A Phase 2 Study of PLX3397 in Patients With Recurrent Glioblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01349036
Recruitment Status : Completed
First Posted : May 6, 2011
Last Update Posted : April 27, 2015
Information provided by (Responsible Party):

May 4, 2011
May 6, 2011
April 27, 2015
August 2011
February 2014   (Final data collection date for primary outcome measure)
  • Safety-Subject incidence of adverse events [ Time Frame: 1 year ]
    Subjects will take oral doses of PLX3397 twice a day. Physical examinations, vital signs, 12-lead electrocardiograms (ECG), adverse events, hematology and serum chemistry will be used to assess safety throughout the study. Adverse events will be monitored and reviewed for safety issues/abnormal changes in the above mentioned tests.
  • Efficacy-6 month progression free survival rate (PFS6), median duration of response, overall survival (OS) [ Time Frame: 2 years ]
    After discontinuation of PLX3397 for reasons other than progression of disease, patients will be followed every 3 months, or as clinically indicated, until progression of disease or death is documented.
  • Efficacy-Overall response rate (ORR) [ Time Frame: 1 year ]
    Subjects will have an MRI brain scan within 28 days of starting dosing with PLX3397 and then every 8 weeks thereafter. Response to treatment will be evaluated using the Response Assessment in Neuro-Oncology (RANO) criteria.
Same as current
Complete list of historical versions of study NCT01349036 on Archive Site
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A Phase 2 Study of PLX3397 in Patients With Recurrent Glioblastoma
A Phase 2 Study of Orally Administered PLX3397 in Patients With Recurrent Glioblastoma
The objective of this study is to evaluate the response of subjects with recurrent glioblastoma to continuous therapy of PLX3397.
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Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Recurrent Glioblastoma
Drug: PLX3397
Capsules administered once or twice daily, continuous dosing
  • Experimental: PLX3397-Cohort 1
    10 patients with recurrent glioblastoma who require reoperation will be treated with PLX3397 for 7 days prior to surgery and their tumor tissue will be evaluated for pharmacokinetic levels and pharmacodynamic effects.
    Intervention: Drug: PLX3397
  • Experimental: PLX3397-Cohort 2
    30 patients will be orally dosed with PLX3397 continuously on 28 day cycles.
    Intervention: Drug: PLX3397
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
January 2015
February 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female patients ≥18 years old with a life expectancy of at least 8 weeks
  • Radiographically proven recurrent (≥ first relapse), intracranial GBM
  • For all patients, availability of at least 10 unstained slides (or archival tumor block sufficient to generate at least 10 unstained slides) from any previous GBM surgery
  • Previous treatment with external beam radiation and temozolomide chemotherapy
  • Before the first dose of PLX3397,adequate recovery from toxicity of prior therapy as follows:

>28 days for cytotoxic therapy >42 days for nitrosoureas >28 days for bevacizumab >7 days for non cytotoxic therapy such as interferon, tamoxifen, thalidomide, cis-retinoic acid, or erlotinib

  • Women of child-bearing potential must have a negative pregnancy test within 7 days of initiation of dosing and must agree to use an acceptable method of birth control while on study drug and for 3 months after the last dose. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥1 year. Men of child-bearing potential must also agree to use an acceptable method of birth control while on study drug.
  • Karnofsky performance status of ≥60
  • Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.0 x 109/L, Hgb >9 g/dL, platelet count ≥50 x 109/L, AST/ALT ≤2.5x ULN, creatinine ≤1.5x ULN)
  • Willing and able to provide written informed consent prior to any study related procedures and to comply with all study requirements

Exclusion Criteria:

  • Investigational drug use within 28 days of the first dose of PLX3397
  • GBM progression within 3 months of previous radiation by RANO criteria
  • History of Grade 2 (CTCAE v4) or greater acute intracranial hemorrhage
  • Previous failure of bevacizumab or other VEGF therapy except in a first line setting
  • History of malignant glioma with co-deletion of 1p/19q
  • A concurrent active cancer that requires non-surgical therapy (e.g. chemotherapy, radiation, adjuvant therapy). Prior history of other cancer is allowed, as long as there was no active disease within the prior 3 years.
  • Refractory nausea and vomiting, malabsorption, biliary shunt, or significant bowel resection that would preclude adequate absorption
  • Patients with serious illnesses, uncontrolled infection, medical conditions, or other medical history including abnormal laboratory results, which in the investigator's opinion would be likely to interfere with a patient's participation in the study, or with the interpretation of the results
  • Women of child-bearing potential who are pregnant or breast feeding
  • QTc ≥450 msec at Screening
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
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April 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP