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Phase I Dose Escalation Study of VS-4718 in Subjects With Metastatic Non-Hematologic Malignancies

Phase 1
Terminated
Conditions
Non Hematologic Cancers
Metastatic Cancer
Interventions
Registration Number
NCT01849744
Lead Sponsor
Verastem, Inc.
Brief 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). 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
48
Inclusion Criteria
  • 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
  • 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.
Exclusion Criteria
  • 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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
VS-4718VS-4718Oral VS-4718 administered BID (QD during first cohort) during a 28 day cycle.
Primary Outcome Measures
NameTimeMethod
Assess the safety and tolerability of VS-4718 in subjects with metastatic non-hematologic malignanciesExpected 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 malignanciesFrom 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.

Secondary Outcome Measures
NameTimeMethod
Assess the pharmacokinetics of VS-4718Time points on Day 1, 2, 8, 15, 16, and 29

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

Evaluate biomarkers of VS-4718 activityDay 1 and Day 15 of treatment

Pre and post dose biomarker analysis in serum and tumor samples to identify possible prognostic factors to VS-4718 response

Evaluate the efficacy of VS-4718Every 8 weeks to end of treatment, expected average of 16 weeks

Response rate and progression-free survival as determined by Response Evaluation Criteria In Solid Tumors (RECIST), version 1.1

Evaluate duration of response to VS-4718 compared with duration of response to prior therapy.Expected average of 16 weeks from start of treatment to end of treatment
Examine if the tumor expression status of pFAK and other plasma biomarkers correlates with response to VS-4718 therapyFrom start of treatment to end of treatment, an expected average of 16 weeks

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

Trial Locations

Locations (5)

Washington University School of Medicine, Division of Oncology

🇺🇸

Saint Louis, Missouri, United States

HonorHealth Research Institute

🇺🇸

Scottsdale, Arizona, United States

Samuel Oschin Comprehensive Cancer Institute, Cedars-Sinai Medical Center

🇺🇸

Los Angeles, California, United States

Florida Cancer Specialists

🇺🇸

Sarasota, Florida, United States

Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

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