Trial · NCT02371369
Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)
NCT02371369, Phase 3, Completed, Daiichi Sankyo; results first posted 2020-01-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS). Trials, record NCT02371369. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02371369
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)
- NCT number
- NCT02371369
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 120 participants, actual
- Lead sponsor
- Daiichi Sankyo
- Conditions
- Pigmented Villonodular Synovitis, Giant Cell Tumors of the Tendon Sheath, Tenosynovial Giant Cell Tumor
- Interventions
- Pexidartinib, Placebo
- First posted
- 2015-02-25
- Last updated
- 2022-05-11
- Other registries' IDs
- eudract:2014-000148-14
- Results first posted
- 2020-01-10
- Limitations and caveats
- Enrollment was stopped on 30 Sep 2016; no new participants received the study drug. After Part 1, those who wished to continue were un-blinded; those on placebo were discontinued.
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 25 | 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. |
| Secondary | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | 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. |
| Secondary | 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 25 | 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 of 0 indicated no evidence of tumor. |
| Secondary | 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 25 | 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. |
| Secondary | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | 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). |
| Secondary | 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 25 | 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). |
| Secondary | Number of Responders to Pexidartinib With and Without Disease Progression | 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. |
| Secondary | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | 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. |
| Secondary | Duration of Response (DOR) Based on RECIST 1.1 | 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. |
| Secondary | Duration of Response (DOR) Based on Tumor Volume Score (TVS) | 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. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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 25 | Week 25 | Best overall response was assessed in the Intent-to-Treat (ITT) population. | Percentage of participants | NUMBER |
| SECONDARY | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Baseline, Week 13, and Week 25 | Range of motion was assessed in the ITT population in participants where data were available. | degrees | LEAST_SQUARES_MEAN |
| SECONDARY | 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 25 | Week 25 | Best overall response was assessed in the ITT population. | Percentage of participants | NUMBER |
| SECONDARY | 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 25 | at Week 9 , Week 17, and Week 25 | Physical function was assessed in the ITT population in participants where data were available. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Baseline, Week 9, Week 17, and Week 25 | Worst stiffness was assessed in the ITT population. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | 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 25 | Week 25 | Worst pain was assessed in the ITT population. | percentage of participants | NUMBER |
| SECONDARY | Number of Responders to Pexidartinib With and Without Disease Progression | By Week 96 | The overall number of responses and the number of participants with and without disease progression were assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only, All Pexidartinib Treated participants, . Participants randomized to Placebo Part 1 only were not analyzed. | participants | NUMBER |
| SECONDARY | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | By Week 120 | The overall number of responses and the number of participants with and without disease progression was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only were not analyzed. | participants | NUMBER |
| SECONDARY | Duration of Response (DOR) Based on RECIST 1.1 | 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) | DOR was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only. Participants randomized to Placebo Part 1 only were not analyzed. DOR was based on numbers of responders only. | months | MEDIAN |
| SECONDARY | Duration of Response (DOR) Based on Tumor Volume Score (TVS) | 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) | DOR was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only. Participants randomized to Placebo Part 1 only were not analyzed. DOR was based on numbers of responders only. | months | MEDIAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group555
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
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 25 | Pexidartinib Part 1 | Complete Response (CR) | 14.8 | Percentage of participants | |
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 25 | Placebo Part 1 | Complete Response (CR) | 0 | Percentage of participants | |
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 25 | Pexidartinib Part 1 | Partial Response (PR) | 24.6 | Percentage of participants | |
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 25 | Placebo Part 1 | Partial Response (PR) | 0 | Percentage of participants | |
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 25 | Pexidartinib Part 1 | Response (CR or PR) | 39.3 | Percentage of participants | |
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 25 | Placebo Part 1 | Response (CR or PR) | 0 | Percentage of participants | |
| Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Pexidartinib Part 1 | Baseline | 62.5 | Standard Error 3.2 | degrees |
| Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Placebo Part 1 | Baseline | 62.9 | Standard Error 2.9 | degrees |
| Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Pexidartinib Part 1 | Week 13 | 13.0 | Standard Error 2.3 | degrees |
| Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Placebo Part 1 | Week 13 | 4.8 | Standard Error 2.6 | degrees |
1 to 10 of 555Source ClinicalTrials.gov
See all 555 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 | STARTED | Pexidartinib Part 1, Then Pexidartinib Part 2 | 61 | |
| Part 1 | STARTED | Placebo Part 1, Then Pexidartinib Part 2 | 59 | |
| Part 1 | COMPLETED | Pexidartinib Part 1, Then Pexidartinib Part 2 | 52 | |
| Part 1 | COMPLETED | Placebo Part 1, Then Pexidartinib Part 2 | 48 | |
| Part 1 | NOT COMPLETED | Pexidartinib Part 1, Then Pexidartinib Part 2 | 9 | |
| Part 1 | NOT COMPLETED | Placebo Part 1, Then Pexidartinib Part 2 | 11 | |
| Part 2 | STARTED | Pexidartinib Part 1, Then Pexidartinib Part 2 | 48 | |
| Part 2 | STARTED | Placebo Part 1, Then Pexidartinib Part 2 | 30 | |
| Part 2 | COMPLETED | Pexidartinib Part 1, Then Pexidartinib Part 2 | 0 | |
| Part 2 | COMPLETED | Placebo Part 1, Then Pexidartinib Part 2 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events625
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypertension | Vascular disorders | Pexidartinib (Part 1) | 9 | 61 | 15 |
| other | Hypertension | Vascular disorders | Placebo (Part 1) | 6 | 59 | 6 |
| other | Hypertension | Vascular disorders | Pexidartinib (Parts 1 and 2) | 14 | 61 | 24 |
| other | Hypertension | Vascular disorders | Placebo (Part 1), Crossover Pexidartinib (Part 2) | 12 | 30 | 21 |
| other | Hypertension | Vascular disorders | All Pexidartinib Treated | 26 | 91 | 45 |
| other | Hypotension | Vascular disorders | Pexidartinib (Part 1) | 0 | 61 | 0 |
| other | Hypotension | Vascular disorders | Placebo (Part 1) | 0 | 59 | 0 |
| other | Hypotension | Vascular disorders | Pexidartinib (Parts 1 and 2) | 0 | 61 | 0 |
| other | Hypotension | Vascular disorders | Placebo (Part 1), Crossover Pexidartinib (Part 2) | 2 | 30 | 2 |
| other | Hypotension | Vascular disorders | All Pexidartinib Treated | 2 | 91 | 2 |
1 to 10 of 625Source ClinicalTrials.gov
See all 625 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pexidartinib (Part 1) | 0 | 61 |
| Total, all-cause mortality | Placebo (Part 1) | 0 | 59 |
| Total, all-cause mortality | Pexidartinib (Parts 1 and 2) | 0 | 61 |
| Total, all-cause mortality | Placebo (Part 1), Crossover Pexidartinib (Part 2) | 1 | 30 |
| Total, all-cause mortality | All Pexidartinib Treated | 1 | 91 |
| Total, other adverse events | Pexidartinib (Part 1) | 60 | 61 |
| Total, other adverse events | Placebo (Part 1) | 55 | 59 |
| Total, other adverse events | Pexidartinib (Parts 1 and 2) | 61 | 61 |
| Total, other adverse events | Placebo (Part 1), Crossover Pexidartinib (Part 2) | 30 | 30 |
| Total, other adverse events | All Pexidartinib Treated | 91 | 91 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers6
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02371369
GET https://conductscience.org/api/trials/v1/NCT02371369/results?page=2