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

NCT04488822 | Active Not Recruiting Phase 3 Results FDA Drug

• 1 other identifier: PL3397-A-A303
• Study Type: interventional
• Target: 40
• Locations: 2 countries
• Sites: 9

Brief Summary

This study will assess pexidartinib in adult participants with symptomatic TGCT that is associated with severe morbidity or functional limitations and not amendable to improvement with surgery.

Conditions

Tenosynovial Giant Cell Tumor

Keywords

Tenosynovial Giant Cell Tumor; Pexidartinib; TURALIO™; PLX3397

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25

  For the assessment of tumor response, participants were classified into the best of the following tumor response categories based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) by centrally reviewed MRI scan. Complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; and overall response rate (ORR), defined as CR+PR, are presented.

  At Week 25 postdose

Secondary Outcomes (9)

• Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25

  At Week 25 postdose

• Mean Percent Change From Baseline for Range of Motion (ROM) Score in Participants Receiving Pexidartinib

  At Week 25 postdose

• Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib

  At Week 25 postdose

• Best Overall Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan

  From baseline until disease progression or death (whichever occurs first), up to approximately 29 months

• Duration of Response of Pexidartinib Based on RECIST 1.1 and TVS by Centrally Reviewed MRI Scan

  Time from the date of first documented response until documented disease progression or death from any cause (whichever occurs first), up to approximately 29 months

Study Arms (1)

Pexidartinib

EXPERIMENTAL

Drug: Pexidartinib

Interventions

Pexidartinib DRUG

400 mg twice daily for a total daily dose of 800 mg (each capsule contains 200 mg of pexidartinib for oral administration)

Also known as: TURALIO™, PLX3397

Pexidartinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Investigational drug/device use within 28 days of enrolment.
• Previous use of pexidartinib or any biologic treatment targeting colony stimulating factor 1 (CSF-1) or the CSF-1 receptor; previous use of oral tyrosine kinase inhibitors are allowed (eg, imatinib or nilotinib).
• Active cancer except for tumor for which a participant is enrolled in the study, (either concurrent or within the last year of starting study drug) 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.
• Known metastatic TGCT.
• Active or chronic infection with hepatitis C or known positive hepatitis B surface antigen, or known active or chronic infection with human immunodeficiency virus.
• Active liver or biliary tract disease
• Known active tuberculosis.
• 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 a participant's study participation or the interpretation of his or her results.
• Use of strong Cytochrome P450 (CYP) 3A inducers, including St John's wort, proton pump inhibitors (PPIs), and other products known to cause hepatotoxicity.
• Women who are breastfeeding.
• A screening Fridericia corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women).
• MRI contraindications.
• History of hypersensitivity to any excipients in the investigational product.
• Inability to swallow capsules.

Sponsors & Collaborators

• Daiichi Sankyo Co., Ltd.: lead

Study Sites (9)

Beijing Ji Shui Tan Hospital

Beijing, China

Location

Peking University Cancer Hospital

Beijing, China

Location

The First Affiliated Hospital, Sun Yat-sen University

Guangzhou, China

Location

The Second Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, China

Location

Zhejiang Cancer Hospital

Hangzhou, China

Location

Fudan University Shanghai Cancer Center

Shanghai, China

Location

Shanghai General Hospital

Shanghai, China

Location

National Taiwan University Hospital

Taipei, Taiwan

Location

Taipei Veterans General Hospital

Taipei, Taiwan

Location

Related Publications (1)

• Xu H, Wu PK, Ye Z, Fan Z, Li T, Chen TW, Shen J, Yan W, Achiwa I, Yoh K, Kikumori K, Hiruma Y, Greenberg J, Niu X. A Phase 3 Study of the Efficacy and Safety of Pexidartinib in East Asian Patients with Tenosynovial Giant Cell Tumor. Oncol Ther. 2025 Dec;13(4):1025-1039. doi: 10.1007/s40487-025-00365-z. Epub 2025 Aug 21.

  PMID: 40841499 DERIVED

MeSH Terms

Conditions

Giant Cell Tumor of Tendon Sheath

Interventions

pexidartinib

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Results Point of Contact

• Title: Clinical Director
• Organization: Daiichi Sankyo, Inc.

CTRinfo@dsi.com

908-992-6400

Study Officials

• Global Clinical Leader

  Daiichi Sankyo

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2020
• First Posted: July 28, 2020
• Study Start: September 25, 2020
• Primary Completion: October 27, 2021
• Study Completion: February 28, 2026
• Last Updated: September 9, 2025
• Results First Posted: April 27, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Completed studies that has reached a global end or completion with all data set collected and analyzed, and for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
• Access Criteria: Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.

Locations

TW (2); CN (7)