Clinical trial NCT02215629: Dose Escalation Study in Acute Myeloid or B-Cell Acute Lymphoblastic Leukemia
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
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
Lead sponsor: Verastem, Inc.
Sponsor class: INDUSTRY
First posted: 2014-08-13
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.
* To find out if there are certain biomarkers in leukemia patients that predict if and how 4718 study drug may or may not work.


Eligibility criteria: Inclusion Criteria:

* 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
* 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

Exclusion Criteria:

* 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)
* 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

Primary outcomes:
measure: To find the maximum tolerated dose of VS-4718
timeFrame: 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
measure: Safety and Tolerability of VS-4718 Measurements
timeFrame: 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:
measure: Measure Pharmacokinetics of VS-4718
timeFrame: 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.
Other outcomes:
Locations:
Study officials:
affiliation: Verastem, Inc.
name: Hagop Youssoufian, MD
role: STUDY_CHAIR
References: