Clinical Trials Catalogue
Clinical trials are carefully conducted research studies meant to determine the safety and effectiveness of a certain treatment. They are the way to know whether new treatments work so they can be approved to treat the disease.
For chordoma patients, some clinical trials may provide an opportunity to receive new, experimental therapies that could be more effective than currently available options. Other clinical trials may provide an opportunity for patients to gain access to clinically available therapies at no cost. In general, care provided through a clinical trial is very methodical and closely monitored by a team of healthcare professionals.
In addition to having the potential to benefit from the latest treatments, chordoma patients who participate in clinical trials contribute to knowledge that can guide treatment for future patients and potentially help identify new ways of treating this rare cancer.
Learn more about clinical trials »
Medical Advisory Board recommendations Although each patient’s situation is unique, in general, our Medical Advisory Board (MAB) recommends that patients considering systemic therapy treatment options do so in the following order of priority, in consultation with a chordoma-experienced medical oncologist: 1. Chordoma-specific clinical trials
Start by considering trials designed specifically for chordoma patients. These trials are likely to have strong scientific justification and be conducted by teams with significant experience caring for chordoma patients. 2. Other relevant clinical trials recommended by an experienced physician
If you are not eligible for any chordoma-specific trials, ask your oncologist about other clinical trials that may be open to you. 3. Off-label therapy with evidence of clinical benefit for chordoma patients
While there are currently no drugs approved for the treatment of chordoma, some drugs that are approved for the treatment of other cancers have shown modest activity in chordoma patients. In some countries, doctors can choose to prescribe these drugs to chordoma patients if they believe they will benefit the patient. This is called off-label use. If you are not eligible for any clinical trials, consult with your oncologist about off-label use of approved drugs that have been used to treat chordoma patients. Read more about systemic therapy for chordoma » Visit our Systemic Therapy Table » |
Below is a list of clinical trials designed specifically for chordoma patients or with chordoma-specific arms (in blue boxes), as well as other relevant trials recommended by the Chordoma Foundation’s Medical Advisory Board, based on scientific rationale (in grey boxes).
How to use the Clinical Trials Catalogue:
- We encourage patients who are considering clinical trials to show this list to their doctors to help guide discussions about trial options.
- Click on the plus signs (+) at the left of the trial listings for additional information about each trial, including links to clinical trial databases like ClinicalTrials.gov where you can find further information such as eligibility criteria.
- The chordoma-specific trials noted with the white Chordoma Foundation icon include links to FAQs pages we’ve created to provide you with more in-depth information about each trial.
 Questions to ask about treatment
Before enrolling in a clinical trial, it is important to understand and carefully evaluate the potential risks and benefits and to discuss your options with your doctors. To help you think about what you want to ask your doctors and the trial coordinators about any clinical trial you consider, check out this list of questions we put together with the help of chordoma community members. If you would like assistance considering clinical trial options, our Patient Navigators can help. View question lists » |
Chordoma-specific trials
The following trials are designed specifically for chordoma patients or are accepting a group of chordoma patients into a specific arm of the trial. These trials are open and currently enrolling patients unless otherwise noted. Trials noted with the Chordoma Foundation icon have been directly supported by our Clinical Trials Program.
Afatinib | EGFR inhibitor
For patients with locally advanced or metastatic tumors that express EGFR
Summary: This is a Phase 2 trial studying the effectiveness of the drug afatinib. Afatinib is an EGFR inhibitor, a type of targeted drug therapy that blocks the protein EGFR, which drives the growth of tumor cells. This drug is relevant to chordoma patients because the majority of chordoma tumors express the EGFR protein and afatinib has been shown to stop the growth of chordoma tumors in mice. Additionally, there have been a number of reported responses to similar EGFR inhibitors in chordoma patients.
More information and FAQs »
ClinicalTrials.gov ID: NCT03083678
Where: Leiden University Medical Center (Leiden, Netherlands)
Contact: Astrid Lipplaa, MD | a.lipplaa@lumc.nl
Where: University College London Hospital
Contact: uclh.afatinibchordoma@nhs.net
Where: Istituto Nazionale dei Tumori: Fondazione IRCCS (Milan, Italy)
Contact: silvia.stacchiotti@istitutotumori.mi.it
Anlotinib hydrochloride | Tyrosine kinase inhibitor
For patients with locally advanced, unresectable, or metastatic tumors
Summary: This is a Phase 2 trial studying the effectiveness of the drug anlotinib hydrochloride versus imatinib. Anlotinib is a tyrosine kinase inhibitor that targets the VEGFR, PDGFR, and EGFR proteins, which drive the growth of tumor cells. This drug is relevant to chordoma patients because the majority of chordoma tumors express these proteins. Imatinib, which has been studied in chordoma in previous trials, targets PDGFR. The trial will compare how the two drugs work in advanced chordoma.
ClinicalTrials.gov ID: NCT04042597
Where: Peking University People’s Hospital (Beijing, China)
Contact: Tingting Ren, PhD | +86 138 009 5026 | tumorcenter@163.com
Nivolumab + relatlimab | PD-1 inhibitor + Anti-LAG-3 inhibitor
For patients with locally advanced or metastatic tumors
Summary: This is a
Phase 2 trial studying the effectiveness of the combination of the drugs nivolumab and relatlimab. These drugs are both in a class of immunotherapies called checkpoint inhibitors. Checkpoint inhibitors interfere with certain proteins expressed on either tumor cells or immune cells, which releases the brakes on the immune system so it can attack the tumor. This trial is relevant to chordoma patients because chordoma tumors have been shown to express the PD-L1 protein, a checkpoint that may contribute to the tumor’s resistance to immune attack. ClinicalTrials.gov ID: NCT03623854
Where: UCLA Jonsson Comprehensive Cancer Center, Los Angeles, California
Contact: Arun Singh, MD | (310) 829-5471 | asingh@mednet.ucla.edu
Nivolumab + stereotactic radiosurgery | PD-1 inhibitor in combination with radiation
For patients with advanced or metastatic tumors
Summary: This is a Phase 1 trial studying the safety and initial effectiveness of the drug nivolumab with and without stereotactic radiosurgery. Nivolumab is a type of immunotherapy called a checkpoint inhibitor. Checkpoint inhibitors block certain proteins on tumor cells that prevent the immune system from attacking the tumor. Nivolumab blocks the protein PD-1 on immune cells, which interacts with the PD-L1 protein on chordoma cells. This trial is relevant to chordoma patients because chordoma tumors have been shown to express the PD-L1 protein, which may contribute to their resistance to immune attack. Additionally, stereotactic radiosurgery is believed to make tumors more susceptible to the immune system. The checkpoint inhibitor is being tested in combination with radiation because this combination has shown synergy in other tumor types.
More information and FAQs »
ClinicalTrials.gov ID: NCT02989636
Where: Johns Hopkins Hospital (Baltimore) and Memorial Sloan Kettering Cancer Center (New York City)
Contact: Whitney Webb, RN, MSN at wwebb10@jhmi.edu
eIRB application number: IRB00117650
Palbociclib | CDK4/6 inhibitor
For patients with locally advanced or metastatic tumors
Summary: This is a
Phase 2 trial studying the effectiveness of the drug palbociclib. Palbociclib is an inhibitor of CDK4 and CDK6, two proteins that enable tumor growth. These proteins are usually controlled by another protein called p16, which is produced by a gene called CDKN2A. This drug is relevant to chordoma patients because nearly all chordoma tumors have a loss of the CDKN2A gene, and therefore don’t produce the p16 protein to help regulate CDK4 and CDK6. Additionally, chordoma cell lines and mouse models have shown response to CDK4/6 inhibitors in the lab. More information and FAQs »
ClinicalTrials.gov ID: NCT03110744
Where: National Center for Tumor Diseases (Heidelberg, Germany)
Contact: Prof. Dr. Richard Schlenk | +49 6221 56 6228 | richard.schlenk@nct-heidelberg.de
Pemetrexed | Antifolate chemotherapy
For patients with any stage of disease
Summary: This is a Phase 1 trial studying the safety and initial effectiveness of the folate antimetabolite drug pemetrexed. This type of chemotherapy interferes with certain enzymes that are critical for cell growth and survival. This trial is relevant to chordoma patients because the majority of chordoma tumors have been found to have low levels of an enzyme called thymidylate synthase (TS), which is believed to allow pemetrexed to be more effective at stopping the growth of tumor cells.
More information and FAQs »
ClinicalTrials.gov ID: NCT03955042
Where: John Wayne Cancer Institute (Santa Monica, California)
Contact: Mini Gill, RN | 310-582-7437 | jaya.gill@providence.org
Tazemetostat | EZH2 inhibitor
For patients with recurrent or metastatic tumors with INI1 loss
Summary: This is a Phase 2 trial evaluating the effectiveness of tazemetostat, a new drug that inhibits the EZH2 protein. This trial is designed specifically for patients whose tumors have lost expression of a protein called INI1. INI1 is a tumor suppressor – it protects cells from turning cancerous. When INI1 is lost in tumor cells, they become vulnerable to inhibition of the EZH2 protein. This trial is relevant to chordoma patients because some chordoma tumors, particularly the poorly differentiated type, have lost INI1 protein expression, which may make them susceptible to EZH2 inhibitors like tazemetostat.
More information and FAQs »
ClinicalTrials.gov ID: NCT02601950
Where: 33 trial locations around the world
Contact: See full list of locations and contacts here
Other trials for adult patients
The following trials are open to patients with multiple tumor types, including chordoma, and are currently enrolling unless otherwise noted. The Foundation’s Medical Advisory Board has determined that these trials may be relevant to chordoma patients based on available scientific evidence.
BCA101 +/- pembrolizumab | EGFR TGFβ inhibitor with or without PD-1 inhibitor
For patients ages 18 years and older with locally recurrent, advanced, or metastatic disease
Summary: This is a Phase 1 trial studying the safety and initial effectiveness of BCA101 given with or without pembrolizumab. BCA101 is a new, experimental drug that targets both EGFR and TGFβ, and pembrolizumab is a PD-1 inhibitor. According to the design of the trial, chordoma patients are only eligible for the group of participants who will receive BCA101 alone. This drug is relevant to chordoma patients because the majority of chordoma tumors express the EGFR protein.
ClinicalTrials.gov ID: NCT04429542
Where: 4 locations in the United States and Canada
Contact: See full list of contacts and locations here
Durvalumab + tremelimumab | PD-L1 inhibitor + CTLA-4 inhibitor
For patients with advanced or metastatic tumors
Summary: This is a Phase 2 trial studying the effectiveness of the combination of the drugs durvalumab and tremelimumab. These drugs are both in a class of immunotherapies called checkpoint inhibitors. Checkpoint inhibitors block certain proteins on tumor cells which prevent the immune system from attacking the tumor. This trial is relevant to chordoma patients because chordoma tumors have been shown to express the PD-L1 protein, which may contribute to their resistance to immune attack. Trial ID: ACTRN12616001019493 (Australia New Zealand Clinical Trials Registry)
Where: 5 sites in Australia
Contact: Dr. Lucille Sebastian | +61 2 9562 5000 | most@ctc.usyd.edu.au
INT230-6 | Direct intratumoral injection of a novel, cell-penetrating, anti-cancer drug formulation
For patients with locally advanced spine or metastatic disease
Summary: This is a P
hase 1/2 trial evaluating the safety and effectiveness of repeated direct intratumoral injections of a new drug, INT230-6, which consists of cisplatin and vinblastine sulfate together with a substance that disperses the drugs throughout the tumor and increases diffusion into the cancer cells. Injections of INT230-6 in mice have shrunk large tumors and induced an immune response against the cancer with uninjected tumors also reducing in size. This trial is relevant to chordoma patients because one chordoma patient enrolled in the trial had a partial response in the injected tumors as well as untreated lung metastases.
ClinicalTrials.gov ID: NCT03058289
Where: 6 locations in the United States and Canada
Contact: See full list of contacts and locations here
KB-0742 | CDK9 inhibitor
For patients with locally advanced spine and sacral tumors or metastatic disease
Summary: This is a Phase 1 trial studying the safety and tolerability of KB-0742, which is a new, experimental drug that targets a protein called CDK9. This drug is relevant to chordoma patients because recent preclinical research has found that inhibiting CDK9 disrupts the expression of brachyury in chordoma cells, which causes the cancer cells to stop growing. Additionally, CDK9 inhibitors have been found to slow chordoma tumor growth in mouse models.
ClinicalTrials.gov ID: NCT04718675
Where: 4 locations in the United States
Contact: See full list of contacts and locations here
MVX-ONCO-1 | Autologous tumor vaccine
For patients with advanced or metastatic tumors who are no longer eligible for standard therapies
Summary: This is a Phase 1 trial studying the safety and initial effectiveness of an autologous tumor vaccine called MVX-ONCO-1. To create the vaccine, tumor cells collected from the patient are killed with radiation and then administered together with encapsulated cells that stimulate the immune system by secreting a natural immune booster named GM-CSF. Two capsules are implanted under the skin (1 cm apart), and the autologous tumor cells are injected between the capsules to become visible to the immune system. This induces an immune response against the cells from all tumor sites in the body. A single chordoma patient on this Phase 1 trial has had a sustained response to the vaccine for more than 18 months after receiving the treatment, so our Medical Advisory Board believes this trial could be considered of interest for the treatment of chordoma.
ClinicalTrials.gov ID: NCT02193503
Where: Hôpitaux Universitaires de Geneve (HUG) (Geneva, Switzerland)
Contact: Eugenio Fernandez, MD | +41 22 372 29 01 | eugenio.fernandez@hcuge.ch
NCI-MATCH | Molecularly guided targeted therapy
For patients with recurrent or metastatic tumors
Summary: This is a Phase 2 trial that matches patients to targeted cancer therapies based on molecular tumor profiling. Patients receive one of 13 drugs selected to target a specific molecular abnormality in their tumor.
This trial is relevant to chordoma patients because it may be able to provide a therapy tailored to the unique makeup of their tumor.
ClinicalTrials.gov ID: NCT02465060
Where: 1,000+ trial locations in the U.S.
Contact: See full list of locations and contacts here
Nivolumab + nab-rapamycin | PD-1 inhibitor + mTOR inhibitor
For patients with advanced disease
Summary: This is a Phase 1/2 trial studying the effectiveness of the combination of the checkpoint inhibitor nivolumab and a new mTOR inhibitor, nab-rapamycin. Checkpoint inhibitors interfere with certain proteins expressed on either tumor cells or immune cells, and release the brakes on the immune system in order to attack the tumor. Nab-rapamycin inhibits an enzyme called mTOR which supports cell growth; interfering with this enzyme in tumor cells can cause them to die. This trial is relevant to chordoma patients because chordoma tumors have been shown to express the PD-L1 protein making them more susceptible to immune attack by immune checkpoint inhibitors and there is documentation of response to mTOR inhibitors in chordoma patients. ClinicalTrials.gov ID: NCT03190174
Where: Sarcoma Oncology Center (Santa Monica, CA)
Contact: Victoria Chua | 310-552-9999 | vchua@sarcomaoncology.com
Regorafenib | Multi-kinase inhibitor; angiogenesis inhibitor
For patients with metastatic tumors
Summary: This is a Phase 2 trial evaluating the effectiveness of regorafenib for patients with metastatic bone sarcomas, including chordoma. Regorafenib is a newer generation multi-kinase inhibitor similar to sorafenib, which has been shown to have modest activity in chordoma. Both of these drugs block the protein VEGF, which prevents the development of blood vessels to supply nutrients to the tumor.
ClinicalTrials.gov ID: NCT02389244
Where: 20 trial locations in France
Contact: Jessy Delaye | +33 (0) 144 230 404 | j-delaye@unicancer.fr
TAEK-VAC-HerBy +/- checkpoint inhibitor | Brachyury vaccine with or without PD-1/PD-L1 inhibitor/HER2 antibody
For patients with locally advanced or metastatic disease
Summary: This is a Phase 1/2 trial studying the safety and initial effectiveness of intravenous TAEK-VAC-HerBy (TVH) vaccine given with or without PD-1/PD-L1 inhibitor or HER2 antibody. TVH is a therapeutic vaccine designed to stimulate and boost the immune system to seek out and destroy cancer cells that contain a protein called brachyury, which is present at very high levels in nearly all chordomas but not in normal tissues. According to the design of the trial, chordoma patients are only eligible for the first stage of the trial, which will administer the TVH vaccine alone.
ClinicalTrials.gov ID: NCT04246671
Where: Mayo Clinics in Minnesota, Florida, and Arizona
Contact: See full list of contacts and locations here
TAPUR | Targeting specific abnormalities in tumor genes
For patients with advanced disease
Summary: This is a Phase 2 trial that matches patients to targeted cancer therapies based on molecular tumor profiling. Patients receive one of 14 drugs and/or drug combinations selected to target the specific molecular abnormality in their tumor. This trial is relevant to chordoma patients because it may be able to provide a therapy tailored to the unique molecular makeup of their tumor. ClinicalTrials.gov ID: NCT02693535
Where: 24 trial locations in the U.S.
Contact: See full list of locations and contacts here
Talimogene laherparepvec, nivolumab, trabectedin | Oncolytic virus, PD-1 inhibitor, chemotherapy
For patients with advanced disease
Summary: This is a Phase 2 trial studying the effectiveness of the combination of three therapies: an oncolytic virus, a checkpoint inhibitor called nivolumab, and a chemotherapy drug. Checkpoint inhibitors interfere with certain proteins expressed on either tumor cells or immune cells, and release the brakes on the immune system in order to attack the tumor. This trial is relevant to chordoma patients because chordoma tumors have been shown to express the PD-L1 protein, making them more susceptible to immune attack by immune checkpoint inhibitors. The combination of the checkpoint inhibitor with the oncolytic virus and chemotherapy could further enhance the effectiveness of the immune system against killing the tumor cells. ClinicalTrials.gov ID: NCT03886311
Where: Sarcoma Oncology Center (Santa Monica, CA)
Contact: Victoria Chua | 310-552-9999 | vchua@sarcomaoncology.com
Target Rare Cancer Knowledge (TRACK) | Comprehensive genomic profiling
For patients with rare tumors (within the United States only)
Summary: This trial is studying the outcomes of rare cancer patients whose treatment is guided by comprehensive genomic profiling (CGP) results. Foundation Medicine will complete CGP on a sample of tumor tissue as well as plasma circulating cell-free DNA from a blood sample. Tumor tissue must have been collected within the past 18 months. The profiling results will be provided directly to the treating physician and will also be presented to the study’s Virtual Molecular Tumor Board (VMTB). The VMTB will analyze the findings and provide a written report to the treating physician on recommended treatments and relevant clinical trials. The treating physician makes all treatment decisions. Participants will be followed over time in order to link any treatments guided by the profiling results to their outcomes.
ClinicalTrials.gov ID: NCT04504604
Where: Anywhere in the United States (remote participation)
Contact: Mary Oster | 617-299-0389 | mary@targetcancerfoundation.org
Other trials for pediatric and young adult patients
The following trials are open to younger patients with multiple tumor types, including chordoma, and are currently enrolling unless otherwise noted. The Foundation’s Medical Advisory Board has determined that these trials may be relevant to chordoma patients based on available scientific evidence.
KB-0742 | CDK9 inhibitor
For patients 16 years and older with locally advanced spine and sacral tumors or metastatic disease
Summary: This is a Phase 1 trial studying the safety and tolerability of KB-0742, which is a new, experimental drug that targets a protein called CDK9. This drug is relevant to chordoma patients because recent preclinical research has found that inhibiting CDK9 disrupts the expression of brachyury in chordoma cells, which causes the cancer cells to stop growing. Additionally, CDK9 inhibitors have been found to slow chordoma tumor growth in mouse models.
ClinicalTrials.gov ID: NCT04718675
Where: 4 locations in the United States
Contact: See full list of contacts and locations here
Nivolumab + ipilimumab | PD-1 inhibitor + CTLA-4 inhibitor
For patients ages 6 months to 30 years with newly diagnosed or recurrent INI1-negative (poorly differentiated) tumors
Summary: This is a Phase 2 trial studying the effectiveness of the combination of the drugs nivolumab and ipilimumab in patients with INI1-negative tumors. These drugs are both in a class of immunotherapies called checkpoint inhibitors, which block certain proteins on tumor cells that prevent the immune system from attacking the tumor. In certain tumor types, the combination of these drugs has been more effective than either drug alone. This trial is relevant to chordoma patients because chordoma tumors have been shown to express various checkpoint molecules, which may contribute to their resistance to immune attack.
ClinicalTrials.gov ID: NCT04416568
Where: Dana-Farber Cancer Institute (Boston, Massachusetts, United States)
Contact: Suzanne Forrest, MD | 617-632-6443 | suzanne_forrest@dfci.harvard.edu
Pediatric NCI-MATCH | Molecularly guided targeted therapy
For pediatric patients ages 12 months to 21 years with recurrent or advanced tumors
Summary: This is a Phase 2 trial that matches patients to targeted cancer therapies based on molecular tumor profiling. Patients receive one of 8 drugs selected to target a specific molecular abnormality in their tumor. This trial is relevant to chordoma patients because it may be able to provide a therapy tailored to the unique makeup of their tumor.
ClinicalTrials.gov ID: NCT03155620
Where: 93 trial locations in the U.S.
Contact: See full list of locations and contacts here.
Tazemetostat | EZH2 inhibitor
For patients ages 6 months to 17 years with recurrent or metastatic INI1-negative (poorly differentiated) tumors
Summary: This is a Phase 1 trial evaluating the safety and initial effectiveness of tazemetostat, a new drug that inhibits the EZH2 protein. This trial is specifically for pediatric patients whose tumor cells have lost expression of a protein called INI1. INI1 is a tumor suppressor – it protects cells from turning cancerous. When INI1 is lost in tumor cells, they become vulnerable to inhibition of the EZH2 protein. This trial is relevant to chordoma patients because many pediatric chordomas have INI1 loss, particularly those classified as poorly differentiated. Loss of INI1 protein expression may make these chordoma tumors more susceptible to EZH2 inhibitors like tazemetostat.
ClinicalTrials.gov ID: NCT02601937
Where: 33 trial locations around the world
Contact: See full list of locations and contacts here
Participate in the Natural History Study of Rare Solid Tumors 
All chordoma patients, including those in active treatment, are invited to participate in the Natural History Study of Rare Solid Tumors, currently underway at the U.S. National Cancer Institute (NCI). By sharing your medical records and tissue samples and answering online surveys about your medical history and quality of life, you can help clinicians and scientists learn more about why and how chordoma progresses, ultimately aiding in the identification and development of new therapies to test. Learn how to get involved in the Natural History Study of Rare Solid Tumors » |
Further information about clinical trials |
Page last updated: February 4, 2021
Disclaimer
The Chordoma Foundation cannot guarantee the completeness of this list of clinical trials, and details about clinical trials are subject to change. We update the information above as soon as the information is made available to us. For more information about clinical trials that you might qualify for, please contact a CF Patient Navigator at chordoma.org/request-help, consult with your physician, or visit ClinicalTrials.gov.
The information provided herein is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your or your loved one’s physician about any questions you have regarding your or your loved one’s medical care. Never disregard professional medical advice or delay in seeking it because of something you have read on this Website.
