Relapsed or Refractory Acute Myeloid LeukemiaPhase 1NCT02215629
Dose Escalation Study in Acute Myeloid or B-Cell Acute Lymphoblastic Leukemia
A Phase 1 study testing VS-4718 in people with Relapsed or Refractory Acute Myeloid Leukemia and Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia, run by Verastem, Inc..
Trial summary
The main purpose of this study is to test the safety and efficacy of VS-4718 in two types of leukemia patients and to find the right dose of VS-4718 for future clinical trials. Other purposes of this study include: Testing for study drug VS-4718 levels in blood over time and what happens to the study drug in patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Relapsed or Refractory Acute Myeloid Leukemia, Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
- 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
- 0 participants
- Where
- No sites listed yet
- Study dates
- Start not givento Nov 2016
- 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
- At least 18 years of age
- Pathologic confirmation of AML or B-ALL
- Must have relapsed or refractory AML or B-ALL with no alternate therapy of proven benefit
- ECOG status of 0 or 1
- Adequate renal function [creatinine less than or equal to 1.5x ULN] or GFR of at least 60mL/min
- Adequate hepatic function via total bilirubin, AST, and ALT
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- Corrected QT interval of less than 470 ms (via Fridericia correction formula)
- Negative pregnancy test for women of child bearing potential
- Willingness to use adequate birth control throughout participation for both men and women
Who cannot join
- Diagnosis of acute promyelocytic leukemia
- Active grade 2 or higher acute GVHD at time of study entry or active chronic GVHD (moderate or severe)
- Gastrointestinal conditions which could interfere with the swallowing or absorption of study medication
- Diagnosis of currently active CNS leukemia
- Known infection with HIV or AIDS (testing not required)
- Known active Hepatitis A, B or C (testing not required)
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- Patients being actively treated for a secondary malignancy
- Cancer-directed therapy within 14 days of the first dose of study drug or 5 half-lives, whichever is longer
- 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 longer
- Women who are pregnant or breastfeeding
- Evidence of uncontrolled infections requiring antibiotic therapy; potential subjects with known or suspected infections on stable antibiotic therapy for 72 hours may be enrolled
- Uncontrolled intercurrent illness including symptomatic congestive heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
About this study
Official title: A Phase I Dose Escalation Study of VS-4718, A Focal Adhesion Kinase Inhibitor, In Subjects With Relapsed or Refractory Acute Myeloid Leukemia or B-Cell Acute Lymphoblastic Leukemia
The main purpose of this study is to test the safety and efficacy of VS-4718 in two types of leukemia patients and to find the right dose of VS-4718 for future clinical trials.
Other purposes of this study include:
- Testing for study drug VS-4718 levels in blood over time and what happens to the study drug in patients.
- To find out if there are certain biomarkers in leukemia patients that predict if and how 4718 study drug may or may not work.
- Conditions
- Relapsed or Refractory Acute Myeloid Leukemia, Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
- Being tested
- VS-4718
- Keywords
- Leukemia, Acute Leukemia, Relapsed Leukemia, Refractory Leukemia, Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia
- Medical subject headings
- Recurrence, Leukemia, Myeloid, Acute, Burkitt Lymphoma, Leukemia
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 |
|---|---|---|
| Experimental VS4718 | Experimental | Oral VS-4718 administered BID 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.
To find the maximum tolerated dose of VS-4718
In the absence of a dose limiting toxicity (DLT), each subject will receive VS-4718 for a minimum of 28 days of continuous daily dosing (1 cycle), and may continue to receive additional cycles until disease progression or other treatment discontinuation criteria have been met. It is estimated that subjects could potentially receive VS-4718 for up to an additional 2-3 cycles before withdrawal criteria apply.
Measured: Dose Escalation will occurr according to the standard 3+3 design where three subjects are dosed with the same dosage in the first Cycle (of 28 days); subjects will be evaluated for DLTs on Days: 1, 8, 15, 22 and 28 of first Cycle
Safety and Tolerability of VS-4718 Measurements
Specific key measures/observations used to assess safety and tolerability will be: serious adverse events, incidence and severity of adverse events, physical exams (including vital sign measurements), ECGs, and clinical laboratory evaluations (chemistry, hematology, coagulation, urinalysis)
Measured: Safety and tolerability of VS-4718 will be evaluated in each Cycle of 28 days; more specifically, evaluations will occur at a minimum on Days 1, 8, 15, 22, and 28 of the first Cycle
Secondary outcomes (1)
Measure Pharmacokinetics of VS-4718
Systemic VS-4718 concentration as measured in plasma samples versus time by dosing cohort using descriptive statistics
Measured: Measured concentrations during Cycle 1 on Days 1, 2, 15, and 16. Also on Cycle 2 Day 1 for subjects that continue on study after Cycle 1.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2014 (estimated)
- Primary completionSep 2016 (estimated)
- Study completionNov 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed or Refractory Acute Myeloid LeukemiaStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 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
- NCT02215629
- Sponsor's protocol ID
- VS-4718-102
- Status last verified
- Oct 2015
- First submitted
- 7 Aug 2014
- Last updated
- 27 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Dose Escalation Study of VS-4718, A Focal Adhesion Kinase Inhibitor, In Subjects With Relapsed or Refractory Acute Myeloid Leukemia or B-Cell Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT02215629
- Acronym / sponsor ID
- VS-4718-102
- Conditions
- Relapsed or Refractory Acute Myeloid Leukemia, Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
- Keywords
- Leukemia, Acute Leukemia, Relapsed Leukemia, Refractory Leukemia, Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Verastem, Inc.Industry (pharma, biotech, devices)
- Primary completion
- Sep 2016 (estimated)
- Study completion
- Nov 2016 (estimated)
- First posted
- 13 Aug 2014 (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.
The main purpose of this study is to test the safety and efficacy of VS-4718 in two types of leukemia patients and to find the right dose of VS-4718 for future clinical trials.
Other purposes of this study include:
- Testing for study drug VS-4718 levels in blood over time and what happens to the study drug in patients.
- To find out if there are certain biomarkers in leukemia patients that predict if and how 4718 study drug may or may not work.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental VS4718 | Experimental | Drug: VS-4718Oral VS-4718 administered BID 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)
- At least 18 years of age
- Pathologic confirmation of AML or B-ALL
- Must have relapsed or refractory AML or B-ALL with no alternate therapy of proven benefit
- ECOG status of 0 or 1
- Adequate renal function [creatinine less than or equal to 1.5x ULN] or GFR of at least 60mL/min
Key exclusion criteria (13 listed)
- Diagnosis of acute promyelocytic leukemia
- Active grade 2 or higher acute GVHD at time of study entry or active chronic GVHD (moderate or severe)
- Gastrointestinal conditions which could interfere with the swallowing or absorption of study medication
- Diagnosis of currently active CNS leukemia
- Known infection with HIV or 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 |
|---|---|---|
| VS-4718 | Drug | Experimental VS4718 |
Primary outcome
To find the maximum tolerated dose of VS-4718
Time frame: Dose Escalation will occurr according to the standard 3+3 design where three subjects are dosed with the same dosage in the first Cycle (of 28 days); subjects will be evaluated for DLTs on Days: 1, 8, 15, 22 and 28 of first Cycle
In the absence of a dose limiting toxicity (DLT), each subject will receive VS-4718 for a minimum of 28 days of continuous daily dosing (1 cycle), and may continue to receive additional cycles until disease progression or other treatment discontinuation criteria have been met. It is estimated that subjects could potentially receive VS-4718 for up to an additional 2-3 cycles before withdrawal criteria apply.
Safety and Tolerability of VS-4718 Measurements
Time frame: Safety and tolerability of VS-4718 will be evaluated in each Cycle of 28 days; more specifically, evaluations will occur at a minimum on Days 1, 8, 15, 22, and 28 of the first Cycle
Specific key measures/observations used to assess safety and tolerability will be: serious adverse events, incidence and severity of adverse events, physical exams (including vital sign measurements), ECGs, and clinical laboratory evaluations (chemistry, hematology, coagulation, urinalysis)
1 secondary outcome
Measure Pharmacokinetics of VS-4718
Time frame: Measured concentrations during Cycle 1 on Days 1, 2, 15, and 16. Also on Cycle 2 Day 1 for subjects that continue on study after Cycle 1.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 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.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VS-4718 in people with Relapsed or Refractory Acute Myeloid Leukemia and Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia, run by Verastem, Inc..
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 13 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: withdrawn, as of the sponsor's last update on 27 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
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 withdrawn before enrolling participants, so there are no results.
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?
To find the maximum tolerated dose of VS-4718 (time frame: Dose Escalation will occurr according to the standard 3+3 design where three subjects are dosed with the same dosage in the first Cycle (of 28 days); subjects will be evaluated for DLTs on Days: 1, 8, 15, 22 and 28 of first Cycle). 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 27 Jan 2017.