Trial Outcomes & Findings for Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor (NCT NCT01207492)
NCT ID: NCT01207492
Last Updated: 2025-07-17
Results Overview
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.
ACTIVE_NOT_RECRUITING
PHASE2
17 participants
6 months
2025-07-17
Participant Flow
Participant milestones
| Measure |
Nilotinib
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Overall Study
STARTED
|
17
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
Nilotinib
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Overall Study
Still on study
|
1
|
|
Overall Study
Withdrawal by Subject
|
9
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Progressive disease
|
3
|
|
Overall Study
Toxicity
|
1
|
Baseline Characteristics
Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor
Baseline characteristics by cohort
| Measure |
Nilotinib
n=17 Participants
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
45.35 years
STANDARD_DEVIATION 13.54 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsTo 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.
Outcome measures
| Measure |
Nilotinib
n=17 Participants
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Percentage of Participants With Progression Free Survival
|
82 percentage of participants with PFS
Interval 57.0 to 96.0
|
SECONDARY outcome
Timeframe: 2 yearsTo 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.
Outcome measures
| Measure |
Nilotinib
n=17 Participants
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Overall Tumor Response Rate (OR)
|
0 percentage of participants
Interval 0.0 to 20.0
|
SECONDARY outcome
Timeframe: 6 monthsTo 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.
Outcome measures
| Measure |
Nilotinib
n=17 Participants
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Clinical Benefit Rate
|
47 percentage of participants
Interval 23.0 to 72.0
|
Adverse Events
Nilotinib
Serious adverse events
| Measure |
Nilotinib
n=17 participants at risk
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
General disorders
Fatigue
|
11.8%
2/17
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
5.9%
1/17
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
5.9%
1/17
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
5.9%
1/17
|
Other adverse events
| Measure |
Nilotinib
n=17 participants at risk
Nilotinib 200 mg taken as 400 mg twice daily, continuously
nilotinib: Taken orally twice daily
|
|---|---|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
11.8%
2/17
|
|
Infections and infestations
Lung infection
|
5.9%
1/17
|
|
Psychiatric disorders
Restlessness
|
5.9%
1/17
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
5.9%
1/17
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
5.9%
1/17
|
|
Blood and lymphatic system disorders
Anemia
|
17.6%
3/17
|
|
Cardiac disorders
Chest pain - cardiac
|
5.9%
1/17
|
|
Eye disorders
Scleral disorder
|
5.9%
1/17
|
|
Eye disorders
Eye disorders - Other, specify
|
17.6%
3/17
|
|
Gastrointestinal disorders
Abdominal pain
|
11.8%
2/17
|
|
Gastrointestinal disorders
Constipation
|
29.4%
5/17
|
|
Gastrointestinal disorders
Diarrhea
|
17.6%
3/17
|
|
Gastrointestinal disorders
Dyspepsia
|
5.9%
1/17
|
|
Gastrointestinal disorders
Nausea
|
52.9%
9/17
|
|
Gastrointestinal disorders
Vomiting
|
23.5%
4/17
|
|
General disorders
Edema face
|
5.9%
1/17
|
|
General disorders
Fatigue
|
47.1%
8/17
|
|
General disorders
Fever
|
5.9%
1/17
|
|
General disorders
Non-cardiac chest pain
|
11.8%
2/17
|
|
General disorders
Pain
|
11.8%
2/17
|
|
Infections and infestations
Upper respiratory infection
|
23.5%
4/17
|
|
Investigations
Alanine aminotransferase increased
|
29.4%
5/17
|
|
Investigations
Aspartate aminotransferase increased
|
29.4%
5/17
|
|
Investigations
Blood bilirubin increased
|
11.8%
2/17
|
|
Investigations
Lipase increased
|
5.9%
1/17
|
|
Investigations
Platelet count decreased
|
5.9%
1/17
|
|
Investigations
Weight loss
|
11.8%
2/17
|
|
Metabolism and nutrition disorders
Anorexia
|
17.6%
3/17
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
5.9%
1/17
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
11.8%
2/17
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
5.9%
1/17
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.9%
1/17
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
11.8%
2/17
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.9%
1/17
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
11.8%
2/17
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
23.5%
4/17
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
17.6%
3/17
|
|
Nervous system disorders
Dizziness
|
11.8%
2/17
|
|
Nervous system disorders
Headache
|
41.2%
7/17
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
5.9%
1/17
|
|
Psychiatric disorders
Insomnia
|
11.8%
2/17
|
|
Renal and urinary disorders
Urinary frequency
|
11.8%
2/17
|
|
Reproductive system and breast disorders
Vaginal dryness
|
5.9%
1/17
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.9%
1/17
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
11.8%
2/17
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.9%
1/17
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
17.6%
3/17
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
23.5%
4/17
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
5.9%
1/17
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
11.8%
2/17
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
23.5%
4/17
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
52.9%
9/17
|
|
Vascular disorders
Hypertension
|
5.9%
1/17
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place