Actively Recruiting
First in Human Dose Escalation Study of AU409 in Patients With Advanced Primary Liver Cancers or Advanced Solid Tumor With Liver Predominant Metastatic Disease
Led by University of Southern California · Updated on 2026-06-01
36
Participants Needed
2
Research Sites
52 weeks
Total Duration
On this page
Sponsors
U
University of Southern California
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating AU409, an RNA transcription modulator, in a phase I trial focused on patients with advanced primary liver cancers or advanced solid tumors that have spread predominantly to the liver. This study aims to determine the safety, side effects, maximum tolerated dose, and recommended phase II dose of AU409. The trial also seeks to assess the drug's preliminary anti-tumor activity and pharmacokinetics in these patients. Participants receive AU409 orally, with dosing guided by a dose-escalation design to find the best dose. During the trial, patients undergo scans such as computed tomography (CT) or magnetic resonance imaging (MRI) and have blood samples collected regularly. A subset of patients at higher dose levels will provide liver biopsy samples to study drug concentration and gene expression. The trial includes a dose-escalation period and may have a dose-expansion phase depending on findings. Throughout the study, researchers monitor safety by evaluating adverse events and measure tumor response using radiologic criteria. Pharmacokinetic data are collected at specific times during treatment cycles. The study monitors participants for up to 30 days after treatment ends or until death for safety, with tumor response followed for up to three years. Total participation duration varies by individual patient progress and treatment response.
CONDITIONS
Brief Title
AU409 for the Treatment of Advanced Primary Liver Cancers or Solid Tumor With Liver Metastatic Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age �3e= 18 years old
- Histopathologically or cytologically confirmed advanced solid tumor refractory to or lacking standard therapies
- Primary liver malignancy (hepatocellular carcinoma or cholangiocarcinoma) or solid tumor with liver dominant disease with no more than two extrahepatic sites
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Recovery from previous treatments with toxic effects �3c= grade 1 (except alopecia), residual neuropathy �3c= grade 2 allowed
- Prior chemotherapy, radiotherapy, targeted or immunotherapy completed at least 4 weeks or five half-lives before AU409 start
- Estimated life expectancy of at least 3 months
- Agreement to use adequate contraception during and after study participation for men and women of child-bearing potential
- Agreement to liver biopsy for selected patients and blood sample collection
- Adequate blood counts: ANC �3e= 1500/mm�5e3, platelets �3e= 100,000/mm�5e3, hemoglobin �3e= 8 g/dL
- For HCC patients with splenic sequestration: ANC �3e= 1000/mm�5e3, platelets �3e= 70,000
- Kidney function with calculated clearance �3e= 60 mL/min/1.73 m�b2
- Liver function within specified limits for bilirubin, AST/ALT, and coagulation parameters
You will not qualify if you...
- Hypersensitivity to pentamidine or AU409 excipients
- Anticancer or investigational therapy within 28 days prior to study entry (except palliative radiation completed �3e= 14 days prior)
- Hepatocellular carcinoma patients with Child Pugh score �3e= B7
- Untreated or symptomatic central nervous system metastases; treated brain metastases allowed if asymptomatic and off steroids
- History of serious cardiac or neurological events within 6 months prior to treatment
- Corrected QT interval > 470 msec on ECG
- Use of warfarin anticoagulation; low molecular weight heparins or Factor Xa inhibitors allowed
- History of gastrointestinal surgery or malabsorption affecting drug absorption
- Active hepatitis B (unless on antiviral therapy with low viral load) or active hepatitis C
- Active infections requiring treatment, including HIV
- Immune compromise from medications or conditions
- Any condition interfering with informed consent or study participation
- Pregnant or nursing women
- Use of strong cytochrome P450 enzyme inducers or inhibitors unless discontinued or replaced before study start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation.
Participants receive AU409 orally and undergo CT or MRI scans and blood sample collections throughout the treatment period to assess safety, dosing, and response.
Multiple visits for imaging and blood sample collection during each cycle
Duration - Up to 30 days after removal from treatment or until death, whichever occurs first
Participants are monitored for adverse events and overall health after treatment ends.
Approximately 1 to 2 visits
Trial Site Locations
Total: 2 locations
1
Los Angeles County-USC Medical Center
Los Angeles, California, United States, 90033
Actively Recruiting
2
USC / Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
Actively Recruiting
Research Team
X
Xiomara Menendez, RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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