Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor
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.
Pigmented Villonodular Synovitis
Diffuse-type Giant Cell Tumor
Tenosynovial Giant Cell Tumor
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Multi-Center Single Agent Phase II Study of the Efficacy of Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor|
- Percentage of Participants With Progression Free Survival [ Time Frame: 6 months ] [ Designated as safety issue: No ]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.
- Overall Tumor Response Rate (OR) [ Time Frame: 2 years ] [ Designated as safety issue: No ]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.
- Clinical Benefit Rate [ Time Frame: 6 months ] [ Designated as safety issue: No ]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.
|Study Start Date:||September 2010|
|Estimated Study Completion Date:||April 2018|
|Primary Completion Date:||April 2014 (Final data collection date for primary outcome measure)|
Nilotinib 200 mg taken as 400 mg twice daily, continuously
Taken orally twice daily
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01207492
|United States, California|
|Sarcoma Oncology Center|
|Santa Monica, California, United States, 90403|
|Stanford University Medical Center|
|Stanford, California, United States, 94305|
|United States, Florida|
|H. Lee Moffitt Cancer Center|
|Tampa, Florida, United States, 33612|
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02215|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, Pennsylvania|
|Fox Chase Cancer Center|
|Philadelphia, Pennsylvania, United States, 19111|
|United States, Texas|
|UT MD Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Andrew J. Wagner, MD, PhD||Dana-Farber Cancer Institute|