Non Hematologic CancersPhase 1NCT01849744
Phase I Dose Escalation Study of VS-4718 in Subjects With Metastatic Non-Hematologic Malignancies
A Phase 1 study testing VS-4718 in people with Non Hematologic Cancers and Metastatic Cancer, run by Verastem, Inc. at 5 sites in United States.
Trial summary
This is a Phase I, open-label, multicenter, dose-escalation trial of VS-4718, a focal adhesion kinase inhibitor, in subjects with metastatic non-hematologic malignancies. This clinical study is comprised of 2 parts: Part 1 (Dose Escalation) and Part 2 (Expansion). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non Hematologic Cancers, Metastatic Cancer
- What is being tested?
- VS-4718
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 5 sitesUnited States
- Study dates
- Jun 2013to Jun 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Age ≥ 18 years.
- Histopathologically confirmed diagnosis of a metastatic non-hematologic malignancy.
- ECOG (Eastern Cooperative Oncology Group) performance status of ≤ 2
- Adequate renal function
- Adequate hepatic function (total bilirubin ≤ 1.5x ULN (upper limit of normal) for the institution; AST [aspartate transaminase] and ALT [alanine transaminase] ≤ 3x ULN, or ≤ 5x ULN if due to liver involvement by tumor).
- Adequate bone marrow function (hemoglobin ≥ 9.0 g/dL; unsupported platelets ≥ 100 x10 9 cells/L; absolute neutrophil count ≥ 1.5x10 9 cells/L
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- Corrected QT interval (QTc) < 470 ms
- Subjects must have at least one tumor lesion that is suitable for repeat biopsy, and must agree to two tumor biopsies (pre- and post- treatment).
- Willing and able to participate in the trial and comply with all trial requirements.
Who cannot join
- Gastrointestinal (GI) condition which could interfere with the swallowing or absorption of study medication.
- Uncontrolled or severe concurrent medical condition (including uncontrolled brain metastases).
- History of upper gastrointestinal bleeding, ulceration, or perforation within 12 months.
- Known history of stroke or cerebrovascular accident within 6 months.
- Subjects being actively treated for a secondary malignancy.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Samuel Oschin Comprehensive Cancer Institute, Cedars-Sinai Medical Center
Los Angeles, California, 90048
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
Florida Cancer Specialists
Sarasota, Florida, 34232
HonorHealth Research Institute
Scottsdale, Arizona, 85258
Washington University School of Medicine, Division of Oncology
St Louis, Missouri, 63110
About this study
Official title: A Phase I Study of VS-4718, a Focal Adhesion Kinase Inhibitor, in Subjects With Metastatic Non-Hematologic Malignancies
This is a Phase I, open-label, multicenter, dose-escalation trial of VS-4718, a focal adhesion kinase inhibitor, in subjects with metastatic non-hematologic malignancies. This clinical study is comprised of 2 parts: Part 1 (Dose Escalation) and Part 2 (Expansion). The purpose of this study is to evaluate the safety (including the recommended Phase II dose), pharmacokinetics (the amount of VS-4718 in your blood) and the anti-cancer activity of VS-4718. The pharmacodynamic effects (genes or proteins that may predict or show how your body may respond to VS-4718) will also be examined in tumor biopsies and blood samples.
- Conditions
- Non Hematologic Cancers, Metastatic Cancer
- Being tested
- VS-4718
- Keywords
- Focal Adhesion Kinase inhibitor, FAK inhibitor, Cancer Stem Cells, CSC
- Medical subject headings
- Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VS-4718Drug
| Study group | Role | What they receive |
|---|---|---|
| VS-4718 | Experimental | Oral VS-4718 administered BID (QD during first cohort) during a 28 day cycle. Receives: Drug: VS-4718 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the safety and tolerability of VS-4718 in subjects with metastatic non-hematologic malignancies
Serious Adverse events, Adverse events and their frequency, duration and severity, physical examination, laboratory parameters, vital signs and ECGs as determined based on CTCAE (Common Toxicity Criteria for Adverse Effects) V4.03. A Safety monitoring committee will review safety information.
Measured: Expected average of 12 weeks from start of treatment to end of treatment
Establish the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D) of VS-4718 in subjects with metastatic non-hematologic malignancies
The RP2D will be determined based on the maximum tolerated dose (MTD) of VS-4718 as determined by number of participants with dose limiting toxicities related to VS-4718. Observations related to pharmacokinetics, pharmacodynamics, and any VS-4718 related toxicities may be included in the rationale supporting the RP2D and will not exceed the MTD.
Measured: From start of treatment to end of cycle 1 (4 week cycles)
Secondary outcomes (5)
Evaluate the efficacy of VS-4718
Response rate and progression-free survival as determined by Response Evaluation Criteria In Solid Tumors (RECIST), version 1.1
Measured: Every 8 weeks to end of treatment, expected average of 16 weeks
Evaluate duration of response to VS-4718 compared with duration of response to prior therapy.
Measured: Expected average of 16 weeks from start of treatment to end of treatment
Assess the pharmacokinetics of VS-4718
PK (pharmacokinetics) parameters, including but not limited to clearance, plasma concentration, AUC (Area Under Curve, 0-24 and 0-t), Cmax, Tmax, and T1/2
Measured: Time points on Day 1, 2, 8, 15, 16, and 29
Evaluate biomarkers of VS-4718 activity
Pre and post dose biomarker analysis in serum and tumor samples to identify possible prognostic factors to VS-4718 response
Measured: Day 1 and Day 15 of treatment
Examine if the tumor expression status of pFAK and other plasma biomarkers correlates with response to VS-4718 therapy
Tumor expression status (pFAK, cancer stem cells, CSC, and other biomarkers) compared with response to VS-4718, as determined by Response Evaluation Criteria In Solid Tumors (RECIST), version 1.1
Measured: From start of treatment to end of treatment, an expected average of 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2013 (estimated)
- Study startJun 2013
- Primary completionApr 2017
- Study completionJun 2017
Why it stopped: Sponsor's decision to deprioritize the program.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non Hematologic CancersStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Verastem, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hagop Youssoufian, MD, Study chair, Verastem, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01849744
- Sponsor's protocol ID
- VS-4718-101
- Status last verified
- Jul 2017
- First submitted
- 6 May 2013
- Last updated
- 28 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of VS-4718, a Focal Adhesion Kinase Inhibitor, in Subjects With Metastatic Non-Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT01849744
- Acronym / sponsor ID
- VS-4718-101
- Conditions
- Non Hematologic Cancers, Metastatic Cancer
- Keywords
- Focal Adhesion Kinase inhibitor, FAK inhibitor, Cancer Stem Cells, CSC
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor's decision to deprioritize the program.
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Verastem, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2013
- Primary completion
- Apr 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 8 May 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase I, open-label, multicenter, dose-escalation trial of VS-4718, a focal adhesion kinase inhibitor, in subjects with metastatic non-hematologic malignancies. This clinical study is comprised of 2 parts: Part 1 (Dose Escalation) and Part 2 (Expansion). The purpose of this study is to evaluate the safety (including the recommended Phase II dose), pharmacokinetics (the amount of VS-4718 in your blood) and the anti-cancer activity of VS-4718. The pharmacodynamic effects (genes or proteins that may predict or show how your body may respond to VS-4718) will also be examined in tumor biopsies and blood samples.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VS-4718 | Experimental | Drug: VS-4718Oral VS-4718 administered BID (QD during first cohort) during a 28 day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Age ≥ 18 years.
- Histopathologically confirmed diagnosis of a metastatic non-hematologic malignancy.
- ECOG (Eastern Cooperative Oncology Group) performance status of ≤ 2
- Adequate renal function
- Adequate hepatic function (total bilirubin ≤ 1.5x ULN (upper limit of normal) for the institution; AST [aspartate transaminase] and ALT [alanine transaminase] ≤ 3x ULN, or ≤ 5x ULN if due to liver involvement by tumor).
Key exclusion criteria (5 listed)
- Gastrointestinal (GI) condition which could interfere with the swallowing or absorption of study medication.
- Uncontrolled or severe concurrent medical condition (including uncontrolled brain metastases).
- History of upper gastrointestinal bleeding, ulceration, or perforation within 12 months.
- Known history of stroke or cerebrovascular accident within 6 months.
- Subjects being actively treated for a secondary malignancy.
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-4718 | Drug | VS-4718 |
Primary outcome
Assess the safety and tolerability of VS-4718 in subjects with metastatic non-hematologic malignancies
Time frame: Expected average of 12 weeks from start of treatment to end of treatment
Serious Adverse events, Adverse events and their frequency, duration and severity, physical examination, laboratory parameters, vital signs and ECGs as determined based on CTCAE (Common Toxicity Criteria for Adverse Effects) V4.03. A Safety monitoring committee will review safety information.
Establish the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D) of VS-4718 in subjects with metastatic non-hematologic malignancies
Time frame: From start of treatment to end of cycle 1 (4 week cycles)
The RP2D will be determined based on the maximum tolerated dose (MTD) of VS-4718 as determined by number of participants with dose limiting toxicities related to VS-4718. Observations related to pharmacokinetics, pharmacodynamics, and any VS-4718 related toxicities may be included in the rationale supporting the RP2D and will not exceed the MTD.
5 secondary outcomes
Evaluate the efficacy of VS-4718
Time frame: Every 8 weeks to end of treatment, expected average of 16 weeks
Evaluate duration of response to VS-4718 compared with duration of response to prior therapy.
Time frame: Expected average of 16 weeks from start of treatment to end of treatment
Assess the pharmacokinetics of VS-4718
Time frame: Time points on Day 1, 2, 8, 15, 16, and 29
Evaluate biomarkers of VS-4718 activity
Time frame: Day 1 and Day 15 of treatment
Examine if the tumor expression status of pFAK and other plasma biomarkers correlates with response to VS-4718 therapy
Time frame: From start of treatment to end of treatment, an expected average of 16 weeks
Results and safety
The study was stopped early (reason given: Sponsor's decision to deprioritize the program.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2017.
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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.
Hagop Youssoufian, MD study chair
Verastem, Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VS-4718 in people with Non Hematologic Cancers and Metastatic Cancer, run by Verastem, Inc. at 5 sites 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 9 inclusion and 5 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 28 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor's decision to deprioritize the program.). Results are not available in the source record.
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?
Assess the safety and tolerability of VS-4718 in subjects with metastatic non-hematologic malignancies (time frame: Expected average of 12 weeks from start of treatment to end of treatment). The study lists 2 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 28 Jul 2017.