Nilotinib With Radiation for High Risk Chordoma
Primary Purpose
Chordoma
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Nilotinib
Radiation therapy
Sponsored by
About this trial
This is an interventional treatment trial for Chordoma focused on measuring High Risk Chordoma
Eligibility Criteria
Inclusion Criteria:
- Histologically confirmed chordoma
- Considered to have high risk disease
- Measurable disease
- Life expectancy > 3 months
- Adequate organ function
- Able to swallow oral capsules
Exclusion Criteria:
- 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
- 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
Sites / Locations
- Massachusetts General Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Nilotinib/XRT
Arm Description
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Survival
To obtain preliminary data regarding local control, distant control, disease-free survival, and overall survival with this regimen
PDGFR signaling
To determine if nilotinib decreases PDGFR signaling in chordoma tumor samples in treated patients
Full Information
NCT ID
NCT01407198
First Posted
July 28, 2011
Last Updated
October 27, 2021
Sponsor
Massachusetts General Hospital
1. Study Identification
Unique Protocol Identification Number
NCT01407198
Brief Title
Nilotinib With Radiation for High Risk Chordoma
Official Title
Phase I Study of Nilotinib Given With Radiation For Patients With High Risk Chordoma
Study Type
Interventional
2. Study Status
Record Verification Date
October 2021
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 2011 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Massachusetts General Hospital
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chordoma
Keywords
High Risk Chordoma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Nilotinib/XRT
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Nilotinib
Other Intervention Name(s)
AMN 107, Tasigna
Intervention Description
Orally, daily 200 - 400 mg BID
Intervention Type
Radiation
Intervention Name(s)
Radiation therapy
Other Intervention Name(s)
External beam radiation
Intervention Description
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.
Primary Outcome Measure Information:
Title
To determine the dose limiting toxicities (DLTs) for participants when treated above the maximum tolerated dose (MTD).
Description
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.
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Number of Participants with Adverse Events
Description
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.
Time Frame
2 years
Title
Survival
Description
To obtain preliminary data regarding local control, distant control, disease-free survival, and overall survival with this regimen
Time Frame
2 years
Title
PDGFR signaling
Description
To determine if nilotinib decreases PDGFR signaling in chordoma tumor samples in treated patients
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Histologically confirmed chordoma
Considered to have high risk disease
Measurable disease
Life expectancy > 3 months
Adequate organ function
Able to swallow oral capsules
Exclusion Criteria:
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
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Edwin Choy, MD, PhD
Organizational Affiliation
Massachusetts General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
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Nilotinib With Radiation for High Risk Chordoma
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