Clinical Trials

Numerous clinical trials evaluate the therapeutic efficacy, safety, and pharmacokinetic properties of pexidartinib across indications such as tenosynovial giant cell tumor, gastrointestinal stromal tumors, colorectal cancer, pancreatic cancer, and hepatic impairment. Spanning Early Phase 1 through Phase 3, these studies are funded by industry sponsors and academic institutions, including Daiichi Sankyo and the National Cancer Institute. Protocol recruitment statuses range from active and recruiting to completed, terminated, and withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT01042379 RECRUITING
Breast Neoplasms; Breast Cancer; Breast Tumors; Angiosarcoma; TNBC - Triple-Negative Breast Cancer; HER2-positive Breast Cancer; HER2-negative Breast Cancer; Hormone Receptor Positive Tumor; Hormone Receptor Negative Tumor; Early-stage Breast Cancer; Locally Advanced Breast Cancer
QuantumLeap Healthcare Collaborative
 2010-03-01 PHASE2
NCT02390752 RECRUITING
Neurofibroma, Plexiform; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Promyelocytic, Acute; Sarcoma
National Cancer Institute (NCI)
 2015-04-29 PHASE1
NCT04526704 COMPLETED
Tenosynovial Giant Cell Tumor
Daiichi Sankyo
 2020-10-20 PHASE4
NCT02584647 TERMINATED
Sarcoma; Malignant Peripheral Nerve Sheath Tumors
Gulam Manji
 2015-11-04 PHASE1; PHASE2
NCT04703322 COMPLETED
Tenosynovial Giant Cell Tumor
Daiichi Sankyo Co., Ltd.
 2021-03-15 PHASE2
NCT04488822 ACTIVE_NOT_RECRUITING
Tenosynovial Giant Cell Tumor
Daiichi Sankyo Co., Ltd.
 2020-09-25 PHASE3
NCT03158103 COMPLETED
Gastrointestinal Stromal Tumor (GIST)
Memorial Sloan Kettering Cancer Center
 2017-04-15 PHASE1
NCT02071940 COMPLETED
Malignant Melanoma
The Christie NHS Foundation Trust
 2015-10 PHASE2
NCT04703322 Recruiting
Tenosynovial Giant Cell Tumor
Daiichi Sankyo Co. Ltd.|Daiichi Sankyo
 2021-03-15 Phase 2
NCT04635111 Recruiting
Hepatotoxicity|Tenosynovial Giant Cell Tumor
Daiichi Sankyo
 2021-01-07 --
NCT04223635 COMPLETED
Moderate Hepatic Impairment
Daiichi Sankyo
 2020-01-07 EARLY_PHASE1
NCT04488822 Active not recruiting
Tenosynovial Giant Cell Tumor
Daiichi Sankyo Co. Ltd.|Daiichi Sankyo
 2020-09-25 Phase 3
NCT02401815 COMPLETED
Gastrointestinal Stromal Tumors
Cogent Biosciences, Inc.
 2015-03-06 PHASE1; PHASE2
NCT04223635 Completed
Moderate Hepatic Impairment
Daiichi Sankyo
 2020-01-07 Early Phase 1
NCT02777710 COMPLETED
Colorectal Cancer; Pancreatic Cancer; Metastatic Cancer; Advanced Cancer
Centre Leon Berard
 2016-06 PHASE1
NCT02472275 COMPLETED
Stage I Prostate Adenocarcinoma; Stage II Prostate Adenocarcinoma; Stage III Prostate Adenocarcinoma
Barbara Ann Karmanos Cancer Institute
 2015-06 PHASE1
NCT03901313 COMPLETED
Healthy Volunteers
Daiichi Sankyo
 2019-04-01 PHASE1
NCT03291288 COMPLETED
Drug Interaction Potential
Daiichi Sankyo
 2018-02-26 PHASE1
NCT02975700 COMPLETED
Melanoma
Daiichi Sankyo Co., Ltd.
 2017-01-31
NCT02452424 TERMINATED
Melanoma; Non-small Cell Lung Cancer; Squamous Cell Carcinoma of the Head and Neck; Gastrointestinal Stromal Tumor (GIST); Ovarian Cancer
Daiichi Sankyo
 2015-07-02 PHASE1; PHASE2
NCT03291288 Completed
Drug Interaction Potential
Daiichi Sankyo
 2018-02-26 Phase 1
NCT01004861 COMPLETED
Solid Tumor
Daiichi Sankyo
 2009-10-01 PHASE1
NCT01525602 COMPLETED
Solid Tumors
Daiichi Sankyo
 2012-05 PHASE1
NCT01790503 COMPLETED
Patients With Newly Diagnosed Glioblastoma
Daiichi Sankyo
 2013-07-18 PHASE1; PHASE2
NCT01596751 COMPLETED
Metastatic Breast Cancer
Hope Rugo, MD
 2012-07-12 PHASE1; PHASE2
NCT02734433 COMPLETED
Advanced Solid Tumors
Daiichi Sankyo Co., Ltd.
 2016-06 PHASE1
NCT03138759 COMPLETED
Pharmacokinetics in Healthy Volunteers
Daiichi Sankyo
 2017-02-27 PHASE1
NCT02371369 COMPLETED
Pigmented Villonodular Synovitis; Giant Cell Tumors of the Tendon Sheath; Tenosynovial Giant Cell Tumor
Daiichi Sankyo
 2015-05-11 PHASE3
NCT01349049 COMPLETED
Acute Myeloid Leukemia
Daiichi Sankyo
 2011-11-21 PHASE1; PHASE2
NCT01826448 TERMINATED
V600-mutated BRAF Unresectable Melanoma; V600-mutated BRAF Metastatic Melanoma; Stage III or Stage IV Metastatic Melanoma That Has Not Been Previously Treated With a Selective BRAF Inhibitor
Daiichi Sankyo
 2013-11-05 PHASE1
NCT01349036 TERMINATED
Recurrent Glioblastoma
Daiichi Sankyo
 2011-12-03 PHASE2
NCT01499043 TERMINATED
Prostate Cancer
Daiichi Sankyo
 2012-05-25 PHASE2
NCT01217229 COMPLETED
Hodgkin Lymphoma
Daiichi Sankyo
 2011-03-03 PHASE2
NCT01090570 WITHDRAWN
Rheumatoid Arthritis
Plexxikon
 2010-05 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-05-06)

Check the Pexidartinib (PLX3397) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pexidartinib functions as an oral multi-targeted receptor tyrosine kinase inhibitor that selectively binds CSF-1R, Kit (c-Kit), and FLT3 with IC50 values of 20 nM, 10 nM, and 160 nM, respectively. By blocking downstream receptor phosphorylation and signaling pathways, it triggers apoptosis and cell necrosis to inhibit tumor proliferation in clinical conditions such as tenosynovial giant cell tumor and gastrointestinal stromal tumors.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.