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

NCT01207492 | Completed Phase 2 Results DMC

• 2 other identifiers: 10-179YUS23T
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 7

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.

Conditions

Diffuse-type Giant Cell Tumor; Tenosynovial Giant Cell Tumor; Pigmented Villonodular Synovitis

Keywords

nilotinib

Outcome Measures

Primary Outcomes (1)

• 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.

  6 months

Secondary Outcomes (2)

• Overall Tumor Response Rate (OR)

  2 years

• Clinical Benefit Rate

  6 months

Study Arms (1)

Nilotinib

EXPERIMENTAL

Nilotinib 200 mg taken as 400 mg twice daily, continuously

Drug: nilotinib

Interventions

nilotinib DRUG

Taken orally twice daily

Nilotinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Andrew J. Wagner, MD, PhD: lead
• Brigham and Women's Hospital: collaborator
• Massachusetts General Hospital: collaborator
• Novartis: collaborator

Study Sites (7)

Sarcoma Oncology Center

Santa Monica, California, 90403, United States

Location

Stanford University Medical Center

Stanford, California, 94305, United States

Location

H. Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

UT MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Synovitis, Pigmented Villonodular; Giant Cell Tumor of Tendon Sheath

Interventions

nilotinib

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Results Point of Contact

• Title: Andrew Wagner, MD, PhD
• Organization: Dana-Farber Cancer Institute

andrew_wagner@dfci.harvard.edu

617.632.5204

Study Officials

• Andrew J. Wagner, MD, PhD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor

Study Record Dates

• First Submitted: September 21, 2010
• First Posted: September 23, 2010
• Study Start: September 1, 2010
• Primary Completion: April 1, 2014
• Study Completion: August 1, 2015
• Last Updated: February 20, 2026
• Results First Posted: June 25, 2015

Record last verified: 2026-02

Locations

US (7)