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Clinical Trial VICCPHI0958


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

Principal Investigator(s)

Vicki Keedy


  • Protocol No. VICCPHI0958
  • Open Date: 02/05/2010
  • Staging: Phase I
  • Age Group: Adults
  • Scope: National
  • Objective: The primary objective for the dose escalation phase of this study is to evaluate the safety and PK of PLX3397 in patients with solid tumors. The primary objective for the Extension cohorts (to be enrolled under a subsequent protocol amendment) is to evaluate the objective tumor response (including overall response rate, duration of response, and progression free survival), safety, and PK of PLX3397 in patients with selected solid or hematologic tumors, depending on the safety, pharmacodynamic activity, and response data observed in the dose escalation phase.
  • Disease Sites: Phase I; GIST
  • Therapies: Molecular Targeted Agents / Immunotherapy / Biologics
  • Drugs: PLX3397 (oral)
  • Participating Institutions: Vanderbilt University
  • National Clinical Trial ID: NCT01004861
  • Secondary Protocol No: PLX108-01


Currently, new patients are being asked to participate in additional parts of this study (i.e. Extension Cohorts) because they have a condition known as pigmented villonodular synovitis (PVNS). Previously, the main purpose of this research study was to help determine the highest dose of the PLX3397 study drug that safely can be given to patients with cancer. Prior patients participated in earlier parts of this study (called the dose escalation phase). We will look at how your body absorbs and breaks down PLX3397 and how PLX3397 affects the body. This is an investigational drug, which means the drug is not approved by the Food and Drug Administration (FDA). Patients will be treated with the study drug, PLX3397, given orally (by mouth) at the highest safest dose, which was determined by how well prior participants tolerated their doses.


Ages Eligible for Study:18 Years and older
Genders Eligible for Study:Both
Accepts Healthy Volunteers:No


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)