A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT

Inclusion Criteria

• •Age ≥ 18 years (Age ≥ 20 years in Taiwan).
• •A diagnosis of TGCT (i) that has been histologically confirmed by a pathologist and (ii) associated with severe morbidity or functional limitations and not amenable to improvement with surgery determined consensually by qualified personnel (eg, 2 surgeons or a multi-disciplinary tumor board).
• •Measurable disease as defined by RECIST 1.1 (except that a minimal size of 2 cm is required), assessed from MRI scan by a central radiologist.
• •Stable prescription of analgesic regimen during the 2 weeks prior to enrollment.
• •Women of childbearing potential must have a negative serum pregnancy test within the 14-day period prior to enrollment (Where demanded by local regulations, this test may be required within 72 hours of enrollment).
• •Females of reproductive potential should be advised to use an effective, non-hormonal method of contraception during treatment with pexidartinib and for 1 month after the last dose. Males with female partners of reproductive potential should be advised to use an effective method of contraception during treatment with pexidartinib and for 1 month after the last dose. Female partners of male participants should concurrently use effective contraceptive methods (hormonal or non-hormonal). Women of nonchildbearing 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 level \> 40 mIU/mL will be considered postmenopausal.
• •Adequate hematologic, hepatic, and renal function, defined by:
• •* Absolute neutrophil count ≥ 1.5 × 10\^9/L
• •* Hemoglobin \> 10 g/dL
• •* Platelet count ≥ 100 × 10\^9/L
• •* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 1.0 × upper limit of normal (ULN)
• •* Total bilirubin and direct bilirubin ≤ 1.0 × ULN
• •* Alkaline phosphatase ≤ 1.0 × ULN
• •* Creatinine clearance (CLcr) \> 15 mL/min
• •Willingness and ability to complete the PROMIS Physical Function Scale.
• •Willingness and ability to use a diary.
• •Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.