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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Dana-Farber Cancer Institute
Sponsor:
Collaborators:
Brigham and Women's Hospital
Massachusetts General Hospital
Novartis
Information provided by (Responsible Party):
Andrew J. Wagner, MD, PhD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:
NCT01207492
First received: September 21, 2010
Last updated: January 23, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention Phase
Pigmented Villonodular Synovitis
Diffuse-type Giant Cell Tumor
Tenosynovial Giant Cell Tumor
Drug: nilotinib
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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

Resource links provided by NLM:


Further study details as provided by Dana-Farber Cancer Institute:

Primary Outcome Measures:
  • Progression free survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To estimate progression free survival at 6 months in participants with recurrent PVNS treated with nilotinib.


Secondary Outcome Measures:
  • Overall tumor response rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine overall tumor response rate [% complete response + % partial response by RECIST 1.1]

  • Clinical Benefit Rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine the clinical benefit rate [% CR + % PR + % stable disease by RECIST 1.1] at 6 months


Estimated Enrollment: 25
Study Start Date: September 2010
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: nilotinib
    Taken orally twice daily
Detailed Description:
  • 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.
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Histologically confirmed diagnosis of recurrent PVNS ( or diffuse-type giant cell tumor or tenosynovial giant cell tumor) that is unresectable, metastatic, or for which the patient refuses surgical intervention
  • Progressive disease in the last 12 months, as demonstrated by imaging or clinical appearance of new tumors, in the opinion of the treating investigator
  • At least one site of measurable disease according to RECIST 1.1 on MRI (or CT scan for metastatic disease)
  • Any number or type of prior systemic therapies, with the exception of known or suspected CSF1 receptor inhibitors as outlined in exclusion criteria below
  • 18 years of age or older
  • Life expectancy greater than 6 months
  • ECOG Performance Status of 0, 1 or 2
  • Normal organ and marrow function as defined in the protocol
  • QTc less than or equal to 450 ms on 12-lead ECG
  • Negative urine or serum pregnancy test within days of start of study drug administration for women of childbearing potential.
  • Women of childbearing potential and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 3 months following study drug discontinuation

Exclusion Criteria:

  • Prior treatment with known or suspected CSF1 receptor inhibitor, including nilotinib, imatinib, sunitinib, or sorafenib, or other approved or investigational tyrosine kinase inhibitors used for treatment of diffuse-type giant cell tumor
  • Concurrent treatment with other investigational agents
  • Inability to tolerate or contraindication to MRI scanning for participants with localized disease
  • Impaired cardiac function
  • Current treatment with strong CYP3A4 inhibitors that cannot either be discontinued or switched to a different medication prior to starting study drug
  • Current treatment with any medications that have the potential to prolong the QT interval and that cannot either be discontinued or switched to a different medication prior to starting study drug
  • Impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug
  • Acute or chronic pancreatic disease
  • Acute or chronic liver disease
  • Another primary malignant disease requiring systemic treatment or radiation
  • History of significant congenital or acquired bleeding disorder unrelated to cancer
  • Major surgery within 28 days prior to Day 1 of the study
  • Treatment with other investigational agents within 28 days of day 1
  • History of non-compliance to medical regimens or inability to grant consent
  • Women who are pregnant or breastfeeding
  • Other comorbidities that would interfere with study participation or safety in the opinion of the investigator
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01207492

Contacts
Contact: Andrew J. Wagner, MD, PhD 617-632-5204 anderw_wagner@dfci.harvard.edu

Locations
United States, California
Sarcoma Oncology Center Not yet recruiting
Santa Monica, California, United States, 90403
Contact: Sant Chawla, MD    310-552-9999      
Principal Investigator: Sant Chawla, MD         
Stanford University Medical Center Recruiting
Stanford, California, United States, 94305
Contact: Kristen Ganjoo, MD    650-498-6000      
Principal Investigator: Kristen Ganjoo, MD         
United States, Florida
H. Lee Moffitt Cancer Center Recruiting
Tampa, Florida, United States, 33612
Contact: Damon Reed, MD    813-745-2297      
Principal Investigator: Damon Reed, MD         
United States, Massachusetts
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Andrew J. Wagner, MD PhD    617-632-5204      
Principal Investigator: Andrew J. Wagner, MD, PhD         
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Edwin Choy, MD PhD    617-643-0376      
Principal Investigator: Edwin Choy, MD, PhD         
United States, Pennsylvania
Fox Chase Cancer Center Recruiting
Philadelphia, Pennsylvania, United States
Contact: Margaret von Mehren, MD    215-728-2814      
Principal Investigator: Margaret von Mehren, MD         
United States, Texas
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Vinod Ravi, MD    713-792-3626      
Principal Investigator: Vinod Ravi, MD         
Sponsors and Collaborators
Andrew J. Wagner, MD, PhD
Brigham and Women's Hospital
Massachusetts General Hospital
Novartis
Investigators
Principal Investigator: Andrew J. Wagner, MD, PhD Dana-Farber Cancer Institute
  More Information

No publications provided

Responsible Party: Andrew J. Wagner, MD, PhD, Sponsor, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT01207492     History of Changes
Other Study ID Numbers: 10-179, YUS23T
Study First Received: September 21, 2010
Last Updated: January 23, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Dana-Farber Cancer Institute:
nilotinib

Additional relevant MeSH terms:
Neoplasms
Synovitis
Giant Cell Tumors
Synovitis, Pigmented Villonodular
Joint Diseases
Musculoskeletal Diseases
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type

ClinicalTrials.gov processed this record on September 18, 2014