Chordoma Foundation

Clinical Trials Program

The Chordoma Foundation Clinical Trials Program supports well-justified clinical trials designed to determine whether, and to what extent, promising new therapies can improve the lives of chordoma patients. The purpose of this program is to eliminate the barriers that typically hinder clinical trials for patients with rare cancers, ensuring that scientifically-sound treatment approaches are tested in chordoma patients as quickly as possible.

Mechanisms of support

Based on the individual needs of each clinical trial in the pipeline, the Foundation provides support for clinical trials in the following four ways:

  • DESIGN: Ensure trials are designed appropriately for the chordoma patient population by providing input from patients and experts on the Foundation’s Medical Advisory Board (MAB), and representing the interests of chordoma patients in dialogue with regulatory agencies.
  • PARTNERSHIPS: Broker relationships between investigators and industry sponsors, and help to recruit participating trial sites.
  • FUNDING: Provide grants to support investigator-initiated proof of concept trials and associated correlative studies.
  • OUTREACH: Work with the trial sponsor to develop and distribute announcements and educational materials about the trial to patients, caregivers, and healthcare providers.

Trial selection

The Foundation invites trial concept proposals from companies and academic investigators on an ongoing basis. To provide patients with the greatest chance of benefitting from clinical trials and to ensure judicious investment of resources, the Foundation only supports clinical trials that have strong scientific rationale and that are designed appropriately for the chordoma patient population. Clinical trials supported by the Foundation are vetted and approved by the Foundation’s MAB and SAB based on their scientific and clinical merits.

The Foundation supports trials to repurpose therapies already approved for other diseases, as well as novel therapies that are not yet approved. These may include targeted therapies, immunotherapies, radiation, combination therapies, or other promising approaches.

Over time, the Foundation aims to facilitate trials relevant to all segments of the chordoma patient population, including adults, children, and individuals with skull base, spine, and sacral tumors.

Trials pipeline

The table below summarize the clinical trials supported by the Chordoma Foundation:

Mechanism Therapy Type Sites CF Support Status
Therapeutic vaccine targeting brachyury plus radiation GI-6301 Phase 2 National Cancer Institute Design, Outreach Completed
Therapeutic vaccine targeting brachyury plus radiation BN-Brachyury Phase 2 Massachusetts General Hospital, Mayo Clinic, Washington University, MD Anderson Cancer Center Design, Partnerships, Outreach Completed
Antifolate Pemetrexed Phase 1 Saint John’s Cancer Institute Partnerships, Funding, Outreach Enrolling
CDK4/6 inhibitor Palbociclib Phase 2 University of Heidelberg (DE), University of Ulm (DE), University of Duisburg-Essen (DE) Outreach Enrolling
EGFR inhibitor Afatinib Phase 2 University of Leiden (NL), Istituto dei Tumori (IT), University College London (UK) Design, Partnerships, Funding, Outreach Enrolling
PD-1 inhibitor plus radiation Nivolumab Phase 1 Memorial Sloan Kettering, Johns Hopkins Design, Partnerships, Funding, Outreach Enrolling
EGFR antibody Cetuximab Phase 2 MD Anderson Cancer Center Design, Partnerships, Funding, Outreach Enrolling

The list above includes only trials that the Chordoma Foundation is actively supporting. For a complete list of trials open to chordoma patients view our Clinical Trials Catalogue

Stages explained:

  • Planning: Concept developed; completing development of trial protocol, commitment of drug supply, regulatory approval, ethics approval, and site initiation.
  • Enrolling: Trial is open and enrolling new patients who meet eligibility criteria.
  • Ongoing: Enrollment is complete. Patients remain on the trial receiving treatment or monitoring.
  • Evaluation: Trial is complete; results are being analyzed.

More information

For patients For investigators For donors
Find all trials currently open to chordoma patients in our Clinical Trials Catalogue, or contact a CF Patient Navigator for assistance Contact Josh Sommer at to discuss support for a trial concept or to request a proposal form Contact to learn about opportunities to sponsor clinical trials


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