Pigmented Villonodular Synovitis, Giant Cell Tumors of the Tendon Sheath, Tenosynovial Giant Cell Tumor
Conditions
Keywords
PLX3397, Pexidartinib, Colony Stimulating Factor 1 Receptor (CSF-1R) inhibitor
Brief summary
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.
Interventions
DRUGPlacebo
Placebo capsule matching pexidartinib capsule for oral administration
DRUGPexidartinib
Each capsule contains 200 mg of pexidartinib for oral administration
Sponsors
Daiichi Sankyo
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) in Part 1, Open-label (no masking) in Part 2
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years. 2. A diagnosis of PVNS or GCT-TS (i) that has been histologically confirmed either by a pathologist at the treating institution or a central pathologist, and (ii) where surgical resection would be associated with potentially worsening functional limitation or severe morbidity (locally advanced disease), with morbidity determined consensually by qualified personnel (eg, two surgeons or a multi-disciplinary tumor board). 3. Measurable disease of at least 2 cm and otherwise based on RECIST 1.1, assessed from MRI scans by a central radiologist. 4. Symptomatic disease because of active PVNS or GCT-TS, defined as one or more of the following: 1. a worst pain of at least 4 at any time during the week preceding the Screening Visit (based on scale of 0 to 10, with 10 representing pain as bad as you can imagine). 2. a worst stiffness of at least 4 at any time during the week preceding the Screening Visit (based on a scale of 0 to 10, with 10 representing stiffness as bad as you can imagine). 5. Stable prescription of analgesic regimen during the 2 weeks prior to randomization. 6. During the 2 weeks prior to randomization, at least 4 of 7 consecutive days of Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) items and Worst Stiffness NRS items completed correctly. 7. Women of childbearing potential must have a negative serum pregnancy test within the 14-day period prior to randomization. (Where demanded by local regulations, this test may be required within 72 hours of randomization.) 8. Males and females of childbearing potential are permitted in the study so long as they consent to avoid getting their partner pregnant or becoming pregnant, respectively, by using a highly effective contraception method, as described below, throughout the study and for up to 90 days after completion. Highly effective methods of contraception include: intra-uterine device (non-hormonal or hormonal), bilateral tubal occlusion, vasectomy, sexual abstinence, or barrier methods (eg, condom, diaphragm) used in combination with hormonal methods associated with inhibition of ovulation. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥ 1 year. Women who have documentation of at least 12 months of spontaneous amenorrhea and have a follicle stimulating hormone (FSH) level \> 40 milli-International units (mIU/mL) will be considered postmenopausal. 9. Adequate hematologic, hepatic, and renal function, defined by: * Absolute neutrophil count ≥ 1.5 × 10\^9/L * aspartate aminotransferase/alanine (AST/ALT) ≤ 1.5 × upper limit of normal (ULN) * Hemoglobin \> 10 g/dL * Total bilirubin ≤ 1.5 × ULN * Platelet count ≥ 100 × 10\^9/L * Serum creatinine ≤ 1.5 × ULN 10. Willingness and ability to complete the Worst Pain NRS item, Worst Stiffness NRS item, Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale, and other self-assessment instruments throughout the study. 11. Willingness and ability to use an electronic diary. 12. Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.
Exclusion criteria
1. Investigational drug use within 28 days of randomization. 2. Previous use of pexidartinib or any biologic treatment targeting CSF-1 or the CSF-1R; previous use of oral tyrosine kinase inhibitors, eg, imatinib or nilotinib, are allowed. 3. Active cancer (either concurrent or within the last year of starting study treatment) that requires therapy (eg, surgical, chemotherapy, or radiation therapy), with the exception of adequately treated basal or squamous cell carcinoma of the skin, melanoma in-situ, carcinoma in-situ of the cervix or breast, or prostate carcinoma with a prostate-specific antigen value \<0.2 ng/mL. 4. Known metastatic PVNS/GCT-TS. 5. Active or chronic infection with hepatitis C virus (HCV) or hepatitis B virus or known active or chronic infection with human immunodeficiency virus. 6. Known active tuberculosis. 7. Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history that, in the Investigator's opinion, would likely interfere with the person's study participation or the interpretation of his or her results. 8. Women who are breastfeeding. 9. A screening Fridericia corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women). 10. MRI contraindications. 11. History of hypersensitivity to any excipients in the investigational product. 12. Inability to swallow capsules.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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). |
| 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. |
| 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. |
| 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. |
| 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. |
| Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | 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. |
| 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. |
| 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. |
| 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). |
Other
| Measure | Time frame | Description |
|---|---|---|
| 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 by Week 49 | By Week 49 | 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. |
| Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline, Week 25, and Week 49 | 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). |
| Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | By Week 49 | The Brief Pain Inventory (BPI) Worst Pain 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). |
| 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 by Week 49 | By Week 49 | Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score 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. |
| 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 49 | 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. |
| Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | By Week 49 | 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. |
Countries
Australia, Canada, Denmark, France, Germany, Hungary, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Participant flow
Recruitment details
Part 1 was a double-blind, randomized, Pexidartinib or placebo in participants with symptomatic TGCT for whom surgical resection would be associated with potentially worsening functional limitation or severe morbidity. Part 2 is a long-term treatment phase in which all eligible participants received open-label Pexidartinib.
Pre-assignment details
Participants were screened for inclusion and exclusion criteria. Screening procedures were performed after consent was obtained and within the 42 days before the first dose of study drug, unless otherwise noted.
Participants by arm
| Arm | Count |
|---|---|
| Pexidartinib Part 1, Then Pexidartinib Part 2 Participants received Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration | 61 |
| Placebo Part 1, Then Pexidartinib Part 2 Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 of study. Eligible participants from this group also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching pexidartinib capsule for oral administration. Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration (Part 2). | 59 |
| Total | 120 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 1 | Adverse Event | 8 | 0 |
| Part 1 | Disease progression | 0 | 1 |
| Part 1 | Physician Decision | 0 | 3 |
| Part 1 | Protocol Violation | 0 | 1 |
| Part 1 | Withdrawal by Subject | 1 | 6 |
| Part 2 | Adverse Event | 6 | 5 |
| Part 2 | Death | 0 | 1 |
| Part 2 | Disease progression | 1 | 0 |
| Part 2 | Investigator decision | 1 | 2 |
| Part 2 | Lost to Follow-up | 1 | 1 |
| Part 2 | Other | 0 | 1 |
| Part 2 | Subject Noncompliance | 1 | 0 |
| Part 2 | Subject transitioned to another DS pexidartinib protocol | 15 | 9 |
| Part 2 | Subject transitioned to commercial supply | 6 | 4 |
| Part 2 | Surgical resection of tumor | 1 | 1 |
| Part 2 | Withdrawal by Subject | 16 | 6 |
Baseline characteristics
| Characteristic | Pexidartinib Part 1, Then Pexidartinib Part 2 | Total | Placebo Part 1, Then Pexidartinib Part 2 |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 4 Participants | 7 Participants | 3 Participants |
| Age, Categorical Between 18 and 65 years | 57 Participants | 113 Participants | 56 Participants |
| Age, Continuous | 44.6 years STANDARD_DEVIATION 13.2 | 44.5 years STANDARD_DEVIATION 13.4 | 44.3 years STANDARD_DEVIATION 13.6 |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 3 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants | 4 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 2 Participants | 4 Participants | 2 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 52 Participants | 106 Participants | 54 Participants |
| Region of Enrollment Australia | 5 participants | 12 participants | 7 participants |
| Region of Enrollment Canada | 2 participants | 5 participants | 3 participants |
| Region of Enrollment Denmark | 1 participants | 3 participants | 2 participants |
| Region of Enrollment France | 2 participants | 7 participants | 5 participants |
| Region of Enrollment Germany | 4 participants | 6 participants | 2 participants |
| Region of Enrollment Hungary | 2 participants | 3 participants | 1 participants |
| Region of Enrollment Italy | 8 participants | 17 participants | 9 participants |
| Region of Enrollment Netherlands | 7 participants | 11 participants | 4 participants |
| Region of Enrollment Poland | 2 participants | 2 participants | 0 participants |
| Region of Enrollment Spain | 5 participants | 8 participants | 3 participants |
| Region of Enrollment United Kingdom | 0 participants | 1 participants | 1 participants |
| Region of Enrollment United States | 23 participants | 45 participants | 22 participants |
| Sex: Female, Male Female | 35 Participants | 71 Participants | 36 Participants |
| Sex: Female, Male Male | 26 Participants | 49 Participants | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 61 | 0 / 59 | 0 / 61 | 1 / 30 | 1 / 91 |
| other Total, other adverse events | 60 / 61 | 55 / 59 | 61 / 61 | 30 / 30 | 91 / 91 |
| serious Total, serious adverse events | 8 / 61 | 1 / 59 | 12 / 61 | 9 / 30 | 21 / 91 |
Outcome results
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
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.
Time frame: Week 25
Population: Best overall response was assessed in the Intent-to-Treat (ITT) population.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | 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 | Complete Response (CR) | 14.8 Percentage of participants |
| Pexidartinib Part 1 | 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 | Partial Response (PR) | 24.6 Percentage of participants |
| Pexidartinib Part 1 | 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 | Response (CR or PR) | 39.3 Percentage of participants |
| Placebo Part 1 | 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 | Complete Response (CR) | 0 Percentage of participants |
| Placebo Part 1 | 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 | Partial Response (PR) | 0 Percentage of participants |
| Placebo Part 1 | 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 | Response (CR or PR) | 0 Percentage of participants |
Comparison: Treatment comparison between the pexidartinib and placebo groups at Week 25p-value: <0.0001Fisher's Exact Test
Secondary
Duration of Response (DOR) Based on RECIST 1.1
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.
Time frame: 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)
Population: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pexidartinib Part 1 | Duration of Response (DOR) Based on RECIST 1.1 | NA months |
| Placebo Part 1 | Duration of Response (DOR) Based on RECIST 1.1 | NA months |
Secondary
Duration of Response (DOR) Based on Tumor Volume Score (TVS)
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.
Time frame: 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)
Population: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pexidartinib Part 1 | Duration of Response (DOR) Based on Tumor Volume Score (TVS) | 52.70 months |
| Placebo Part 1 | Duration of Response (DOR) Based on Tumor Volume Score (TVS) | NA months |
Secondary
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to 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.
Time frame: Baseline, Week 13, and Week 25
Population: Range of motion was assessed in the ITT population in participants where data were available.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Week 13 | 13.0 degrees | Standard Error 2.3 |
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Week 25 | 15.1 degrees | Standard Error 2.1 |
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Baseline | 62.5 degrees | Standard Error 3.2 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Baseline | 62.9 degrees | Standard Error 2.9 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Week 13 | 4.8 degrees | Standard Error 2.6 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25 | Week 25 | 6.2 degrees | Standard Error 2.4 |
Comparison: Treatment comparison between the pexidartinib and placebo groups at Week 25p-value: 0.0043Fisher's Exact Test
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
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).
Time frame: Baseline, Week 9, Week 17, and Week 25
Population: Worst stiffness was assessed in the ITT population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | 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 | 5.6 units on a scale | Standard Error 0.2 |
| Pexidartinib Part 1 | 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 | Week 9 | -1.5 units on a scale | Standard Error 0.3 |
| Pexidartinib Part 1 | 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 | Week 17 | -2.4 units on a scale | Standard Error 0.3 |
| Pexidartinib Part 1 | 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 | Week 25 | -2.5 units on a scale | Standard Error 0.3 |
| Placebo Part 1 | 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 | Week 25 | -0.3 units on a scale | Standard Error 0.3 |
| Placebo Part 1 | 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 | 5.9 units on a scale | Standard Error 0.3 |
| Placebo Part 1 | 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 | Week 17 | -0.4 units on a scale | Standard Error 0.3 |
| Placebo Part 1 | 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 | Week 9 | -0.5 units on a scale | Standard Error 0.3 |
Comparison: Treatment comparison between the pexidartinib and placebo groups at Week 25p-value: <0.0001Mixed effects model for repeated measure
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
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.
Time frame: at Week 9 , Week 17, and Week 25
Population: Physical function was assessed in the ITT population in participants where data were available.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | 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 | Week 9 | 2.8 units on a scale | Standard Error 1 |
| Pexidartinib Part 1 | 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 | Week 17 | 3.2 units on a scale | Standard Error 1.1 |
| Pexidartinib Part 1 | 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 | Week 25 | 4.1 units on a scale | Standard Error 1.1 |
| Placebo Part 1 | 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 | Week 9 | -0.4 units on a scale | Standard Error 0.8 |
| Placebo Part 1 | 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 | Week 17 | 0.2 units on a scale | Standard Error 1 |
| Placebo Part 1 | 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 | Week 25 | -0.9 units on a scale | Standard Error 1 |
Comparison: Treatment comparison between pexidartinib and placebo groups at Week 25p-value: 0.0019Mixed effects model for repeated measure
Secondary
Number of Responders to Pexidartinib With and Without Disease Progression
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.
Time frame: By Week 96
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); Without disease progression | 9 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); With disease progression | 0 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); Without disease progression | 2 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); With disease progression | 1 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); Without disease progression | 15 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Number of responses | 23 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); With disease progression | 0 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); Without disease progression | 23 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); With disease progression | 1 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); Without disease progression | 23 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); With disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); Without disease progression | 9 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Number of responses | 12 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); Without disease progression | 12 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); Without disease progression | 12 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); Without disease progression | 3 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); Without disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); With disease progression | 0 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); Without disease progression | 3 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); Without disease progression | 12 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); With disease progression | 0 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Number of responses | 35 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 72 (Day 504); With disease progression | 1 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 12 (Day 84); Without disease progression | 35 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); Without disease progression | 24 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); With disease progression | 0 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 96 (Day 672); With disease progression | 1 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 48 (Day 336); With disease progression | 1 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression | Week 24 (Day 168); Without disease progression | 35 participants |
Secondary
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
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.
Time frame: By Week 120
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); Without disease progression | 1 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); With disease progression | 3 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); Without disease progression | 22 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); With disease progression | 0 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); Without disease progression | 13 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); With disease progression | 3 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Number of responders | 34 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); Without disease progression | 33 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); With disease progression | 3 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); Without disease progression | 32 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); With disease progression | 3 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); Without disease progression | 3 participants |
| Pexidartinib Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); With disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Number of responders | 18 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); Without disease progression | 18 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); Without disease progression | 18 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); With disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); Without disease progression | 13 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); Without disease progression | 3 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); With disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); Without disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); With disease progression | 1 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); Without disease progression | 0 participants |
| Placebo Part 1 | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); With disease progression | 1 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); With disease progression | 4 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); Without disease progression | 50 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); Without disease progression | 1 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); Without disease progression | 4 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); With disease progression | 0 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Number of responders | 52 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 96 (Day 672); With disease progression | 4 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); With disease progression | 4 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 48 (Day 336); Without disease progression | 35 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 12 (Day 84); Without disease progression | 51 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 72 (Day 504); Without disease progression | 16 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 24 (Day 168); With disease progression | 0 participants |
| All Pexidartinib Treated | Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score | Week 120 (Day 840); With disease progression | 4 participants |
Secondary
Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term
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.
Time frame: After the first dose of treatment up to 28 days after the last dose
Population: All safety events were assessed in the Safety Analysis Set.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Abdominal Pain | 16.4 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hair color changes | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash maculopapular | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash maculopapular | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis generalized | 8.2 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis generalized | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Erythema | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Erythema | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry skin | 3.3 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry skin | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Photosensitivity reaction | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Photosensitivity reaction | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Nausea | 37.7 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Nausea | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Diarrhea | 19.7 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Diarrhea | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Vomiting | 19.7 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Vomiting | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hair color changes | 67.2 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Abdominal Pain | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry mouth | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry mouth | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Constipation | 11.5 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Constipation | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Stomatitis | 6.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Stomatitis | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Fatigue | 54.1 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Fatigue | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Edema peripheral | 13.1 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Edema peripheral | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Face edema | 13.1 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Face edema | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Asthenia | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Asthenia | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pyrexia | 6.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥ Pyrexia | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any AST increased | 39.3 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 AST increased | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALT increased | 27.9 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALT increased | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALP increased | 14.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALP increased | 6.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any LDH increased | 11.5 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 LDH increased | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Weight increased | 3.3 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Weight increased | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dysgeusia | 24.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dysgeusia | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Headache | 19.7 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Headache | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dizziness | 9.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dizziness | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Paresthesia | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Paresthesia | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Memory impairment | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Memory impairment | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Arthralgia | 23.0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Arthralgia | 3.3 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pain in extremity | 6.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pain in extremity | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Periorbital edema | 18.0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Periorbital edema | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Eyelid edema | 3.3 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Eyelid edema | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Decreased appetite | 16.4 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Decreased appetite | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hypertension | 14.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hypertension | 4.9 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Upper respiratory tract infection | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Upper respiratory tract infection | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Cough | 4.9 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Cough | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dyspnea | 1.6 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dyspnea | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Insomnia | 4.9 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Insomnia | 0 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash | 14.8 Percentage of participants |
| Pexidartinib Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash | 1.6 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Decreased appetite | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Face edema | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis generalized | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Weight increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Cough | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 LDH increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Face edema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Abdominal Pain | 10.2 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any LDH increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALP increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALP increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Asthenia | 5.1 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Diarrhea | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALT increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Cough | 5.1 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash | 5.1 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Asthenia | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis generalized | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALT increased | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 AST increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Upper respiratory tract infection | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pyrexia | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Eyelid edema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any AST increased | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥ Pyrexia | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hypertension | 10.2 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Abdominal Pain | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Nausea | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash maculopapular | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash maculopapular | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Photosensitivity reaction | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry mouth | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Photosensitivity reaction | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hair color changes | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry mouth | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry skin | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Eyelid edema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hair color changes | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Periorbital edema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Constipation | 5.1 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Vomiting | 5.1 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Periorbital edema | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pain in extremity | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Insomnia | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Constipation | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Nausea | 40.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pain in extremity | 6.8 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hypertension | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Stomatitis | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Decreased appetite | 10.2 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Arthralgia | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Arthralgia | 25.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry skin | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Stomatitis | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Insomnia | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Memory impairment | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Memory impairment | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dyspnea | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Fatigue | 35.6 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Vomiting | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Paresthesia | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Paresthesia | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dizziness | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Fatigue | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Diarrhea | 25.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dizziness | 15.3 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Headache | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dyspnea | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Edema peripheral | 3.4 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Erythema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Headache | 18.6 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dysgeusia | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Upper respiratory tract infection | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Edema peripheral | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Erythema | 0 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dysgeusia | 1.7 Percentage of participants |
| Placebo Part 1 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Weight increased | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Upper respiratory tract infection | 11.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Diarrhea | 26.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Decreased appetite | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Diarrhea | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Vomiting | 23.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Vomiting | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Abdominal Pain | 21.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Abdominal Pain | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hypertension | 19.7 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry mouth | 13.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry mouth | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Constipation | 14.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Constipation | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Stomatitis | 8.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hypertension | 4.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Stomatitis | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Fatigue | 55.7 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Fatigue | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Edema peripheral | 16.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Edema peripheral | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Face edema | 14.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Face edema | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Asthenia | 11.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Asthenia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pyrexia | 8.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Upper respiratory tract infection | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥ Pyrexia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any AST increased | 44.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 AST increased | 9.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash | 27.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALT increased | 31.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALT increased | 9.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Cough | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALP increased | 14.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALP increased | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any LDH increased | 11.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 LDH increased | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Weight increased | 4.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Cough | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Weight increased | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dysgeusia | 27.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dysgeusia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Headache | 23.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Headache | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dyspnea | 4.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dizziness | 13.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dizziness | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Paresthesia | 8.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Paresthesia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Memory impairment | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dyspnea | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Memory impairment | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Arthralgia | 27.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Arthralgia | 3.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pain in extremity | 9.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pain in extremity | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Insomnia | 4.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Periorbital edema | 24.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Periorbital edema | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hair color changes | 73.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hair color changes | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Eyelid edema | 4.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis | 16.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash maculopapular | 14.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash maculopapular | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis generalized | 8.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Eyelid edema | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis generalized | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Erythema | 3.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Erythema | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry skin | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry skin | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Decreased appetite | 18.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Photosensitivity reaction | 1.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Photosensitivity reaction | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Nausea | 44.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Insomnia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Nausea | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dizziness | 13.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Memory impairment | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Headache | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hair color changes | 83.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dyspnea | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry mouth | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hypertension | 30.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Stomatitis | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hair color changes | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dysgeusia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Memory impairment | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Vomiting | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Nausea | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dyspnea | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Eyelid edema | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry mouth | 13.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Photosensitivity reaction | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dizziness | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Arthralgia | 30.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Eyelid edema | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash maculopapular | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Erythema | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Decreased appetite | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Stomatitis | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Abdominal Pain | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥ Pyrexia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry skin | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Upper respiratory tract infection | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pyrexia | 13.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Diarrhea | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any AST increased | 16.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Arthralgia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Headache | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Paresthesia | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash maculopapular | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 AST increased | 6.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Constipation | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dysgeusia | 23.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Asthenia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pain in extremity | 13.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALT increased | 23.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Edema peripheral | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Fatigue | 26.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Asthenia | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Abdominal Pain | 6.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALT increased | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Erythema | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Diarrhea | 30.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Vomiting | 6.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis generalized | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALP increased | 3.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pain in extremity | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Cough | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Paresthesia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Decreased appetite | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALP increased | 3.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Constipation | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Nausea | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Face edema | 3.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Photosensitivity reaction | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any LDH increased | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Periorbital edema | 13.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash | 23.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Upper respiratory tract infection | 3.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Insomnia | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 LDH increased | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Fatigue | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Insomnia | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Face edema | 20.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hypertension | 6.7 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Weight increased | 10.0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry skin | 3.3 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis generalized | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Periorbital edema | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Edema peripheral | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Weight increased | 0 Percentage of participants |
| Placebo Part 1, Pexidartinib Part 2 | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Cough | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Weight increased | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dysgeusia | 26.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Cough | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Edema peripheral | 17.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Vomiting | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dysgeusia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash | 26.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Decreased appetite | 15.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Headache | 22.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Fatigue | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Headache | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Erythema | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Eyelid edema | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dizziness | 13.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hypertension | 5.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dizziness | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dyspnea | 7.7 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Fatigue | 46.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Diarrhea | 27.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Paresthesia | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Paresthesia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Stomatitis | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Erythema | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Memory impairment | 4.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Decreased appetite | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Vomiting | 17.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Memory impairment | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Stomatitis | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Nausea | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Arthralgia | 28.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dyspnea | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Arthralgia | 2.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Constipation | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry skin | 7.7 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pain in extremity | 11.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Insomnia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Constipation | 13.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pain in extremity | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Periorbital edema | 20.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hypertension | 13.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Periorbital edema | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Insomnia | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry mouth | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Dry skin | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Hair color changes | 76.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Nausea | 36.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Hair color changes | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Diarrhea | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Dry mouth | 13.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis | 17.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Eyelid edema | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Abdominal Pain | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Photosensitivity reaction | 4.4 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Rash maculopapular | 13.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pyrexia | 9.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥ Pyrexia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any AST increased | 35.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Upper respiratory tract infection | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Asthenia | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 AST increased | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Rash maculopapular | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Abdominal Pain | 16.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALT increased | 28.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Asthenia | 14.3 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALT increased | 9.9 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Upper respiratory tract infection | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any ALP increased | 11.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Face edema | 2.2 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Pruritis generalized | 8.8 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 ALP increased | 5.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Cough | 7.7 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any LDH increased | 11.0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 LDH increased | 1.1 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Face edema | 16.5 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Photosensitivity reaction | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Any Weight increased | 6.6 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Edema peripheral | 0 Percentage of participants |
| All Pexidartinib Treated | Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term | Grade ≥3 Pruritis generalized | 0 Percentage of participants |
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
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).
Time frame: Week 25
Population: Worst pain was assessed in the ITT population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pexidartinib Part 1 | 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 | 31.1 percentage of participants |
| Placebo Part 1 | 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 | 15.3 percentage of participants |
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
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.
Time frame: Week 25
Population: Best overall response was assessed in the ITT population.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | 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 | Complete Response (CR) | 4.9 Percentage of participants |
| Pexidartinib Part 1 | 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 | Partial Response (PR) | 50.8 Percentage of participants |
| Pexidartinib Part 1 | 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 | Response (CR or PR) | 55.7 Percentage of participants |
| Placebo Part 1 | 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 | Complete Response (CR) | 0 Percentage of participants |
| Placebo Part 1 | 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 | Partial Response (PR) | 0 Percentage of participants |
| Placebo Part 1 | 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 | Response (CR or PR) | 0 Percentage of participants |
Comparison: Treatment comparison between the pexidartinib and placebo groups at Week 25p-value: <0.0001Fisher's Exact Test
Other Pre-specified
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
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.
Time frame: By Week 49
Population: Range of Motion (ROM) was assessed in the ITT population.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | 15.6 degrees | Standard Deviation 14.9 |
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 62.5 degrees | Standard Deviation 24.8 |
| Pexidartinib Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | 14.4 degrees | Standard Deviation 19.5 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | 13.1 degrees | Standard Deviation 12.9 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 66.5 degrees | Standard Deviation 22.9 |
| Placebo Part 1 | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | 12.0 degrees | Standard Deviation 13.4 |
| All Pexidartinib Treated | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 63.8 degrees | Standard Deviation 24.2 |
| All Pexidartinib Treated | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | 13.4 degrees | Standard Deviation 17.3 |
| All Pexidartinib Treated | Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | 14.8 degrees | Standard Deviation 14.2 |
Other Pre-specified
Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Brief Pain Inventory (BPI) Worst Pain 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).
Time frame: By Week 49
Population: Worst pain was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -2.7 units on a scale | Standard Deviation 2.2 |
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.6 units on a scale | Standard Deviation 1.6 |
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -3.3 units on a scale | Standard Deviation 1.7 |
| Placebo Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -2.6 units on a scale | Standard Deviation 3.1 |
| Placebo Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.2 units on a scale | Standard Deviation 2.5 |
| Placebo Part 1 | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -2.8 units on a scale | Standard Deviation 3.4 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.5 units on a scale | Standard Deviation 1.9 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -3.2 units on a scale | Standard Deviation 2.3 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -2.7 units on a scale | Standard Deviation 2.5 |
Other Pre-specified
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
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).
Time frame: Baseline, Week 25, and Week 49
Population: Worst stiffness was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -2.7 units on a scale | Standard Deviation 2.2 |
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.6 units on a scale | Standard Deviation 1.7 |
| Pexidartinib Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -3.5 units on a scale | Standard Deviation 1.9 |
| Placebo Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -3.0 units on a scale | Standard Deviation 3.1 |
| Placebo Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.7 units on a scale | Standard Deviation 2.3 |
| Placebo Part 1 | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -2.2 units on a scale | Standard Deviation 2.8 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Baseline | 5.6 units on a scale | Standard Deviation 1.9 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 49 | -3.1 units on a scale | Standard Deviation 2.3 |
| All Pexidartinib Treated | Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49 | Week 25 | -2.8 units on a scale | Standard Deviation 2.5 |
Other Pre-specified
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 by Week 49
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.
Time frame: By Week 49
Population: Physical function was assessed in the ITT population.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pexidartinib Part 1 | 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 by Week 49 | Week 25 | 3.6 units on a scale | Standard Deviation 4.9 |
| Pexidartinib Part 1 | 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 by Week 49 | Week 49 | 4.7 units on a scale | Standard Deviation 4.4 |
| Placebo Part 1 | 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 by Week 49 | Week 25 | 4.9 units on a scale | Standard Deviation 6.3 |
| Placebo Part 1 | 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 by Week 49 | Week 49 | 7.6 units on a scale | Standard Deviation 6.3 |
| All Pexidartinib Treated | 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 by Week 49 | Week 25 | 4.0 units on a scale | Standard Deviation 5.4 |
| All Pexidartinib Treated | 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 by Week 49 | Week 49 | 5.8 units on a scale | Standard Deviation 5.2 |
Other Pre-specified
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 by Week 49
Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score 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.
Time frame: By Week 49
Population: Best overall response on Tumor Volume Score was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed..
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pexidartinib Part 1 | 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 by Week 49 | 63.9 Percentage of participants |
| Placebo Part 1 | 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 by Week 49 | 66.7 Percentage of participants |
| All Pexidartinib Treated | 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 by Week 49 | 64.8 Percentage of participants |
Other Pre-specified
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 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.
Time frame: By Week 49
Population: Best overall response was assessed in the ITT population.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pexidartinib Part 1 | 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 49 | Partial Response (PR) | 29.5 Percentage of participants |
| Pexidartinib Part 1 | 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 49 | Complete Response (CR) | 24.6 Percentage of participants |
| Pexidartinib Part 1 | 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 49 | Response (CR or PR) | 54.1 Percentage of participants |
| Placebo Part 1 | 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 49 | Partial Response (PR) | 30.0 Percentage of participants |
| Placebo Part 1 | 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 49 | Complete Response (CR) | 23.3 Percentage of participants |
| Placebo Part 1 | 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 49 | Response (CR or PR) | 53.3 Percentage of participants |
| All Pexidartinib Treated | 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 49 | Complete Response (CR) | 24.2 Percentage of participants |
| All Pexidartinib Treated | 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 49 | Response (CR or PR) | 53.8 Percentage of participants |
| All Pexidartinib Treated | 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 49 | Partial Response (PR) | 29.7 Percentage of participants |