Malignant Pleural MesotheliomaPhase 2NCT02004028
Window of Opportunity Study of VS-6063 (Defactinib) in Surgical Resectable Malignant Pleural Mesothelioma Participants
A Phase 2 study testing VS-6063 in people with Malignant Pleural Mesothelioma, run by Verastem, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Malignant Pleural Mesothelioma
- What is being tested?
- VS-6063
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Dec 2013to 19 Jun 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Brigham and Women's Hospital
Boston, Massachusetts, 02115
About this study
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.
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.
- Conditions
- Malignant Pleural Mesothelioma
- Being tested
- VS-6063
- Keywords
- Mesothelioma, adenoma, Neoplasms, Mesothelial, Focal Adhesion Kinase Inhibitor
- Medical subject headings
- Mesothelioma, Malignant, Mesothelioma, Adenoma, Neoplasms, Mesothelial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VS-6063Drug
Also called defactinib
| Study group | Role | What they receive |
|---|---|---|
| VS-6063 (defactinib) | Experimental | Administered orally (BID) for 12, 21 or 35 days (+/- 2 days) Receives: Drug: VS-6063 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of pFAK Inhibition in Tumor Tissue
percentage VS-6063 (defactinib)
Measured: Cohort 1, From Baseline to Day 12; Cohort 2, From Baseline to Day 35; Cohorts 3, From Baseline to Day 21; Cohorts 3, From Baseline to day 21 day.
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), CMax
Maximum observed plasma concentration
Measured: 0-24 hours
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), AUC (Area Under the Curve)
Area under plasma Concentration (AUC) 0 to t
Measured: 0-8 hours
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), Median Tmax (h)
Time to Maximum concentration (Tmax)
Measured: 0-24 hours
Secondary outcomes (2)
Number of Patients With at Least One Adverse Event
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.
Measured: Cohort 1, 40 days; Cohort 2, 42 days; Cohort 3, 28 days; Cohort 4, 28 days.
To Evaluate the Tumor Response to VS-6063 (Defactinib)
Modified RECIST criteria for assessment of response in malignant pleural mesothelioma Ann Oncol 2004. Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the in the volume of target lesions; Progressive Disease (PD) at least a 20% increase in the volume of target lesions Stable Disease (SD):
Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of the longest diameter.
Measured: Cohort 1, 40 days; Cohort 2, 42 days; Cohort 3, 28 days; Cohort 4, 28 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2013 (estimated)
- Study start12 Dec 2013
- Primary completion19 Jun 2019
- Study completion19 Jun 2019
Why it stopped: Company decided to discontinue trial to focus on development program next steps
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Malignant Pleural MesotheliomaStudies of the same condition, open and under way.
- More trials run by Verastem, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Verastem, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Raphael Bueno, M.D., Study chair, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02004028
- Sponsor's protocol ID
- VS-6063-203
- Status last verified
- Feb 2024
- First submitted
- 2 Dec 2013
- Last updated
- 20 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT02004028
- Acronym / sponsor ID
- VS-6063-203
- Conditions
- Malignant Pleural Mesothelioma
- Keywords
- Mesothelioma, adenoma, Neoplasms, Mesothelial, Focal Adhesion Kinase Inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company decided to discontinue trial to focus on development program next steps
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Verastem, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Dec 2013 (actual)
- Primary completion
- 19 Jun 2019 (actual)
- Study completion
- 19 Jun 2019 (actual)
- First posted
- 6 Dec 2013 (estimated)
- Results
- Posted 20 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VS-6063 (defactinib) | Experimental | Drug: VS-6063Administered orally (BID) for 12, 21 or 35 days (+/- 2 days) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (12 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VS-6063Also: defactinib | Drug | VS-6063 (defactinib) |
Primary outcome
Percentage of pFAK Inhibition in Tumor Tissue
Time frame: Cohort 1, From Baseline to Day 12; Cohort 2, From Baseline to Day 35; Cohorts 3, From Baseline to Day 21; Cohorts 3, From Baseline to day 21 day.
percentage VS-6063 (defactinib)
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), CMax
Time frame: 0-24 hours
Maximum observed plasma concentration
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), AUC (Area Under the Curve)
Time frame: 0-8 hours
Area under plasma Concentration (AUC) 0 to t
Evaluate the Pharmacokinetics of VS-6063 (Defactinib), Median Tmax (h)
Time frame: 0-24 hours
Time to Maximum concentration (Tmax)
2 secondary outcomes
Number of Patients With at Least One Adverse Event
Time frame: Cohort 1, 40 days; Cohort 2, 42 days; Cohort 3, 28 days; Cohort 4, 28 days.
To Evaluate the Tumor Response to VS-6063 (Defactinib)
Time frame: Cohort 1, 40 days; Cohort 2, 42 days; Cohort 3, 28 days; Cohort 4, 28 days.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Raphael Bueno, M.D. study chair
Brigham and Women's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VS-6063 in people with Malignant Pleural Mesothelioma, run by Verastem, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of pFAK Inhibition in Tumor Tissue (time frame: Cohort 1, From Baseline to Day 12; Cohort 2, From Baseline to Day 35; Cohorts 3, From Baseline to Day 21; Cohorts 3, From Baseline to day 21 day.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2024.