Nilotinib is a drug that is used to treat a form of a blood cancer called leukemia. Nilotinib works by blocking the action of a protein that might be important for the growth of pigmented villonodular synovitis (PVNS). In this research study the investigators are testing whether nilotinib can stop the growth of PVNS or improve the symptoms experienced from PVNS.
* In this research study, each cycle of study drug dosing will last 4 weeks (28 days). During each cycle, participants will take nilotinib by mouth twice daily. During the first cycle, participants will come to the clinic on Days 1 and 8. For Cycles 2-4 and every 3 cycles thereafter, they will come to the clinic on Day 1. * The following tests and procedures will be performed at specific time points during study treatment: MRI or CT scans; physical examinations; vital signs; blood work; questionnaires and EKG. * Participants may continue in this research study for as long as they do not have serious side effects or their disease does not get worse.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
17
Taken orally twice daily
Sarcoma Oncology Center
Santa Monica, California, United States
Stanford University Medical Center
Stanford, California, United States
H. Lee Moffitt Cancer Center
Tampa, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Fox Chase Cancer Center
Philadelphia, Pennsylvania, United States
UT MD Anderson Cancer Center
Houston, Texas, United States
Percentage of Participants With Progression Free Survival
To estimate progression free survival at 6 months in participants with recurrent PVNS treated with nilotinib. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Time frame: 6 months
Overall Tumor Response Rate (OR)
To determine overall tumor response rate \[% complete response (CR) + % partial response (PR) by RECIST 1.1\]. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR) is a disappearance of all target lesions. Partial Response (PR) is a \>=30% decrease in the sum of the longest diameter of target lesions. Overall Response (OR) = CR + PR.
Time frame: 2 years
Clinical Benefit Rate
To determine the clinical benefit rate \[% CR + % PR + % stable disease by RECIST 1.1\] at 6 months. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR) is a disappearance of all target lesions. Partial Response (PR) is a \>=30% decrease in the sum of the longest diameter of target lesions. Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Time frame: 6 months
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