Trial · NCT04703322
A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan
NCT04703322, Phase 2, Completed, Daiichi Sankyo Co., Ltd.; results first posted 2025-03-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan. Trials, record NCT04703322. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04703322
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan
- NCT number
- NCT04703322
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- Daiichi Sankyo Co., Ltd.
- Conditions
- Tenosynovial Giant Cell Tumor
- Interventions
- Pexidartinib
- First posted
- 2021-01-11
- Last updated
- 2026-06-24
- Results first posted
- 2025-03-03
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Dose-limiting Toxicity (DLT) in Part 1 | Cycle 1, Day 1 to Cycle 1, Day 28 (each cycle is 28 days) | The number of participants with any-grade DLT treatment-emergent adverse events (TEAEs) were assessed. |
| Primary | Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hr post-dose of Cycle 1, Day 1 (C1D1) and Predose, 0.5 hr, 1 hr, 2 hr, 4 hr, and 6 hr postdose of Cycle 1, Day 15 (C1D15) (each cycle is 28 days) | Pharmacokinetic parameters were assessed using non-compartmental methods. Cmax is the maximum concentration of pexidartinib and ZAAD-1006a in plasma. |
| Primary | Pharmacokinetic Parameter Area Under the Concentration-Time Curve From Time Zero to Time of Last Measurable Concentration of Pexidartinib and ZAAD-1006a (AUClast) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hrs post-dose of Cycle 1, Day 1 (C1D1) (each cycle is 28 days) | Pharmacokinetic parameters were assessed using non-compartmental methods. AUClast is the area under the concentration-time curve from time zero to time of last measurable concentration of pexidartinib and ZAAD-1006a in plasma. |
| Primary | Pharmacokinetic Parameter Area Under the Concentration-Time Curve Up to Infinity of Pexidartinib and ZAAD-1006a (AUCinf) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hrs post-dose of Cycle 1, Day 1 (C1D1) (each cycle is 28 days) | Pharmacokinetic parameters were assessed using non-compartmental methods. AUCinf is the area under the concentration-time curve up to infinity of pexidartinib and ZAAD-1006a in plasma. |
| Primary | Pharmacokinetic Parameter Time to Reach Maximum Concentration of Pexidartinib and ZAAD-1006a (Tmax) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hr post-dose of Cycle 1, Day 1 (C1D1) and Predose, 0.5 hr, 1 hr, 2 hr, 4 hr, and 6 hr postdose of Cycle 1, Day 15 (C1D15) (each cycle is 28 days) | Pharmacokinetic parameters were assessed using non-compartmental methods. Tmax is the time to reach maximum concentration of pexidartinib and ZAAD-1006a in plasma. |
| Primary | Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST Version 1.1) in Part 2 | Week 25 | ORR will be assessed by centrally reviewed MRI scan based on RECIST version 1.1. Complete response (CR) was defined as a disappearance of all target lesions, partial response (PR) was defined as at least a 30% decrease in the sum of diameters of target lesions, and stable disease (SD) was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD; at least a 20% increase in the sum of diameters of target lesions. |
| Secondary | ORR Based on Tumor Volume Score (TVS) in Part 2 | Week 25 | ORR will be assessed by centrally reviewed MRI scan based on TVS. |
| Secondary | Range of Motion (ROM) in Part 2 | Week 25 | Mean change from baseline in ROM of the affected joint, relative to a reference standard for the same joint will be assessed. |
| Secondary | Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale in Part 2 | Week 25 | Mean change from baseline score in the PROMIS Physical Function Scale will be assessed. |
| Secondary | Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) in Part 2 | Week 25 | Proportion of responders will be assessed based on the BPI Worst Pain NRS item and analgesic use by BPI-30 definition (ie, 30% or more improvement in average NRS). |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Dose-limiting Toxicity (DLT) in Part 1 | Cycle 1, Day 1 to Cycle 1, Day 28 (each cycle is 28 days) | All DLT TEAEs were assessed in the DLT Evaluable Set. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hr post-dose of Cycle 1, Day 1 (C1D1) and Predose, 0.5 hr, 1 hr, 2 hr, 4 hr, and 6 hr postdose of Cycle 1, Day 15 (C1D15) (each cycle is 28 days) | Pharmacokinetic data were assessed in the Pharmacokinetic Analysis Set. | ng/mL | MEAN |
| PRIMARY | Pharmacokinetic Parameter Area Under the Concentration-Time Curve From Time Zero to Time of Last Measurable Concentration of Pexidartinib and ZAAD-1006a (AUClast) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hrs post-dose of Cycle 1, Day 1 (C1D1) (each cycle is 28 days) | Pharmacokinetic data were assessed in the Pharmacokinetic Analysis Set. | h*ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Pharmacokinetic Parameter Area Under the Concentration-Time Curve Up to Infinity of Pexidartinib and ZAAD-1006a (AUCinf) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hrs post-dose of Cycle 1, Day 1 (C1D1) (each cycle is 28 days) | Pharmacokinetic data were assessed in participants with available data in the Pharmacokinetic Analysis Set. | h*ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Pharmacokinetic Parameter Time to Reach Maximum Concentration of Pexidartinib and ZAAD-1006a (Tmax) in Part 1 | Predose, 0.5 hours (hr), 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 24 hr post-dose of Cycle 1, Day 1 (C1D1) and Predose, 0.5 hr, 1 hr, 2 hr, 4 hr, and 6 hr postdose of Cycle 1, Day 15 (C1D15) (each cycle is 28 days) | Pharmacokinetic data were assessed in the Pharmacokinetic Analysis Set. | hours | MEDIAN |
| PRIMARY | Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST Version 1.1) in Part 2 | Week 25 | Part 2 of this study was not conducted and efficacy data are not available for reporting. Only the final safety data of patients who remain in the study will be reported at the final database cut (estimated May 31, 2026). | ||
| SECONDARY | ORR Based on Tumor Volume Score (TVS) in Part 2 | Week 25 | Part 2 of this study was not conducted and efficacy data are not available for reporting. Only the final safety data of patients who remain in the study will be reported at the final database cut (estimated May 31, 2026). | ||
| SECONDARY | Range of Motion (ROM) in Part 2 | Week 25 | Part 2 of this study was not conducted and efficacy data are not available for reporting. Only the final safety data of patients who remain in the study will be reported at the final database cut (estimated May 31, 2026). | ||
| SECONDARY | Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale in Part 2 | Week 25 | Part 2 of this study was not conducted and efficacy data are not available for reporting. Only the final safety data of patients who remain in the study will be reported at the final database cut (estimated May 31, 2026). | ||
| SECONDARY | Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) in Part 2 | Week 25 | Part 2 of this study was not conducted and efficacy data are not available for reporting. Only the final safety data of patients who remain in the study will be reported at the final database cut (estimated May 31, 2026). |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade DLT TEAE | 2 | Participants | |
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade Hepatic function abnormal | 1 | Participants | |
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade Alanine aminotransferase increased | 1 | Participants | |
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade Aspartate aminotransferase increased | 1 | Participants | |
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade Blood alkaline phosphatase increased | 1 | Participants | |
| Dose-limiting Toxicity (DLT) in Part 1 | Pexidartinib | Any Grade Gamma-glutamyltransferase increased | 1 | Participants | |
| Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Pexidartinib | Pexidartinib, C1D1 | 7920 | Standard Deviation 1720 | ng/mL |
| Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Pexidartinib | Pexidartinib, C1D15 | 8810 | Standard Deviation 1110 | ng/mL |
| Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Pexidartinib | ZAAD-1006a, C1D1 | 8000 | Standard Deviation 1990 | ng/mL |
| Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1 | Pexidartinib | ZAAD-1006a, C1D15 | 11700 | Standard Deviation 3340 | ng/mL |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pexidartinib | 9 | |
| Overall Study | COMPLETED | Pexidartinib | 0 | |
| Overall Study | NOT COMPLETED | Pexidartinib | 9 |
Source ClinicalTrials.gov
Adverse events36
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 | Hair colour changes | Skin and subcutaneous tissue disorders | Pexidartinib | 7 | 9 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Pexidartinib | 4 | 9 | |
| other | Dermatitis | Skin and subcutaneous tissue disorders | Pexidartinib | 1 | 9 | |
| other | Eczema | Skin and subcutaneous tissue disorders | Pexidartinib | 1 | 9 | |
| other | Skin discolouration | Skin and subcutaneous tissue disorders | Pexidartinib | 1 | 9 | |
| other | Aspartate aminotransferase increased | Investigations | Pexidartinib | 7 | 9 | |
| other | Alanine aminotransferase increased | Investigations | Pexidartinib | 4 | 9 | |
| other | Blood lactate dehydrogenase increased | Investigations | Pexidartinib | 3 | 9 | |
| other | Gamma-glutamyltransferase increased | Investigations | Pexidartinib | 3 | 9 | |
| other | Blood alkaline phosphatase increased | Investigations | Pexidartinib | 2 | 9 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pexidartinib | 0 | 9 |
| Total, other adverse events | Pexidartinib | 9 | 9 |
| Total, serious adverse events | Pexidartinib | 0 | 9 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT04703322
GET https://conductscience.org/api/trials/v1/NCT04703322/events?page=2