18 and older, any sex, with Solid Tumor. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Summary of Derived Best Tumor Response Per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 - Dose Escalation (Efficacy Evaluable Population)Primary· Every 2 months beginning Cycle 3, Day 1 until disease progression (up to approximately 30 months postdose)
Best overall tumor response (complete response \[CR\], partial response \[PR\], stable disease \[SD\], progressive disease \[PD\]) is reported, including participants who were not evaluable (NE).
RECIST v1.1 for target lesions are assessed by magnetic resonance imaging, computed tomography, or positron emission tomography-computed tomography and are summarized as: CR, Disappearance of all target lesions. Any pathological lymph nodes must have reduction in short axis to \<10 mm; PR, At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameter
Summary of Derived Best Tumor Response Per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 - Dose Extension (Efficacy Evaluable Population)Primary· Every 2 months beginning Cycle 3, Day 1 until disease progression (up to approximately 30 months postdose)
Best overall tumor response (complete response \[CR\], partial response \[PR\], stable disease \[SD\], progressive disease \[PD\]) is reported, including participants who were not evaluable (NE).
RECIST v1.1 for target lesions are assessed by magnetic resonance imaging, computed tomography, or positron emission tomography-computed tomography and are summarized as: CR, Disappearance of all target lesions. Any pathological lymph nodes must have reduction in short axis to \<10 mm; PR, At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameter
CR
Group
Value
95% CI
Dose Extension: MEC Cohort
0
Dose Extension: PVNS Cohort
2
Dose Extension: GIST Cohort
0
Dose Extension: ATC Cohort
0
Dose Extension: Malignant Effusion Cohort
0
Dose Extension: Other Solid Tumor Type Cohort
0
PR
Group
Value
95% CI
Dose Extension: MEC Cohort
0
Dose Extension: PVNS Cohort
22
Dose Extension: GIST Cohort
0
Dose Extension: ATC Cohort
0
Dose Extension: Malignant Effusion Cohort
0
Dose Extension: Other Solid Tumor Type Cohort
1
SD
Group
Value
95% CI
Dose Extension: MEC Cohort
1
Dose Extension: PVNS Cohort
8
Dose Extension: GIST Cohort
4
Dose Extension: ATC Cohort
0
Dose Extension: Malignant Effusion Cohort
3
Dose Extension: Other Solid Tumor Type Cohort
7
PD
Group
Value
95% CI
Dose Extension: MEC Cohort
1
Dose Extension: PVNS Cohort
1
Dose Extension: GIST Cohort
5
Dose Extension: ATC Cohort
6
Dose Extension: Malignant Effusion Cohort
2
Dose Extension: Other Solid Tumor Type Cohort
6
NE
Group
Value
95% CI
Dose Extension: MEC Cohort
0
Dose Extension: PVNS Cohort
4
Dose Extension: GIST Cohort
0
Dose Extension: ATC Cohort
0
Dose Extension: Malignant Effusion Cohort
1
Dose Extension: Other Solid Tumor Type Cohort
0
Overall response rate (CR or PR)
Group
Value
95% CI
Dose Extension: MEC Cohort
0
Dose Extension: PVNS Cohort
24
Dose Extension: GIST Cohort
0
Dose Extension: ATC Cohort
0
Dose Extension: Malignant Effusion Cohort
0
Dose Extension: Other Solid Tumor Type Cohort
1
Duration of Response (Efficacy Evaluable Population) - Dose ExtensionPrimary· From initial response until disease progression or death, up to approximately 30 months postdose
Duration of Response (DOR) is defined as the number of days from the date of initial response (CR or PR confirmed at least 28 days later) to the date of first documented disease progression/relapse or death, whichever occurs first. If no disease progression or death is documented prior to study termination, analysis cutoff, or the start of confounding anticancer therapy, DOR is censored as of the date of their last imaging exam of target or non-target lesions prior to post-surgery and/or off-treatment scans.
Group
Value
95% CI
Dose Extension: PVNS Cohort
NA
943 – NA
Dose Extension: Other Solid Tumor Type Cohort
115
NA – NA
Progression-free Survival (Efficacy Evaluable Population) - Dose ExtensionPrimary· From Cycle 1 Day 1 to disease progression or death
Progression-Free Survival (PFS) is defined as the number of days from the first day of treatment to the first documented disease progression or date of death, whichever occurs first. If no disease progression or death is documented prior to study termination, analysis cutoff, or the start of confounding anticancer therapy, PFS is censored at the date of last evaluable tumor assessment.
Group
Value
95% CI
Dose Extension: MEC Cohort
NA
43 – NA
Dose Extension: PVNS Cohort
NA
667 – NA
Dose Extension: GIST Cohort
54
40 – 164
Dose Extension: ATC Cohort
50
34 – 56
Dose Extension: Malignant Effusion Cohort
78
51 – NA
Dose Extension: Other Solid Tumor Type Cohort
161
54 – 504
Best Overall Tumor Response (PVNS Cohort) Per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 - Dose ExtensionPrimary· Every 2 months beginning Cycle 3, Day 1 until disease progression
Best overall tumor response (complete response \[CR\], partial response \[PR\], stable disease \[SD\], progressive disease \[PD\]) is reported, including participants who were not evaluable (NE).
RECIST v1.1 for target lesions are assessed by magnetic resonance imaging, computed tomography, or positron emission tomography-computed tomography and are summarized as: CR, Disappearance of all target lesions. Any pathological lymph nodes must have reduction in short axis to \<10 mm; PR, At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameter
CR
Group
Value
95% CI
PVNS Cohort
2
PR
Group
Value
95% CI
PVNS Cohort
22
SD
Group
Value
95% CI
PVNS Cohort
8
PD
Group
Value
95% CI
PVNS Cohort
1
NE
Group
Value
95% CI
PVNS Cohort
4
Overall response rate (CR or PR)
Group
Value
95% CI
PVNS Cohort
24
Summary Statistics for Selected PLX3397 Pharmacokinetics Parameter Cmax, Study Day Cycle 1 Day 15, Stratified by Cohort - Dose EscalationPrimary· Cycle 1, Day 15
Cycle 1 Day 15 pharmacokinetic (PK) timepoints taken are: For once daily (QD) dosing, obtained predose (morning) and 0.5, 1, 2, 4, and 8 hours postdose. For twice a day (BID) dosing predose (morning) and 1, 2, 4, and 7 hours postdose. The second dose will then be administered, and PK obtained 1 hour post the second dose (8 hours post the first dose). For BID dosing, no run-in is planned.
Summary Statistics for Selected PLX3397 Pharmacokinetics Parameter Tmax, Study Day Cycle 1 Day 15, Stratified by Cohort - Dose EscalationPrimary· Cycle 1, Day 15
Cycle 1 Day 15 PK timepoints taken are: For QD dosing, obtained predose (morning) and 0.5, 1, 2, 4, and 8 hours postdose. For BID dosing predose (morning) and 1, 2, 4, and 7 hours postdose. The second dose will then be administered, and PK obtained 1 hour post the second dose (8 hours post the first dose). For BID dosing, no run-in is planned.
Summary Statistics for Selected PLX3397 Pharmacokinetics Parameter Area Under the Curve (AUC0-24), Study Day Cycle 1 Day 15, Stratified by Cohort - Dose EscalationPrimary· Cycle 1, Day 15 (QD dosing: predose [morning] and 0.5, 1, 2, 4, and 8 hours postdose; BID dosing: predose [morning] and 1, 2, 4, and 7 hours postdose)
Cycle 1 Day 15 PK timepoints taken are: For QD dosing, obtained predose (morning) and 0.5, 1, 2, 4, and 8 hours postdose. For BID dosing, predose (morning) and 1, 2, 4, and 7 hours postdose. The second dose will then be administered, and PK obtained 1 hour post the second dose (8 hours post the first dose). For BID dosing, no run-in is planned.
Numeric Rating Scale (NRS) for PVNS Symptoms Sum of Scores Through Cycle 2 (Efficacy Evaluable Population) - Dose ExtensionPrimary· Baseline, Cycle 1 Day 15, Cycle 2, Cycle 3, Cycle 12, Cycle 24, Cycle 36, and Cycle 46 (each cycle was 28 days)
The NRS for PVNS Symptoms instrument is a 5-item self-administered questionnaire to assess the "worst" of each of the symptoms pain, swelling, stiffness, instability and limited motion in the last 24 hours. A 0 to 10 NRS is provided for each symptom. For pain, 0 indicates "no pain" and 10 indicates "pain as bad as you can imagine". For the other 4 symptoms, 0 indicates "no (symptom)" and 10 indicates "(symptom) worst imaginable", e.g., "swelling - worst imaginable." Higher scores indicated worse outcome.
Pain; Baseline
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
5.2
± 2.1
Pain; Cycle 1 Day 15
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
3.5
± 2.9
Pain; Cycle 2
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
3.6
± 2.6
Pain; Cycle 3
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
2.3
± 1.9
Pain; Cycle 12
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
1.3
± 1.8
Pain; Cycle 24
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
1.5
± 1.4
Pain; Cycle 36
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
2.0
± 2.8
Pain; Cycle 46
Group
Value
95% CI
NRS for PVNS Symptoms Evaluable Population
1.8
± 2.0
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events (and serious adverse events) were recorded from the time the participant received the first dose of study drug up to 30 days after the last dose, up to approximately 30 months postdose..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
PLX3397 is a selective inhibitor of Fms, Kit, and oncogenic Flt3 activity. The primary objective of this study is to evaluate the safety and pharmacokinetics of orally administered PLX3397 in patients with advanced, incurable, solid tumors in which these target kinases are linked to disease pathophysiology. The secondary objective is to measure the pharmacodynamic activity of PLX3397 via blood, plasma and urine biomarkers of Fms activity.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT02975700 — A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma
· NA
· completed
NCT02452424 — A Combination Clinical Study of PLX3397 and Pembrolizumab To Treat Advanced Melanoma and Other Solid Tumors
· Phase 1, PHASE2
· terminated
NCT01826448 — A Phase 1b Open Label, Dose Escalation Study of PLX3397 in Combination With Vemurafenib in V600-mutated BRAF Melanoma
· Phase 1
· terminated
NCT01790503 — A Phase 1b/2 Study of PLX3397 + Radiation Therapy + Temozolomide in Patients With Newly Diagnosed Glioblastoma
· Phase 1, PHASE2
· completed
NCT01596751 — Phase Ib/II Study of PLX 3397 and Eribulin in Patients With Metastatic Breast Cancer
· Phase 1, PHASE2
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Daiichi Sankyo
Last refreshed: 4 January 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01004861.