Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

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.
 
ClinicalTrials.gov Identifier: NCT02004028
Recruitment Status : Terminated (Company decided to discontinue trial to focus on development program next steps)
First Posted : December 6, 2013
Last Update Posted : October 22, 2019
Sponsor:
Information provided by (Responsible Party):
Verastem, Inc.

Tracking Information
First Submitted Date  ICMJE December 2, 2013
First Posted Date  ICMJE December 6, 2013
Last Update Posted Date October 22, 2019
Actual Study Start Date  ICMJE December 12, 2013
Actual Primary Completion Date June 19, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 19, 2016)
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).
Original Primary Outcome Measures  ICMJE
 (submitted: December 2, 2013)
Assess biomarker responses to VS-6063 in tumor tissue [ Time Frame: From Baseline to 12 days (+/- 2 days) post-treatment ]
To determine biomarker responses to the Focal Adhesion Kinase (FAK) inhibitor, VS-6063 (defactinib), in Malignant Pleural Mesothelioma (MPM).
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 19, 2016)
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: December 2, 2013)
  • Evaluate the safety of VS-6063 (defactinib) [ Time Frame: Start of treatment through 30 days after the end of treatment, expected average of 6 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.
  • Evaluate the pharmacokinetics of VS-6063 (defactinib) [ Time Frame: Day 11 and Day 12 (+/-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.
  • To evaluate the tumor response to VS-6063 (defactinib) [ Time Frame: Baseline and 12 (+/-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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Window of Opportunity Study of VS-6063 (Defactinib) in Surgical Resectable Malignant Pleural Mesothelioma Participants
Official Title  ICMJE An Open Label Window of Opportunity Phase II Study of the FAK Inhibitor Defactinib VS-6063 in Participants With Surgical Resectable Malignant Pleural Mesothelioma.
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 or 100 mg formulation twice daily for 21 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Malignant Pleural Mesothelioma
Intervention  ICMJE Drug: VS-6063
Other Name: defactinib
Study Arms  ICMJE Experimental: VS-6063 (defactinib)
Administered orally (BID) for 12, 21 or 35 days (+/- 2 days)
Intervention: Drug: VS-6063
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: October 18, 2019)
35
Original Estimated Enrollment  ICMJE
 (submitted: December 2, 2013)
20
Actual Study Completion Date  ICMJE June 19, 2019
Actual Primary Completion Date June 19, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02004028
Other Study ID Numbers  ICMJE VS-6063-203
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Verastem, Inc.
Study Sponsor  ICMJE Verastem, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Raphael Bueno, M.D. Brigham and Women's Hospital
PRS Account Verastem, Inc.
Verification Date October 2019

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