18 and older, any sex, with Pigmented Villonodular Synovitis or Giant Cell Tumors of the Tendon Sheath. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 at Week 25Primary· Week 25
Complete response (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Complete Response (CR)
Group
Value
95% CI
Pexidartinib Part 1
14.8
Placebo Part 1
0
Partial Response (PR)
Group
Value
95% CI
Pexidartinib Part 1
24.6
Placebo Part 1
0
Response (CR or PR)
Group
Value
95% CI
Pexidartinib Part 1
39.3
Placebo Part 1
0
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25Secondary· Baseline, Week 13, and Week 25
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Baseline
Group
Value
95% CI
Pexidartinib Part 1
62.5
± 3.2
Placebo Part 1
62.9
± 2.9
Week 13
Group
Value
95% CI
Pexidartinib Part 1
13.0
± 2.3
Placebo Part 1
4.8
± 2.6
Week 25
Group
Value
95% CI
Pexidartinib Part 1
15.1
± 2.1
Placebo Part 1
6.2
± 2.4
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo at Week 25Secondary· Week 25
Complete response (CR) and partial response (PR) were assessed using tumor volume score (TVS). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference. TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score
Complete Response (CR)
Group
Value
95% CI
Pexidartinib Part 1
4.9
Placebo Part 1
0
Partial Response (PR)
Group
Value
95% CI
Pexidartinib Part 1
50.8
Placebo Part 1
0
Response (CR or PR)
Group
Value
95% CI
Pexidartinib Part 1
55.7
Placebo Part 1
0
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25Secondary· at Week 9 , Week 17, and Week 25
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
Week 9
Group
Value
95% CI
Pexidartinib Part 1
2.8
± 1.0
Placebo Part 1
-0.4
± 0.8
Week 17
Group
Value
95% CI
Pexidartinib Part 1
3.2
± 1.1
Placebo Part 1
0.2
± 1.0
Week 25
Group
Value
95% CI
Pexidartinib Part 1
4.1
± 1.1
Placebo Part 1
-0.9
± 1.0
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25Secondary· Baseline, Week 9, Week 17, and Week 25
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Baseline
Group
Value
95% CI
Pexidartinib Part 1
5.6
± 0.2
Placebo Part 1
5.9
± 0.3
Week 9
Group
Value
95% CI
Pexidartinib Part 1
-1.5
± 0.3
Placebo Part 1
-0.5
± 0.3
Week 17
Group
Value
95% CI
Pexidartinib Part 1
-2.4
± 0.3
Placebo Part 1
-0.4
± 0.3
Week 25
Group
Value
95% CI
Pexidartinib Part 1
-2.5
± 0.3
Placebo Part 1
-0.3
± 0.3
Percentage of Participants Who Responded With a Decrease of at Least 30% in the Mean Brief Pain Inventory Worst Pain Numeric Rating Scale Score Among Participants Receiving Pexidartinib Compared With Those on Placebo at Week 25Secondary· Week 25
The Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Group
Value
95% CI
Pexidartinib Part 1
31.1
Placebo Part 1
15.3
Number of Responders to Pexidartinib With and Without Disease ProgressionSecondary· By Week 96
Duration of response (DOR) based on RECIST 1.1 is defined as the date of the first recorded response to the first date of documented disease progression. The overall number of responses and the number of participants with and without disease progression was assessed.
Number of responses
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
23
Placebo Part 1, Pexidartinib Part 2
12
All Pexidartinib Treated
35
Week 12 (Day 84); Without disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
23
Placebo Part 1, Pexidartinib Part 2
12
All Pexidartinib Treated
35
Week 12 (Day 84); With disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Week 24 (Day 168); Without disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
23
Placebo Part 1, Pexidartinib Part 2
12
All Pexidartinib Treated
35
Week 24 (Day 168); With disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Week 48 (Day 336); Without disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
15
Placebo Part 1, Pexidartinib Part 2
9
All Pexidartinib Treated
24
Week 48 (Day 336); With disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
1
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
1
Week 72 (Day 504); Without disease progression
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
9
Placebo Part 1, Pexidartinib Part 2
3
All Pexidartinib Treated
12
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume ScoreSecondary· By Week 120
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
Number of responders
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
34
Placebo Part 1, Pexidartinib Part 2
18
All Pexidartinib Treated
52
Week 12 (Day 84); Without disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
33
Placebo Part 1, Pexidartinib Part 2
18
All Pexidartinib Treated
51
Week 12 (Day 84); With disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Week 24 (Day 168); Without disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
32
Placebo Part 1, Pexidartinib Part 2
18
All Pexidartinib Treated
50
Week 24 (Day 168); With disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Week 48 (Day 336); Without disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
22
Placebo Part 1, Pexidartinib Part 2
13
All Pexidartinib Treated
35
Week 48 (Day 336); With disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
3
Placebo Part 1, Pexidartinib Part 2
1
All Pexidartinib Treated
4
Week 72 (Day 504); Without disease progression
Group
Value
95% CI
Pexidartinib in Part 1 and Part 2
13
Placebo Part 1, Pexidartinib Part 2
3
All Pexidartinib Treated
16
Duration of Response (DOR) Based on RECIST 1.1Secondary· Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Duration of response (DOR) based on RECIST 1.1 is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
NA
31.01 – NA
Placebo Part 1, Pexidartinib Part 2
NA
39.0 – NA
Duration of Response (DOR) Based on Tumor Volume Score (TVS)Secondary· Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Duration of response (DOR) based on TVS is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
52.70
38.60 – NA
Placebo Part 1, Pexidartinib Part 2
NA
NA – NA
Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred TermSecondary· After the first dose of treatment up to 28 days after the last dose
Treatment-emergent adverse events (TEAEs) were defined as adverse events that started or worsened after the first dose of treatment and within 28 days after the last dose. The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 was used to grade adverse events. Any Grade and Grade ≥3 (severe) TEAEs are reported. TEAEs were coded using MedDRA version 17.1.
Any Hair color changes
Group
Value
95% CI
Pexidartinib Part 1
67.2
Placebo Part 1
3.4
Pexidartinib Part 1 and Part 2
73.8
Placebo Part 1, Pexidartinib Part 2
83.3
All Pexidartinib Treated
76.9
Grade ≥3 Hair color changes
Group
Value
95% CI
Pexidartinib Part 1
0
Placebo Part 1
0
Pexidartinib Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Any Pruritis
Group
Value
95% CI
Pexidartinib Part 1
9.8
Placebo Part 1
3.4
Pexidartinib Part 1 and Part 2
16.4
Placebo Part 1, Pexidartinib Part 2
20.0
All Pexidartinib Treated
17.6
Grade ≥3 Pruritis
Group
Value
95% CI
Pexidartinib Part 1
0
Placebo Part 1
0
Pexidartinib Part 1 and Part 2
1.6
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
1.1
Any Rash maculopapular
Group
Value
95% CI
Pexidartinib Part 1
9.8
Placebo Part 1
1.7
Pexidartinib Part 1 and Part 2
14.8
Placebo Part 1, Pexidartinib Part 2
10.0
All Pexidartinib Treated
13.2
Grade ≥3 Rash maculopapular
Group
Value
95% CI
Pexidartinib Part 1
0
Placebo Part 1
0
Pexidartinib Part 1 and Part 2
1.6
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
1.1
Any Pruritis generalized
Group
Value
95% CI
Pexidartinib Part 1
8.2
Placebo Part 1
0
Pexidartinib Part 1 and Part 2
8.2
Placebo Part 1, Pexidartinib Part 2
10.0
All Pexidartinib Treated
8.8
Grade ≥3 Pruritis generalized
Group
Value
95% CI
Pexidartinib Part 1
0
Placebo Part 1
0
Pexidartinib Part 1 and Part 2
0
Placebo Part 1, Pexidartinib Part 2
0
All Pexidartinib Treated
0
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 by Week 49Secondary· By Week 49
Complete (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Complete Response (CR)
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
24.6
Placebo Part 1, Pexidartinib Part 2
23.3
All Pexidartinib Treated
24.2
Partial Response (PR)
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
29.5
Placebo Part 1, Pexidartinib Part 2
30.0
All Pexidartinib Treated
29.7
Response (CR or PR)
Group
Value
95% CI
Pexidartinib Part 1 and Part 2
54.1
Placebo Part 1, Pexidartinib Part 2
53.3
All Pexidartinib Treated
53.8
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were monitored throughout the study from the time the participant signed the informed consent form to 28 days after the final treatment dose, up to 71 months..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Pexidartinib (Part 1)
Serious: 8/61 (13%)
Deaths: 0/61
Placebo (Part 1)
Serious: 1/59 (2%)
Deaths: 0/59
Pexidartinib (Parts 1 and 2)
Serious: 12/61 (20%)
Deaths: 0/61
Placebo (Part 1), Crossover Pexidartinib (Part 2)
Serious: 9/30 (30%)
Deaths: 1/30
All Pexidartinib Treated
Serious: 21/91 (23%)
Deaths: 1/91
Serious adverse events (36 terms)
Reaction
System
Pexidartinib (Part 1)
Placebo (Part 1)
Pexidartinib (Parts 1 and 2)
Placebo (Part 1), Crossove…
All Pexidartinib Treated
Transaminases increased
Investigations
—
—
—
—
—
Liver function test abnormal
Investigations
—
—
—
—
—
Hepatic enzyme abnormal
Investigations
—
—
—
—
—
Blood bilirubin increased
Investigations
—
—
—
—
—
Adenosquamous carcinoma of the cervix
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Rectal adenocarcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Endometrial cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Squamous cell carcinoma of skin
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Cardiac arrest
Cardiac disorders
—
—
—
—
—
Asthma
Respiratory, thoracic and mediastinal disorders
—
—
—
—
—
Migraine
Nervous system disorders
—
—
—
—
—
Local swelling
General disorders
—
—
—
—
—
Non-cardiac chest pain
General disorders
—
—
—
—
—
Hepatoxicity
Hepatobiliary disorders
—
—
—
—
—
Liver disorder
Hepatobiliary disorders
—
—
—
—
—
Rash papular
Skin and subcutaneous tissue disorders
—
—
—
—
—
Rash
Skin and subcutaneous tissue disorders
—
—
—
—
—
Neck pain
Musculoskeletal and connective tissue disorders
—
—
—
—
—
Hepatitis A
Infections and infestations
—
—
—
—
—
Hepatitis E
Infections and infestations
—
—
—
—
—
Lymphangitis
Infections and infestations
—
—
—
—
—
Lipoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Rectal cancer stage II
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Squamous cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good.
The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS).
The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2.
Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04703322 — A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan
· Phase 2
· active not recruiting
NCT04526704 — Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously
· Phase 4
· completed
NCT04488822 — A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT
· Phase 3
· active not recruiting
NCT04223635 — Study of Pexidartinib in Participants With Moderate Hepatic Impairment Compared With Healthy Participants
· EARLY_PHASE1
· completed
NCT03291288 — Effect of Pexidartinib on the Way the Body Processes CYP3A4 and CYP2C9 Substrates (Pharmacokinetics)
· Phase 1
· completed
Other Daiichi Sankyo trials
Trials by the same sponsor.
NCT07474649 — A Study of Bempedoic Acid/Ezetimibe/High-intensity Statin in Patients Without Cardiovascular Events
· Phase 3
· not yet recruiting
NCT07206472 — A Study of Bempedoic Acid or Its Single-pill Combination Therapy With Ezetimibe in Patients With Primary Hypercholestero
· active not recruiting
NCT07220616 — A First-in-Human Trial of DS3790a in Participants With Hematological Malignancies
· Phase 1, PHASE2
· recruiting
NCT07268625 — Evaluating Bioequivalence of a Fixed Dose Combination Versus Individual Tablets of Bempedoic Acid / Ezetimibe, and Atorv
· Phase 1
· completed
NCT07244341 — A Study of Valemetostat (DS-3201b) in Combination With Darolutamide in Metastatic Castration Resistant Prostate Cancer (
· Phase 1
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Daiichi Sankyo
Last refreshed: 11 May 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02371369.