A Phase 2 interventional study of Pexidartinib in Tenosynovial Giant Cell Tumor, sponsored by Daiichi Sankyo Co., Ltd.. Completed at 6 sites in Japan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2026-06-24.
Sponsored by Daiichi Sankyo Co., Ltd. · Phase 2, Interventional, and Treatment
This phase 2, multicenter, two-part, open-label, single-arm study will be conducted in Japan and will evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of pexidartinib in adult participants with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitation and not amenable to improvement with surgery.
This study will consist of 2 parts. In Part 1, pexidartinib 800 mg/day (400 mg twice a day [BID]) will be administered on an empty stomach and tolerability and PK of pexidartinib will be evaluated to determine the initiation of Part 2. In Part 2, pexidartinib 800 mg/day (400 mg BID) will be administered on an empty stomach and efficacy, safety, and PK of pexidartinib will be evaluated.
22 studies on the registry are indexed under Giant Cell Tumor of Tendon Sheath; 5 are open to participants now.
This study's enrollment of 9 is below the median of 34 across 20 interventional studies indexed under Giant Cell Tumor of Tendon Sheath.
Browse Giant Cell Tumor of Tendon Sheath studies →Daiichi Sankyo Co., Ltd. is the lead sponsor of 73 studies on the registry; none are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants with TGCT who will receive oral pexidartinib 800 mg (400 mg twice daily \[BID\]).
Drug: Pexidartinib
400 mg twice daily for a total daily dose of 800 mg (each capsule contains 200 mg of pexidartinib for oral administration)
Also known as: TURALIO™, PLX3397
Dose-limiting Toxicity (DLT) in Part 1
The number of participants with any-grade DLT treatment-emergent adverse events (TEAEs) were assessed.
Time frame: Cycle 1, Day 1 to Cycle 1, Day 28 (each cycle is 28 days)
Pharmacokinetic Parameter Maximum Concentration of Pexidartinib and ZAAD-1006a (Cmax) in Part 1
Pharmacokinetic parameters were assessed using non-compartmental methods. Cmax is the maximum concentration of pexidartinib and ZAAD-1006a in plasma.
Time frame: 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 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
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.
Time frame: 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 Parameter Area Under the Concentration-Time Curve Up to Infinity of Pexidartinib and ZAAD-1006a (AUCinf) in Part 1
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.
Time frame: 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 Parameter Time to Reach Maximum Concentration of Pexidartinib and ZAAD-1006a (Tmax) in Part 1
Pharmacokinetic parameters were assessed using non-compartmental methods. Tmax is the time to reach maximum concentration of pexidartinib and ZAAD-1006a in plasma.
Time frame: 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)
Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST Version 1.1) in Part 2
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.
Time frame: Week 25
ORR Based on Tumor Volume Score (TVS) in Part 2
ORR will be assessed by centrally reviewed MRI scan based on TVS.
Time frame: Week 25
Range of Motion (ROM) in Part 2
Mean change from baseline in ROM of the affected joint, relative to a reference standard for the same joint will be assessed.
Time frame: Week 25
Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale in Part 2
Mean change from baseline score in the PROMIS Physical Function Scale will be assessed.
Time frame: Week 25
Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) in Part 2
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).
Time frame: Week 25
Best Overall Response (BOR) Based on RECIST Version 1.1 in Part 2
BOR will be assessed by centrally reviewed magnetic resonance imaging (MRI) scan based on RECIST version 1.1.
Time frame: Post Week 25 visit or following discontinuation from Part 2 of the study (whichever occurs first), up to approximately 1 year 6 months
BOR Based on TVS in Part 2
BOR will be assessed by centrally reviewed MRI scan based on TVS.
Time frame: Post Week 25 visit or following discontinuation from Part 2 of the study (whichever occurs first), up to approximately 1 year 6 months
Duration of Response (DoR) Based on RECIST Version 1.1 in Part 2
DoR will be assessed by centrally reviewed MRI scan based on RECIST version 1.1.
Time frame: Post Week 25 visit or following discontinuation from Part 2 of the study (whichever occurs first), up to approximately 1 year 6 months
DoR Based on TVS
DoR will be assessed by centrally reviewed MRI scan based on TVS.
Time frame: Post Week 25 visit or following discontinuation from Part 2 of the study (whichever occurs first), up to approximately 1 year 6 months
Number of Participants Reporting Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events
Time frame: Baseline up to 28 (+/- 7 days) after last dose, up to approximately 6 years 9 months
A total of 9 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study. These results are based on Part 1 of the study. All safety and pharmacokinetic primary outcome analysis data for Part 1 are reported. Part 2 of the study was not conducted. Eight patients remain in the study and are being followed until study completion. Only the final safety data of the patients who remain on study will be reported at the final database cut (estimated May 31, 2026).
| Milestone | Pexidartinib |
|---|---|
| Started | 9 |
| Completed | 0 |
| Not completed | 9 |
| Withdrew: Ongoing | 8 |
| Withdrew: Withdrawal by subject | 1 |
The number of participants with any-grade DLT treatment-emergent adverse events (TEAEs) were assessed.
| Participants | Pexidartinib |
|---|---|
| Any Grade DLT TEAE | 2 |
| Any Grade Hepatic function abnormal | 1 |
| Any Grade Alanine aminotransferase increased | 1 |
| Any Grade Aspartate aminotransferase increased | 1 |
| Any Grade Blood alkaline phosphatase increased | 1 |
| Any Grade Gamma-glutamyltransferase increased | 1 |
Pharmacokinetic parameters were assessed using non-compartmental methods. Cmax is the maximum concentration of pexidartinib and ZAAD-1006a in plasma.
| ng/mL | Pexidartinib |
|---|---|
| Pexidartinib, C1D1 | 7920 ± 1720 |
| Pexidartinib, C1D15 | 8810 ± 1110 |
| ZAAD-1006a, C1D1 | 8000 ± 1990 |
| ZAAD-1006a, C1D15 | 11700 ± 3340 |
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.
| h*ng/mL | Pexidartinib |
|---|---|
| Pexidartinib, C1D1 | 62000 ± 27.3 |
| ZAAD-1006a, C1D1 | 90900 ± 48.4 |
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.
| h*ng/mL | Pexidartinib |
|---|---|
| Pexidartinib, C1D1 | 8700 ± 28.1 |
| ZAAD-1006a, C1D1 | 195000 ± 40.2 |
Pharmacokinetic parameters were assessed using non-compartmental methods. Tmax is the time to reach maximum concentration of pexidartinib and ZAAD-1006a in plasma.
| hours | Pexidartinib |
|---|---|
| Pexidartinib, C1D1 | 2.05 (1.97 to 2.10) |
| Pexidartinib, C1D15 | 2.10 (1.92 to 2.15) |
| ZAAD-1006a, C1D1 | 3.97 (1.97 to 7.92) |
| ZAAD-1006a, C1D15 | 3.83 (2.10 to 3.92) |
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.
No measurements were reported for this outcome.
ORR will be assessed by centrally reviewed MRI scan based on TVS.
No measurements were reported for this outcome.
Mean change from baseline in ROM of the affected joint, relative to a reference standard for the same joint will be assessed.
No measurements were reported for this outcome.
Mean change from baseline score in the PROMIS Physical Function Scale will be assessed.
No measurements were reported for this outcome.
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).
No measurements were reported for this outcome.
BOR will be assessed by centrally reviewed magnetic resonance imaging (MRI) scan based on RECIST version 1.1.
No measurements were reported for this outcome.
BOR will be assessed by centrally reviewed MRI scan based on TVS.
No measurements were reported for this outcome.
DoR will be assessed by centrally reviewed MRI scan based on RECIST version 1.1.
No measurements were reported for this outcome.
DoR will be assessed by centrally reviewed MRI scan based on TVS.
No measurements were reported for this outcome.
Results for this outcome have not been posted.
Collected over Adverse events were collected from screening to 28 days (+/-7 days) after last dose of study treatment, up to 3 years 7 months (data cut-off date).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pexidartinib | 0/9 (0%) | 0/9 (0%) | 9/9 (100%) |
| Event | Pexidartinib |
|---|---|
| Hair colour changesSkin and subcutaneous tissue disorders | 7/9 |
| Aspartate aminotransferase increasedInvestigations | 7/9 |
| PruritusSkin and subcutaneous tissue disorders | 4/9 |
| Alanine aminotransferase increasedInvestigations | 4/9 |
| Blood lactate dehydrogenase increasedInvestigations | 3/9 |
| Gamma-glutamyltransferase increasedInvestigations | 3/9 |
| Blood alkaline phosphatase increasedInvestigations | 2/9 |
| Blood bilirubin increasedInvestigations | 2/9 |
| Blood creatinine phosphokinase increasedInvestigations | 2/9 |
| Blood creatinine increasedInvestigations | 2/9 |
Demographic and baseline characteristics were assessed in the Safety Analysis Set.
| Age, Categorical(Participants) | Pexidartinib |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 9 |
| >=65 years | 0 |
| Age, Continuous(years) | Pexidartinib |
|---|---|
| Mean | 35.9 ± 11.16 |
| Sex: Female, Male(Participants) | Pexidartinib |
|---|---|
| Female | 4 |
| Male | 5 |
| Race (NIH/OMB)(Participants) | Pexidartinib |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 9 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
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Studies of the drug tested here: Pexidartinib
Giant Cell Tumor of Tendon Sheath →