This trial is active, not recruiting.

Condition at this time, all cohorts are closed to enrollment with the exception of the pigmented villo-nodular synovitis (pvns) cohort.
Treatment plx3397
Phase phase 1
Sponsor Plexxikon
Start date September 2009
End date June 2017
Trial size 150 participants
Trial identifier NCT01004861, PLX108-01


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
Capsules administered once or twice daily, continuous dosing

Primary Outcomes

Safety: subject incidence of adverse events, first-cycle DLTs and clinically significant changes in vital signs, ECGs and clinical laboratory tests
time frame: till end of study

Secondary Outcomes

PK profile: PLX3397 PK parameters including, but not limited to, maximum observed concentration (Cmax), area under the plasma concentration-time curve and half-life
time frame: till end of study

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Age 18 and older - Solid tumors refractory to standard therapy - For the Extension cohorts, patients must have measurable disease by RECIST criteria and meet the following disease-specific criteria: - For advanced or recurrent mucoepidermal carcinoma (MEC) of the salivary gland, patients must not be candidates for curative surgery or radiotherapy. - For pigmented villo-nodular synovitis (PVNS), patients must have a histologically confirmed diagnosis of inoperable progressive or relapsing PVNS, or resectable tumor requesting mutilating surgery, as well as demonstrated progressive disease in the last 12 months. - For gastrointestinal stromal tumors (GIST), patients must have failed previous therapy with imatinib and sunitinib. Patients with known PDGFR mutations are excluded, but mutation testing is not required for study entry. - For anaplastic thyroid cancer (ATC), patients must have histologically or cytologically diagnosed advanced ATC. - For metastatic solid tumors with documented malignant pleural and/or peritoneal effusions, patients must not be receiving specific therapy for the effusion or have an indwelling drain. - ECOG performance status 0 or 1 - Life expectancy >= 3 months - Adequate hepatic, renal, and bone marrow function Exclusion Criteria: - Specific anti-cancer therapy within 3 weeks of study start - Uncontrolled intercurrent illness - Refractory nausea or vomiting, or malabsorption - Mean QTc >= 450 msec (for males) or QTc >= 470 msec (for females)

Additional Information

Official title A Phase 1 Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of PLX3397 in Patients With Advanced, Incurable, Solid Tumors in Which the Target Kinases Are Linked to Disease Pathophysiology
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Plexxikon.
Location data was received from the National Cancer Institute and was last updated in August 2016.