Trial · NCT04488822
A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT
NCT04488822, Phase 3, Active not recruiting, Daiichi Sankyo Co., Ltd.; results first posted 2023-04-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT. Trials, record NCT04488822. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04488822
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT
- NCT number
- NCT04488822
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 40 participants, actual
- Lead sponsor
- Daiichi Sankyo Co., Ltd.
- Conditions
- Tenosynovial Giant Cell Tumor
- Interventions
- Pexidartinib
- First posted
- 2020-07-28
- Last updated
- 2026-06-26
- Results first posted
- 2023-04-27
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | At Week 25 postdose | For the assessment of tumor response, participants were classified into the best of the following tumor response categories based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) by centrally reviewed MRI scan. Complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; and overall response rate (ORR), defined as CR+PR, are presented. |
| Secondary | Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | At Week 25 postdose | For the assessment of tumor response, participants were classified into the best of the following tumor response categories based on Tumor Volume Score (TVS) by centrally reviewed MRI scan. 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. Complete response (CR), disappearance of lesion; partial response (PR), at least 50% decrease in volume score relative to baseline; overall response rate (ORR), defined as CR+PR, are presented. |
| Secondary | Mean Percent Change From Baseline for Range of Motion (ROM) Score in Participants Receiving Pexidartinib | At Week 25 postdose | Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. The relative ROM was calculated (expressed in percent) as follows: Relative ROM = 100 x (absolute ROM measured)/(reference ROM standard). The change from baseline in relative ROM at Week 25 was calculated as the difference in relative ROM at Week 25 visit minus relative ROM at Screening visit. |
| Secondary | Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib | At Week 25 postdose | The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess the physical function of the upper and lower limbs. The scale ranges 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 | Best Overall Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan | From baseline until disease progression or death (whichever occurs first), up to approximately 29 months | Best overall response (CR or PR) of pexidartinib based on RECIST 1.1 and TVS are summarized. Tumor categories for RECIST v1.1 were as follows: complete response (CR), disappearance of all target lesions; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions; stable disease (SD), neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions; and not evaluable (NE). Tumor categories for TVS were as follows: complete response (CR), disappearance of all target lesions; partial response (PR), at least a 50% decrease in volume score relative to baseline; stable disease (SD), does not meet any of the prior criteria based on score during study; progressive disease (PD), at least a 30% increase in in volume relative to lowest score during the study whether at baseline or some other visit; and not evaluable (NE). |
| Secondary | Duration of Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan | Time from the date of first documented response until documented disease progression or death from any cause (whichever occurs first), up to approximately 29 months | Duration of response (DOR) is defined as the date of the first recorded response to the first date of documented disease progression. |
| Secondary | Number of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Up to approximately 29 months | A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug). |
| Secondary | Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | AUC(0-6h) was calculated using standard non-compartmental analysis. AUC(0-6h) for Pexidartinib and primary metabolite, ZAAD-1006a, are presented. |
| Secondary | Maximum Plasma Concentration (Cmax) in Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | Cmax was calculated using standard non-compartmental analysis. Cmax for Pexidartinib and primary metabolite, ZAAD-1006a, are presented. |
| Secondary | Time to Reach Maximum Observed Plasma Concentration (Tmax) of Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | Tmax was calculated using standard non-compartmental analysis. Tmax for Pexidartinib and primary metabolite, ZAAD-1006a, are presented. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | At Week 25 postdose | ORR based on RECIST 1.1 was assessed in the Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | At Week 25 postdose | ORR based on TVS was assessed in the Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Percent Change From Baseline for Range of Motion (ROM) Score in Participants Receiving Pexidartinib | At Week 25 postdose | Mean Percent Change From Baseline for Range of Motion (ROM) was assessed in Full Analysis Set; however, participant assessments falling outside the visit window were excluded from the change from baseline analysis. | Percent Change | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib | At Week 25 postdose | Mean Change From PROMIS was assessed in the Full Analysis Set; however, participant assessments falling outside the visit window were excluded from the change from baseline analysis. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Best Overall Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan | From baseline until disease progression or death (whichever occurs first), up to approximately 29 months | |||
| SECONDARY | Duration of Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan | Time from the date of first documented response until documented disease progression or death from any cause (whichever occurs first), up to approximately 29 months | |||
| SECONDARY | Number of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Up to approximately 29 months | |||
| SECONDARY | Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | AUC(0-6h) was assessed in the Pharmacokinetic Analysis Set. | ng*h/mL | MEAN |
| SECONDARY | Maximum Plasma Concentration (Cmax) in Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | Cmax was assessed in the Pharmacokinetic Analysis Set. | ng/mL | MEAN |
| SECONDARY | Time to Reach Maximum Observed Plasma Concentration (Tmax) of Participants Receiving Pexidartinib | Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days) | Tmax was assessed in the Pharmacokinetic Analysis Set. | hours | MEDIAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | CR | 0 | Participants | |
| Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | PR | 9 | Participants | |
| Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | ORR | 9 | Participants | |
| Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | CR | 0 | Participants | |
| Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | PR | 19 | Participants | |
| Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 | Pexidartinib | ORR | 19 | Participants | |
| Mean Percent Change From Baseline for Range of Motion (ROM) Score in Participants Receiving Pexidartinib | Pexidartinib | 23.05 | Standard Deviation 3.207 | Percent Change | |
| Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib | Pexidartinib | 0.46 | Standard Deviation 1.384 | Score on a scale | |
| Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib | Pexidartinib | Pexidartinib | 51500 | Standard Deviation 28.5 | ng*h/mL |
| Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib | Pexidartinib | ZAAD-1006a | 108000 | Standard Deviation 52.1 | ng*h/mL |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 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 | 40 | |
| Overall Study | COMPLETED | Pexidartinib | 31 | |
| Overall Study | NOT COMPLETED | Pexidartinib | 9 |
Source ClinicalTrials.gov
Adverse events67
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 color changes | Skin and subcutaneous tissue disorders | Pexidartinib | 31 | 40 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Pexidartinib | 19 | 40 | |
| other | Rash | Skin and subcutaneous tissue disorders | Pexidartinib | 17 | 40 | |
| other | Skin discoloration | Skin and subcutaneous tissue disorders | Pexidartinib | 7 | 40 | |
| other | Drug eruption | Skin and subcutaneous tissue disorders | Pexidartinib | 5 | 40 | |
| other | Hair texture abnormal | Skin and subcutaneous tissue disorders | Pexidartinib | 5 | 40 | |
| other | Alopecia | Skin and subcutaneous tissue disorders | Pexidartinib | 2 | 40 | |
| other | Urticaria | Skin and subcutaneous tissue disorders | Pexidartinib | 2 | 40 | |
| other | Alanine aminotransferase increased | Investigations | Pexidartinib | 24 | 40 | |
| other | Aspartate aminotransferase increased | Investigations | Pexidartinib | 23 | 40 |
1 to 10 of 67Source ClinicalTrials.gov
See all 67 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pexidartinib | 0 | 40 |
| Total, other adverse events | Pexidartinib | 40 | 40 |
| Total, serious adverse events | Pexidartinib | 6 | 40 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40841499 | DERIVED |
Source PubMed
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/NCT04488822
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