients must meet the following basic criteria to be eligible:

  • Confirmed diagnosis of chordoma
  • Recurrent or metastatic disease confirmed by imaging (cannot be newly diagnosed, primary chordoma)
  • Age 15 years or older
  • No major laboratory abnormalities
  • No prior use of an anti-PD-1, anti-PD-L1, or anti-PD-L2 antibody
  • Eligible to receive radiation to site of tumor or metastasis
  • Willing and able to travel to the study site (Johns Hopkins Hospital in Baltimore) for all treatment and follow-up visits

Full eligibility criteria can be found on

How the trial will work

  • Participants are assigned to one of two treatment groups:
    • Participants in the first group will receive nivolumab by IV over 30 minutes on day 1, and again every 2 weeks for about 4 months (8 doses). Beginning with dose 9, participants will receive nivolumab every 4 weeks at a higher dose.
    • Participants in the second group will receive the same schedule of nivolumab treatments, plus stereotactic radiosurgery treatment on day 8.
  • The trial doctor will determine during the enrollment process which group is the best fit for each new participant.
  • A check-up exam will be performed at each visit, before participants receive the scheduled nivolumab treatments.
  • Patients can remain on the trial up to 2 years, or until there is disease progression or unacceptable side effects are experienced.
  • Participants will be evaluated for side effects for 100 days after stopping treatment. MRIs will be performed approximately every 10 weeks for 2 years, as part of standard follow-up.
  • 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 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.

Contact a CF Patient Navigator

eIRB application number: IRB00117650

Learn more about immunotherapy

How is Immunotherapy Used to Fight Cancer? From Dana-Farber Cancer Institute and Science Illustrated

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PD-1/PD-L1 Pathway: A Security Checkpoint

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Information from Bristol-Myers Squibb, the maker of nivolumab


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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