NCT05791448

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

First in Human Dose Escalation Study of AU409 in Patients With Advanced Primary Liver Cancers or Advanced Solid Tumor With Liver Predominant Metastatic Disease

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: University of Southern California · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase I trial tests the safety, side effects, and best dose of a new intervention, AU409, in treating patients with primary liver cancers that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or advanced solid tumors that have spread to the liver (liver metastatic disease). AU409 may stop cancer from growing and spreading. This trial may help researchers determine if AU409 is safe and effective in treating patients with liver cancers and solid tumors with liver metastatic disease.

Detailed 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.

Conditions

• Advanced Cholangiocarcinoma
• Advanced Hepatocellular Carcinoma
• Advanced Malignant Solid Neoplasm
• Metastatic Malignant Neoplasm in the Liver
• Refractory Malignant Solid Neoplasm
• Stage III Hepatocellular Carcinoma AJCC v8
• Stage IV Hepatocellular Carcinoma AJCC v8

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo collection of blood samples
PROCEDURE	Computed Tomography	Undergo CT scan
PROCEDURE	Magnetic Resonance Imaging	Undergo MRI
DRUG	RNA Transcription Modulator AU-409	Given PO

Timeline

• Start date: 2023-03-29
• Primary completion: 2026-03-29
• Completion: 2027-03-29
• First posted: 2023-03-30
• Last updated: 2026-02-02

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study