Clinical Trial: Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Multi-Center Single Agent Phase II Study of the Efficacy of Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Teno

Brief Summary: 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.

Detailed Summary:

  • 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.

Sponsor: Andrew J. Wagner, MD, PhD

Current Primary Outcome: Percentage of Participants With Progression Free Survival [ Time Frame: 6 months ]

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.


Original Primary Outcome: Progression Free Survival [ Time Frame: 2 years ]

To estimate progression free survival at 6 months in participants with recurrent PVNS treated with nilotinib.


Current Secondary Outcome:

  • Overall Tumor Response Rate (OR) [ Time Frame: 2 years ]
    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 ]
    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.


Original Secondary Outcome:

  • Overall Tumor Response Rate [ Time Frame: 2 years ]
    To determine overall tumor response rate [% complete response + % partial response by RECIST 1.1]
  • Clinical Benefit Rate [ Time Frame: 2 years ]
    To determine the clinical benefit rate [% CR + % PR + % stable disease by RECIST 1.1] at 6 months


Information By: Dana-Farber Cancer Institute

Dates:
Date Received: September 21, 2010
Date Started: September 2010
Date Completion: April 2018
Last Updated: March 6, 2017
Last Verified: March 2017