AU409 for the Treatment of Advanced Primary Liver Cancers or Solid Tumor With Liver Metastatic Disease

Description

PRIMARY OBJECTIVES:

I. To determine maximum tolerated dose (MTD) of RNA transcription modulator AU-409 (AU409) and the recommended phase II dose (RP2D).

II. To characterize the safety and tolerability of AU409 by assessing toxicities per Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0 criteria.

SECONDARY OBJECTIVES:

I. To obtain a preliminary assessment of anti-tumor activity of AU409 via objective radiologic response using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria.

II. To determine pharmacokinetics of AU409 in patients with advanced-stage solid tumors.

EXPLORATORY OBJECTIVES:

I. To evaluate the concentration of AU409 in tumor tissue from liver biopsy samples obtained from a subset of patients treated with AU409 at dose level 4 (300 mg) and above.

II. To evaluate expression of genes with TATA box promotion regions on pre- and post- treatment liver biopsy samples.

OUTLINE: This is a dose-escalation study.

Patients receive AU409 orally (PO) on study. Patients also undergo computed tomography (CT) or magnetic resonance imaging (MRI) and collection of blood samples throughout the trial.