We encourage participation in clinical trials for research leading to a cure for mesothelioma. It is known that nationally the statistics for participation in adult cancer trials is between 3-5% of adults who have a cancer diagnosis.

For mesothelioma patients and families who are interested in clinical trials, researching clinical trials and eligibility can be a time consuming barrier to participation.   The information about clinical trials is available on www.clinicaltrials.gov .

Our goal is to feature one mesothelioma clinical trial monthly and break it down to understandable terms, to decipher the eligibility requirements, and to possibly encourage participation in these trials.

CLINICAL TRIAL – NCT02004028

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

Official Title- An Open Label Window of Opportunity Phase 11 Study of the FAK Inhibitor VS-6063 in Participants With Surgical Resectable Malignant Pleural Mesothelioma

Listing on www.clinicaltrials.gov

Sponsor-Verastem Inc.

Location- Brigham and Women’s Hospital Boston Massachusetts

Principal Investigator- Raphael Bueno M.D.

Contact- Julianne Barlow- 617-525-8541 jbarlow1@partners.org

Number of Participants- expected to be around 25

Purpose- This clinical trial is for people who have been diagnosed with malignant pleural mesothelioma and are a candidate for surgery. It is a drug given at specific times before surgery to participants who meet the eligibility criteria. The drug is a FAK inhibitor, named Defactinib. This drug will be given prior to surgery. Current therapy is surgery followed by chemotherapy and other treatments. The drug is a type of enzyme inhibitor that blocks the activation of specific signaling pathways in the body. The theory is that blocking these path ways may prevent tumor cells from growing and migrating to different areas of the body. After taking this drug, surgery will be scheduled about 7 days after completion.

Screening- Some of these tests you will undergo if you are considering surgery with or without this clinical trial.

• A medical history
• physical exam
• vital signs
• performance status
• pulmonary function tests
• quantitative ventilation-perfusion scan
• PET scans
• MRI for evaluation of your tumor
• blood tests for biomarkers in addition to routine blood draws, biomarkers are genes or proteins that may predict how your body will respond to the study drug,
• EKG
• Pregnancy test if needed.

Eligibility Criteria

• The patient must have a confirmed diagnosis of malignant pleural mesothelioma. The disease is confined to one side of the chest.
• Be older than 18, male or female, not pregnant, be a candidate for surgery.
• 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.

Exclusions

• Participants who have had chemotherapy or radiotherapy for mesothelioma any time prior to entering the study.
• 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 the 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 the study drug.
• Known infections with human immunodeficiency virus (HIV) or Acquired Immune Deficiency Syndrome (AIDS).
• Confirmed Hepatitis A, B, or C.
• Being actively treated for a secondary malignancy or any malignancy within the last 3 years, with the exception of nonmelanomatous 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.
• Known history of malignant hypertension.
• Uncontrolled intercurrent illness including symptomatic congestive heart failure, cardiac arrhythmias, or psychiatric illness/ social situations.

How the Research Study is Designed to Work

If you are eligible, a biopsy will be done if you don’t have previous tissue available to evaluate the specimens and biomarkers. After the biopsy- the participant starts taking the study drug by mouth twice a day for 35 days. The participant will have a study drug dosing diary. The tumor will be assessed by scans – CT, MRI, and a PET- CT at different points in the study. Different days will require clinic visits at specific times during the trial.

With any clinical trial, all participation is voluntary. Before enrollment a detailed consent form will be reviewed with the participant and signed.

The participant can withdraw from a clinical trial at any point in time. Think about participating if able, the only way progress to a cure for mesothelioma can be made is through clinical trials.