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. The Foundation’s goal is to initiate 10 clinical trials by 2020.

Trials pipeline

The table below summarize the clinical trials the Chordoma Foundation is currently supporting:

Mechanism Therapy Manufacturer Type Sites CF Support Status
Therapeutic vaccine targeting brachyury plus radiation GI-6301 Celgene Phase 2 National Cancer Institute Design, Outreach Completed
Therapeutic vaccine targeting brachyury plus
BN-Brachyury Bavarian Nordic Phase 2 Massachusetts General Hospital
Mayo Clinic, Arizona
Mayo Clinic, Florida
Washington University
MD Anderson Cancer Center
Design, Partnerships, Outreach Enrollment complete
Antifolate Pemetrexed Lilly Phase 1 John Wayne Cancer Institute Partnerships, Funding, Outreach Enrolling
CDK4/6 inhibitor Palbociclib Pfizer Phase 2 University of Heidelberg (DE)
University of Ulm
University of Duisburg-Essen
Outreach Enrolling
EGFR inhibitor Afatinib Boehringer Ingelheim 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 Bristol-Myers Squibb Phase 1 Memorial Sloan Kettering, Johns Hopkins Design, Partnerships, Funding, Outreach Enrolling
Autologous tumor vaccine Not yet disclosed Not yet disclosed Phase 2 TBD Design, Partnerships, Outreach Planning
EGFR antibody Not yet disclosed Not yet disclosed Phase 2 US Institutions TBD Design, Partnerships, Funding, Outreach Planning
Oncolytic bacteria Not yet disclosed Not yet disclosed Phase 1 US institutions TBD Design, Partnerships, Outreach Planning


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.

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. Additionally, when a highly promising treatment approach is identified by the Foundation’s Scientific Advisory Board (SAB), the Foundation will proactively recruit investigators to initiate a trial.

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 on the market. 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.

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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