Non Hematologic CancersPhase 1NCT01943292

Phase I Dose Escalation Study of VS-6063 in Japanese Subjects With Non-Hematologic Malignancies

A Phase 1 study testing Defactinib in people with Non Hematologic Cancers, run by Verastem, Inc. at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

This is a phase I, open-label, dose-escalation trial of defactinib (VS-6063), a focal adhesion kinase inhibitor, in Japanese patients with non-hematologic malignancies. The purpose of this study is to assess the safety (including the recommended phase 2 dose), the pharmacokinetics, and the anti-cancer activity of… Read more about this study

Who is it for?: Ages 20 years and older
Conditions the study is for: Non Hematologic Cancers
What is being tested?: Defactinib
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 1 siteJapan
Study dates: 2 Sep 2013to 9 Jun 2014
Phase: Phase 1
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 20 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Able to provide signed and dated informed consent prior to initiation of any study procedures.
Age ≥ 20 years.
Subject must be of Japanese descent.
Subjects must have a histopathologically confirmed diagnosis of a non-hematologic malignancy.
Subjects must have no further standard of care options or have refused standard therapy.
All persistent clinically significant toxicities from prior chemotherapy must be ≤ Grade 1.

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ECOG performance status of 0 or 1, measured within 72 hours before the start of treatment.
Predicted life expectancy of ≥ 3 months.
Adequate renal function [creatinine ≤ 1.5x ULN (upper limit of normal)] or GFR of ≥ 50mL/min.
Adequate hepatic function (total bilirubin ≤ 1.5x ULN 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; platelets ≥ 100 x109 cells/L; absolute neutrophil count ≥ 1.5x109 cells/L).
Corrected QT interval (QTc) < 470 ms (as calculated by the Fridericia correction formula).
Negative pregnancy test for women of child-bearing potential. (A woman of childbearing potential is defined as one who is biologically capable of becoming pregnant except for women who have undergone permanent contraceptive surgery or are postmenopausal (defined as the absence of menstruation for at least 12 months without other medical reasons).
Men and women of child bearing potential must agree to use adequate birth control throughout their participation in the study and for 90 days following the last study 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). Stable brain metastases either treated or being treated with a stable dose of steroids/ anticonvulsants, with no dose change within 28 days prior to the first dose of study drug, will be allowed.
History of upper gastrointestinal bleeding, ulceration, or perforation within 12 months prior to the first dose of study drug.
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) (testing not required).
Subjects with known Hepatitis B or C (Including known seropositivity Hepatitis B virus surface antigen (HBsAg), hepatitis C virus antibody (HCV antibody).

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Subjects being actively treated for a secondary malignancy.
Cancer-directed therapy (chemotherapy, radiotherapy, hormonal therapy, biologic or immunotherapy, etc.) within 28 days of the first dose of study drug or 5 half-lives, whichever is shorter.
Major surgery within 28 days prior to the first dose of study drug.
Use of an investigational drug within 28 days or 5 half-lives (whichever is shorter) prior to the first dose of study drug. A minimum of 10 days between termination of the investigational drug and administration of the study treatment is required. In addition, any drug-related toxicity except alopecia should have recovered to grade 1 or less.
Women who are pregnant or breastfeeding.
Any evidence of serious active infections.
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 that would limit compliance with study requirements.

Trial locations and contacts

1 site in 1 country.

Japan1 site

Kinki University Hospital
Osaka

Other trials in Japan

About this study

Official title: A Phase I Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of VS-6063, a Focal Adhesion Kinase Inhibitor, in Japanese Subjects With Non-Hematologic Malignancies

Conditions: Non Hematologic Cancers
Being tested: Defactinib
Keywords: Focal Adhesion Kinase inhibitor, FAK inhibitor, Cancer Stem Cells, CSC

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

DefactinibDrug
Also called VS-6063

Study groups in NCT01943292
Study group	Role	What they receive
Defactinib	Experimental	Oral defactinib (VS-6063) administered twice a day (BID) during a 21 day cycle. Receives: Drug: Defactinib

What is being measured

The primary outcome is the main question the study is designed to answer.

Assess the Safety and Tolerability of Defactinib (VS-6063) in Japanese Subjects With Non-hematologic Malignancies
A composite by dose level to include incidence of AEs, SAEs, dose interruptions and dose reductions as a measure of safety and tolerability. Abnormal Clinical significant laboratory results, ECG measurements, vital signs measurement, physical examination findings, and ECOG performance status were captured as adverse events.
The severity of AEs were evaluated according to CTCAE (Common Toxicity Criteria for Adverse Effects) 4.03
Measured: From start of treatment to end of treatment, an expected average of 12 weeks

Secondary outcomes (3)

Define the Maximum Tolerated Dose (MTD), if Achieved, and Establish the Recommended Phase 2 Dose (RP2D) of Defactinib (VS-6063) in Japanese Subjects.
The RP2D will be determined based on the MTD of defactinib (VS-6063) as determined by number of participants with dose limiting toxicities (DLTs) related to defactinib.
Measured: From start of treatment to end of cycle 1 (21 day cycles)
Assess the Pharmacokinetics, Metabolism and Elimination of Defactinib (VS-6063) in Plasma and Urine.
PK parameters, including but not limited to plasma concentration, AUC (Area Under Curve) 0-t, Cmax, Tmax, and T1/2. Total 24-hour urine output will be collected in conjunction with PK sampling to assess the elimination of defactinib (VS-6063) and its potential metabolites.
Measured: Time points at Day 1 and Day 15 in Cycle 1
Evaluate the Efficacy (Response Rate and Progression-free Survival) of Subjects Treated With Defactinib (VS-6063).
Response rate and progression-free survival, as determined by Response Evaluation Criteria In Solid Tumors (RECIST), version 1.1
Measured: Every 8 weeks up to end of treatment, an expected average of 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Sep 20139 Jun 2014

First posted16 Sep 2013 (estimated)
Study start2 Sep 2013
Primary completion9 Jun 2014
Study completion9 Jun 2014

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 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: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01943292
Sponsor's protocol ID: VS-6063-102
Status last verified: Jan 2016
First submitted: 11 Sep 2013
Last updated: 9 Mar 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 9 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of VS-6063, a Focal Adhesion Kinase Inhibitor, in Japanese Subjects With Non-Hematologic Malignancies
ClinicalTrials.gov ID: NCT01943292
Acronym / sponsor ID: VS-6063-102
Conditions: Non Hematologic Cancers
Keywords: Focal Adhesion Kinase inhibitor, FAK inhibitor, Cancer Stem Cells, CSC
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: Verastem, Inc.Industry (pharma, biotech, devices)
Study start: 2 Sep 2013 (actual)
Primary completion: 9 Jun 2014 (actual)
Study completion: 9 Jun 2014 (actual)
First posted: 16 Sep 2013 (estimated)
Results: Posted 20 Nov 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Defactinib	Experimental	Drug: DefactinibOral defactinib (VS-6063) administered twice a day (BID) during a 21 day cycle.

Who may qualify

Ages: 20 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (15 listed)

Able to provide signed and dated informed consent prior to initiation of any study procedures.
Age ≥ 20 years.
Subject must be of Japanese descent.
Subjects must have a histopathologically confirmed diagnosis of a non-hematologic malignancy.
Subjects must have no further standard of care options or have refused standard therapy.

Key exclusion criteria (15 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). Stable brain metastases either treated or being treated with a stable dose of steroids/ anticonvulsants, with no dose change within 28 days prior to the first dose of study drug, will be allowed.
History of upper gastrointestinal bleeding, ulceration, or perforation within 12 months prior to the first dose of study drug.
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) (testing not required).

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
DefactinibAlso: VS-6063	Drug	Defactinib

Primary outcome

Assess the Safety and Tolerability of Defactinib (VS-6063) in Japanese Subjects With Non-hematologic Malignancies
Time frame: From start of treatment to end of treatment, an expected average of 12 weeks
A composite by dose level to include incidence of AEs, SAEs, dose interruptions and dose reductions as a measure of safety and tolerability. Abnormal Clinical significant laboratory results, ECG measurements, vital signs measurement, physical examination findings, and ECOG performance status were captured as adverse events. The severity of AEs were evaluated according to CTCAE (Common Toxicity Criteria for Adverse Effects) 4.03

3 secondary outcomes

Define the Maximum Tolerated Dose (MTD), if Achieved, and Establish the Recommended Phase 2 Dose (RP2D) of Defactinib (VS-6063) in Japanese Subjects.
Time frame: From start of treatment to end of cycle 1 (21 day cycles)
Assess the Pharmacokinetics, Metabolism and Elimination of Defactinib (VS-6063) in Plasma and Urine.
Time frame: Time points at Day 1 and Day 15 in Cycle 1
Evaluate the Efficacy (Response Rate and Progression-free Survival) of Subjects Treated With Defactinib (VS-6063).
Time frame: Every 8 weeks up to end of treatment, an expected average of 12 weeks

Results and safety

Results posted to the registry on 20 Nov 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Defactinib 200 mg Bid	3	2
Defactinib 400 mg Bid	3	3
Defactinib 600 mg Bid	3	3

Primary outcome: Assess the Safety and Tolerability of Defactinib (VS-6063) in Japanese Subjects With Non-hematologic Malignancies

Time frame: From start of treatment to end of treatment, an expected average of 12 weeks. Number. Unit: participants

Measure	Defactinib 200 mg Bid n = 3	Defactinib 400 mg Bid n = 3	Defactinib 600 mg Bid n = 3
At least 1 TEAE (treatment emergent AE)	3	3	3
At least 1 TEAE related to study drug	3	3	3
Non-Lab TEAE with Severity ≥ Grade 3	0	0	0
Lab TEAE with Severity ≥ Grade 3	1	0	0
At least 1 Serious TEAE	0	0	0
Dose Interruptions	1	1	0
Dose Reductions	0	0	0

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Treatment Emergent Adverse Events (TEAEs) include all events (related and unrelated) from the time patient doses through 30 days post last dose of study treatment. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Defactinib 200 mg Bid	0 of 3 (0%)	3 of 3 (100%)	Not reported
Defactinib 400 mg Bid	0 of 3 (0%)	3 of 3 (100%)	Not reported
Defactinib 600 mg Bid	0 of 3 (0%)	3 of 3 (100%)	Not reported

Limitations and caveats (from the sponsor): There were no limitations of the trial.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2017.
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.

Hagop Youssoufian, m study chair

Verastem, Inc.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Defactinib in people with Non Hematologic Cancers, run by Verastem, Inc. at 1 site in Japan.

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 15 inclusion and 15 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: finished, as of the sponsor's last update on 9 Mar 2017. 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 Japan. 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?

Yes. Results are posted in the registry (first posted 20 Nov 2015). 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?

Assess the Safety and Tolerability of Defactinib (VS-6063) in Japanese Subjects With Non-hematologic Malignancies (time frame: From start of treatment to end of treatment, an expected average of 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01943292.

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 9 Mar 2017.