ChordomaPhase 1NCT01407198
Nilotinib With Radiation for High Risk Chordoma
A Phase 1 study testing Nilotinib and Radiation therapy in people with Chordoma, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The study drug, Nilotinib, is believed to slow down tumor growth by regulating a gene involved in cellular growth of chordoma cells. During this research study, subjects will also receive radiation therapy which is considered a standard treatment for advanced chordomas. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chordoma
- What is being tested?
- Nilotinib and Radiation therapy
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2011to Jun 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically confirmed chordoma
- Considered to have high risk disease
- Measurable disease
- Life expectancy > 3 months
- Adequate organ function
- Able to swallow oral capsules
Who cannot join
- Previous treatment with any other tyrosine kinase inhibitor
- Previous treatment with radiotherapy to the primary or recurrent chordomas
- Impaired cardiac function
- Currently receiving treatment with strong CYP3A4 inhibitors
- Requires anticoagulation with coumadin
- Impaired GI function or GI disease that may significantly alter the absorption of study drug
Show all 15Show fewer
- Acute or chronic pancreatic disease
- Known cytopathologically confirmed CNS infiltration
- Another primary malignant disease which requires systemic treatment
- Acute or chronic liver disease or severe renal disease considered unrelated to the cancer
- History of significant congenital or acquired bleeding disorder unrelated to cancer
- Major surgery within 4 weeks prior to Day 1 of the study or who have not recovered from prior surgery
- Treatment with other investigational agents within 30 days of Day 1
- History of non-compliance to medical regimens
- Pregnant or breast-feeding
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: Phase I Study of Nilotinib Given With Radiation For Patients With High Risk Chordoma
The study drug, Nilotinib, is believed to slow down tumor growth by regulating a gene involved in cellular growth of chordoma cells. During this research study, subjects will also receive radiation therapy which is considered a standard treatment for advanced chordomas. It is hoped by adding nilotinib, the benefits of radiation therapy can be enhanced without adding significant toxicities.
The purpose of this research study is to determine the safety of nilotinib when used in combination with radiation therapy, and the highest dose of nilotinib that can be given safely with radiation therapy.
Read the detailed description
Nilotinib will be taken orally daily in two cycles of 28 days each. Two weeks after taking nilotinib, subjects will begin radiation therapy. Radiation therapy will continue every weekday until Day 56 of the study. If it is determined that the subject's tumor cannot be removed by surgery, an additional 3 weeks of radiation therapy will be applied after Day 56 of the study.
During study visits subjects will have physical exams, routine blood tests, urine and blood clotting tests, and EKGs. Subjects will also have tumor assessment by chest CT and MRI or CT of the tumor at screening, on approximately Day 56 of the study, then every 6 months for one year and then annually thereafter if ther is no disease progression.
- Conditions
- Chordoma
- Being tested
- Nilotinib, Radiation therapy
- Keywords
- High Risk Chordoma
- Medical subject headings
- Chordoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NilotinibDrug
Also called AMN 107, Tasigna
Orally, daily 200 - 400 mg BID
Radiation therapyRadiation
Also called External beam radiation
External beam radiation will be delivered at 1.8 Gy per day, 5 days per week (excluding holidays)for a total of 28 fractions over a 6 weeks period.
| Study group | Role | What they receive |
|---|---|---|
| Nilotinib/XRT | Experimental | Nilotinib is administered 400mg PO BID for 2 weeks and then administered concurrently with daily radiation therapy until completion of radiation therapy. Participants can then undergo surgery if clinically possible. After either surgery or definitive radiation, participants have the option to continue on nilotinib therapy. Receives: Drug: Nilotinib; Radiation: Radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the dose limiting toxicities (DLTs) for participants when treated above the maximum tolerated dose (MTD).
A DLT will be defined as any of the following events occurring during days 1-56 of study according to CTCAE version 4.0. MTD is then determined by identifying a dose cohort demonstrating > or = 30% rate of DLTs, which are defined as:
- Any Grade ≥ 3 nonhematologic toxicity except alopecia, nausea, or vomiting not otherwise controlled by maximual supportive care.
- Grade 4 neutropenia (ANC < 500/µL) lasting > 5 days, Grade 3 thrombocytopenia lasting > 7 days, or Grade 4 thrombocytopenia
- Failure to resume treatment delays within a defined period of time.
Measured: 2 years
Secondary outcomes (3)
Number of Participants with Adverse Events
Adverse event profile, as determined by CTC AE version 4.0, will be determined for patients treated with nilotinib and radiation therapy.
Adverse events analyses will include:
- Detailed examination of adverse events
- Laboratory test results
- Vital signs or other physical findings
- Frequency and extent of dose modification
The assessment of adverse events will be based mainly on the frequency of adverse events, particularly adverse events leading to discontinuation of treatment and on the number of significant laboratory abnormalities.
Measured: 2 years
Survival
To obtain preliminary data regarding local control, distant control, disease-free survival, and overall survival with this regimen
Measured: 2 years
PDGFR signaling
To determine if nilotinib decreases PDGFR signaling in chordoma tumor samples in treated patients
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2011 (estimated)
- Study startAug 2011
- Primary completionFeb 2016
- Study completionJun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ChordomaStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Edwin Choy, MD, Massachusetts General Hospital
- Investigators
- Edwin Choy, MD, PhD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01407198
- Sponsor's protocol ID
- 11-072
- Status last verified
- Dec 2025
- First submitted
- 28 Jul 2011
- Last updated
- 19 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Nilotinib Given With Radiation For Patients With High Risk Chordoma
- ClinicalTrials.gov ID
- NCT01407198
- Acronym / sponsor ID
- 11-072
- Conditions
- Chordoma
- Keywords
- High Risk Chordoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- Aug 2011
- Primary completion
- Feb 2016 (actual)
- Study completion
- Jun 2025 (actual)
- First posted
- 2 Aug 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study drug, Nilotinib, is believed to slow down tumor growth by regulating a gene involved in cellular growth of chordoma cells. During this research study, subjects will also receive radiation therapy which is considered a standard treatment for advanced chordomas. It is hoped by adding nilotinib, the benefits of radiation therapy can be enhanced without adding significant toxicities.
The purpose of this research study is to determine the safety of nilotinib when used in combination with radiation therapy, and the highest dose of nilotinib that can be given safely with radiation therapy.
Detailed description
Nilotinib will be taken orally daily in two cycles of 28 days each. Two weeks after taking nilotinib, subjects will begin radiation therapy. Radiation therapy will continue every weekday until Day 56 of the study. If it is determined that the subject's tumor cannot be removed by surgery, an additional 3 weeks of radiation therapy will be applied after Day 56 of the study.
During study visits subjects will have physical exams, routine blood tests, urine and blood clotting tests, and EKGs. Subjects will also have tumor assessment by chest CT and MRI or CT of the tumor at screening, on approximately Day 56 of the study, then every 6 months for one year and then annually thereafter if ther is no disease progression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nilotinib/XRT | Experimental | Drug: Nilotinib, Radiation: Radiation therapyNilotinib is administered 400mg PO BID for 2 weeks and then administered concurrently with daily radiation therapy until completion of radiation therapy. Participants can then undergo surgery if clinically possible. After either surgery or definitive radiation, participants have the option to continue on nilotinib therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Histologically confirmed chordoma
- Considered to have high risk disease
- Measurable disease
- Life expectancy > 3 months
- Adequate organ function
Key exclusion criteria (15 listed)
- Previous treatment with any other tyrosine kinase inhibitor
- Previous treatment with radiotherapy to the primary or recurrent chordomas
- Impaired cardiac function
- Currently receiving treatment with strong CYP3A4 inhibitors
- Requires anticoagulation with coumadin
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NilotinibAlso: AMN 107, TasignaOrally, daily 200 - 400 mg BID | Drug | Nilotinib/XRT |
| Radiation therapyAlso: External beam radiationExternal beam radiation will be delivered at 1.8 Gy per day, 5 days per week (excluding holidays)for a total of 28 fractions over a 6 weeks period. | Radiation | Nilotinib/XRT |
Primary outcome
To determine the dose limiting toxicities (DLTs) for participants when treated above the maximum tolerated dose (MTD).
Time frame: 2 years
A DLT will be defined as any of the following events occurring during days 1-56 of study according to CTCAE version 4.0. MTD is then determined by identifying a dose cohort demonstrating > or = 30% rate of DLTs, which are defined as: * Any Grade ≥ 3 nonhematologic toxicity except alopecia, nausea, or vomiting not otherwise controlled by maximual supportive care. * Grade 4 neutropenia (ANC < 500/µL) lasting > 5 days, Grade 3 thrombocytopenia lasting > 7 days, or Grade 4 thrombocytopenia * Failure to resume treatment delays within a defined period of time.
3 secondary outcomes
Number of Participants with Adverse Events
Time frame: 2 years
Survival
Time frame: 2 years
PDGFR signaling
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Chordoma
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
Children and Adults With Chordoma
Chordoma
Active, not recruitingObservational study
Genetic Clues to Chordoma Etiology: A Protocol to Identify Sporadic Chordoma Patients for Studies of Cancer-Susceptibility Genes
Genes, Sporadic Chordoma
RecruitingObservational study
Exploration of Personalized Biomarkers During Neoadjuvant Radiation Therapy for Spinal and Sacral Chordoma
Chordoma of Spine, Chordoma of Skull Base
Active, not recruiting
High Dose Intensity Modulated Proton Radiation Treatment +/- Surgical Resection of Sarcomas of the Spine, Sacrum and Base of Skull
Chordoma of Spine, Chordoma of Sacrum
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edwin Choy, MD, PhD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nilotinib and Radiation therapy in people with Chordoma, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the dose limiting toxicities (DLTs) for participants when treated above the maximum tolerated dose (MTD). (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2025.