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Window of Opportunity Study of VS-6063 (Defactinib) in Participants With Surgical Resectable Malignant Pleural Mesothelioma.

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02004028
Recruitment Status : Recruiting
First Posted : December 6, 2013
Last Update Posted : October 15, 2018
Information provided by (Responsible Party):
Verastem, Inc.

Brief Summary:
This is an open label neoadjuvant (treatment with VS-6063 prior to mesothelioma surgery) study in subjects with malignant pleural mesothelioma who are eligible for surgery. Subjects will receive VS-6063 (defactinib) 400 mg twice daily for 12, 21, or 35 days. Pre- and post-treatment biopsies and blood samples will be collected. The purpose of this study is to assess biomarker responses from tumor tissue. The safety, pharmacokinetics, and tumor response rate to VS-6063 (defactinib) will be also be assessed.

Condition or disease Intervention/treatment Phase
Malignant Pleural Mesothelioma Drug: VS-6063 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Window of Opportunity Phase II Study of the FAK Inhibitor Defactinib VS-6063 in Participants With Surgical Resectable Malignant Pleural Mesothelioma.
Study Start Date : December 2013
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Mesothelioma

Arm Intervention/treatment
Experimental: VS-6063 (defactinib)
Administered orally (BID) for 12, 21 or 35 days (+/- 2 days)
Drug: VS-6063
Other Name: defactinib

Primary Outcome Measures :
  1. Assess biomarker responses to VS-6063 in tumor tissue [ Time Frame: From Baseline to 12, 21 or 35 days (+/- 2 days) post-treatment ]
    To determine biomarker responses to the Focal Adhesion Kinase (FAK) inhibitor, VS-6063 (defactinib), in Malignant Pleural Mesothelioma (MPM).

Secondary Outcome Measures :
  1. Evaluate the safety of VS-6063 (defactinib) [ Time Frame: Start of treatment through 30 days after the end of treatment, expected average of 6-8 weeks ]
    Adverse events will be graded by the CTCAE (Common Terminology Criteria for Adverse Events) 4.0 and summarized according to the worst grade observed since the first treatment dose.

  2. Evaluate the pharmacokinetics of VS-6063 (defactinib) [ Time Frame: Day 11 and Day 12, Day 21 and 28, or Day 35 and 42 (+/-2) days post-treatment ]
    PK (pharmacokinetic) parameters will include but not limited to peak and trough concentrations and AUC (area under curve), clearance, and elimination half-life will be characterized by average and variation summaries in the population.

  3. To evaluate the tumor response to VS-6063 (defactinib) [ Time Frame: Baseline and 12, 21 or 35 (+/-2) days post-treatment ]
    Tumor response will be assessed by Recist 1.1 modified for mesothelioma. The response rate will be reported with 95% exact confidence intervals based on the exact binomial distribution.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed malignant pleural mesothelioma that is not metastatic or unresectable
  • Eligible to undergo excisional surgery such as pleurectomy/decortication (P/DC) or any other mesothelioma surgery.
  • Localized disease. The malignancy is confined to one affected hemithorax. Mediastinal N2 lymph nodes via cervical mediastinoscopy or EBUS (endobronchial ultrasound) must be negative in order to be eligible
  • Grossly normal pulmonary, cardiac function, renal, hepatic hematologic and performance functions
  • Male or non-pregnant female
  • Age ≥ 18 years of age
  • Tissue is required prior to enrollment. If patient was diagnosed outside and tumor tissue is not available, a pleural biopsy for frozen tissue collection is required.

Exclusion Criteria:

  • Participants who have had chemotherapy or radiotherapy any time prior to entering the study or at any prior time for mesothelioma. Patients receiving chemotherapy type drugs for benign conditions can participate in this trial
  • History of upper gastrointestinal bleeding, ulceration, or perforation within 12 months prior to the first dose of study drug
  • Known history of Gilbert's Syndrome or any current hyperbilirubinemia of any cause
  • Known history of stroke or cerebrovascular accident within 6 months prior to the first dose of study drug
  • Subjects with known infection with human immunodeficiency virus (HIV) or Acquired Immune Deficiency Syndrome (AIDS)
  • Subjects with confirmed Hepatitis A, B or C
  • Subjects being actively treated for a secondary malignancy or any malignancy within the last 3 years, with the exception of non-melanomatous skin cancer or localized, definitively treated cervical cancer. Men under observation for local prostate cancer are also eligible if they have had stable disease for at least 1 year.
  • Uncontrolled or severe cardiovascular disease, including myocardial infarct or unstable angina within 6 months prior to study treatment, New York Heart Association (NYHA) Class II or greater congestive heart failure, serious arrhythmias requiring medication for treatment, clinically significant pericardial disease, or cardiac amyloidosis
  • Known history of malignant hypertension
  • Uncontrolled intercurrent illness including symptomatic congestive heart failure, cardiac arrhythmia, or psychiatric illness/social situations which in the opinion of the study investigators would be associated with undue risk of participation in the study
  • Use of an investigational drug within 28 days or 5 half-lives prior to first dose.
  • Pregnant or breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02004028

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Contact: Laura Massey 781-469-1542
Contact: Deborah Lloyd 781-469-1583

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United States, Massachusetts
Brigham and Women's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Julie Barlow, MD MPH    617-525-8704   
Contact: Raphael Bueno, M.D.    617-732-8148   
Principal Investigator: Raphael Bueno, M.D.         
Sub-Investigator: David Jackman, M.D.         
Sponsors and Collaborators
Verastem, Inc.
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Study Chair: Raphael Bueno, M.D. Brigham and Women's Hospital

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Responsible Party: Verastem, Inc. Identifier: NCT02004028     History of Changes
Other Study ID Numbers: VS-6063-203
First Posted: December 6, 2013    Key Record Dates
Last Update Posted: October 15, 2018
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Verastem, Inc.:
Neoplasms, Mesothelial
Focal Adhesion Kinase Inhibitor

Additional relevant MeSH terms:
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Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Mesothelial