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

Trial details

Mechanism	Therapy	Manufacturer	Type	Sites	CF Support	Status
Therapeutic vaccine targeting brachyury plus radiation	GI-6301	Celgene	Phase 2	National Cancer Institute	Design, 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	Planning
PD-1 inhibitor plus radiation	Nivolumab	Bristol-Myers Squibb	Phase 1	Memorial Sloan Kettering, Johns Hopkins	Design, Partnerships, Funding, Outreach	Enrolling at Johns Hopkins
Oncolytic bacteria	Not yet disclosed	Not yet disclosed	Phase 1	US institutions TBD	Design, Partnerships, Outreach	Planning
CDK4/6 inhibitor	Not yet disclosed	Not yet disclosed	Phase 2	University of Heidelberg (DE)	Outreach	Planning
Therapeutic vaccine targeting brachyury plus PD-L1 inhibitor	Not yet disclosed	Not yet disclosed	Phase 2	TBD	Design, Partnerships, Outreach	Planning
Autologous tumor vaccine	Not yet disclosed	Not yet disclosed	Phase 2	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 visit chordomafoundation.org/clinical-trials.

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.

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