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

NCT ID: NCT01207492

Last Updated: 2025-07-17

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-09-30
• Study Completion Date: 2026-07-31

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 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.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Nilotinib

Nilotinib 200 mg taken as 400 mg twice daily, continuously

Group Type: EXPERIMENTAL

nilotinib

Intervention Type: DRUG

Taken orally twice daily

Interventions

nilotinib

Taken orally twice daily

Intervention Type: DRUG

Eligibility 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)

Exclusion Criteria

• 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

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Brigham and Women's Hospital

OTHER

Sponsor Role: collaborator

Massachusetts General Hospital

OTHER

Sponsor Role: collaborator

Novartis

INDUSTRY

Sponsor Role: collaborator

Andrew J. Wagner, MD, PhD

OTHER

Sponsor Role: lead

Responsible Party

Andrew J. Wagner, MD, PhD

Sponsor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Andrew J. Wagner, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Dana-Farber Cancer Institute

Locations

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

Countries

United States

Other Identifiers

YUS23T

Identifier Type: OTHER

Identifier Source: secondary_id

10-179

Identifier Type: -

Identifier Source: org_study_id