Pigmented Villonodular SynovitisPhase 2NCT01207492
Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor
A Phase 2 study testing nilotinib in people with Pigmented Villonodular Synovitis and Diffuse-type Giant Cell Tumor, run by Andrew J. Wagner, MD, PhD at 7 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pigmented Villonodular Synovitis, Diffuse-type Giant Cell Tumor, Tenosynovial Giant Cell Tumor
- What is being tested?
- nilotinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 7 sitesUnited States
- Study dates
- Sep 2010to Aug 2015
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
UT MD Anderson Cancer Center
Houston, Texas, 77030
Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111
Sarcoma Oncology Center
Santa Monica, California, 90403
Stanford University Medical Center
Stanford, California, 94305
H. Lee Moffitt Cancer Center
Tampa, Florida, 33612
About this study
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
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.
Read the 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
- Being tested
- nilotinib
- Keywords
- nilotinib
- Medical subject headings
- Synovitis, Pigmented Villonodular, Giant Cell Tumor of Tendon Sheath
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nilotinibDrug
Taken orally twice daily
| Study group | Role | What they receive |
|---|---|---|
| Nilotinib | Experimental | Nilotinib 200 mg taken as 400 mg twice daily, continuously Receives: Drug: nilotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (2)
Overall Tumor Response Rate (OR)
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.
Measured: 2 years
Clinical Benefit Rate
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2010 (estimated)
- Study startSep 2010
- Primary completionApr 2014
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pigmented Villonodular SynovitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Andrew J. Wagner, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Brigham and Women's Hospital, Massachusetts General Hospital, Novartis
- Responsible party
- Sponsor-investigator, Andrew J. Wagner, MD, PhD, Dana-Farber Cancer Institute
- Investigators
- Andrew J. Wagner, MD, PhD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01207492
- Sponsor's protocol ID
- 10-179
- Other IDs
- YUS23T
- Status last verified
- Feb 2026
- First submitted
- 21 Sep 2010
- Last updated
- 20 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01207492
- Acronym / sponsor ID
- 10-179
- Conditions
- Pigmented Villonodular Synovitis, Diffuse-type Giant Cell Tumor, Tenosynovial Giant Cell Tumor
- Keywords
- nilotinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Andrew J. Wagner, MD, PhDUniversity, hospital or other
- Collaborators
- Brigham and Women's Hospital, Massachusetts General Hospital, Novartis
- Study start
- Sep 2010 (actual)
- Primary completion
- Apr 2014 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 23 Sep 2010 (estimated)
- Results
- Posted 25 Jun 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nilotinib | Experimental | Drug: nilotinibNilotinib 200 mg taken as 400 mg twice daily, continuously |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| nilotinibTaken orally twice daily | Drug | Nilotinib |
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.
2 secondary outcomes
Overall Tumor Response Rate (OR)
Time frame: 2 years
Clinical Benefit Rate
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew J. Wagner, MD, PhD principal investigator
Dana-Farber Cancer Institute
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing nilotinib in people with Pigmented Villonodular Synovitis and Diffuse-type Giant Cell Tumor, run by Andrew J. Wagner, MD, PhD at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Jun 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Progression Free Survival (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.