What side effects are associated with this type of intervention?

Common treatments for cholangiocarcinoma, such as gemcitabine combined with cisplatin or oxaliplatin, and targeted therapies like bevacizumab, often result in side effects including fatigue, nausea, vomiting, diarrhea, and hematologic toxicities like neutropenia and thrombocytopenia. Targeted therapies may also cause skin rash, hypertension, and proteinuria. These side effects are generally manageable with supportive care and dose adjustments.

What prior FDA approvals exist?

The FDA has approved several treatments for cholangiocarcinoma that focus on inhibiting cancer cell growth and metastasis. Notably, gemcitabine combined with cisplatin is a commonly used regimen. Additionally, durvalumab, an immunotherapy drug, has been combined with gemcitabine and cisplatin for advanced biliary tract cancers, showing potential benefits. These treatments aim to halt the progression and spread of cancer, similar to the investigational drug AU409.

What other types of research exist?

Promising novel alternatives to AU409 for cholangiocarcinoma patients include: 1) Durvalumab plus gemcitabine and cisplatin, which has shown efficacy in advanced biliary tract cancer. 2) Gemcitabine combined with oxaliplatin (GEMOX), which has been studied in advanced biliary tract adenocarcinoma. These treatments have demonstrated potential in clinical trials and may be considered for patients in 2024.

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AU409 for Liver Cancer

Phase 1

Recruiting

Led By Anthony B El-Khoueiry, MD

Research Sponsored by University of Southern California

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests AU409, a new drug, in patients with advanced liver cancers or solid tumors that have spread to the liver. AU409 aims to stop cancer cells from growing and spreading. Researchers are checking if it is safe and effective.

Who is the study for?

This trial is for adults with advanced primary liver cancers or solid tumors that have spread to the liver. Participants must have tried all standard treatments, be in good physical condition (ECOG 0-1), and not be pregnant or breastfeeding. They should also meet specific blood count and organ function criteria, agree to use contraception, and not have certain medical conditions like active infections or recent heart issues.

What is being tested?

The trial tests AU409's safety, side effects, and optimal dosage on patients with advanced liver cancer or those whose solid tumors have metastasized to the liver. It aims to find out if AU409 can halt cancer growth and spread by collecting biospecimens and using imaging techniques like CT scans and MRIs.

What are the potential side effects?

While specific side effects of AU409 are being studied in this trial, common ones may include reactions at the infusion site, fatigue, nausea, changes in blood counts leading to increased infection risk or bleeding problems. Organ-specific inflammation could also occur.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of adverse events

Maximum Tolerated Dose

Recommended phase II dose

Secondary study objectives

Objective radiologic response

Pharmacokinetics evaluation - Peak plasma concentration (Cmax)

Pharmacokinetics evaluation - Peak time (Tmax)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (AU409)Experimental Treatment4 Interventions

Patients receive AU409 PO on study. Patients also undergo CT or MRI and collection of blood samples throughout the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Biospecimen Collection

2004

Completed Phase 3

~2020

Computed Tomography

2017

Completed Phase 2

~2740

Magnetic Resonance Imaging

2017

Completed Phase 3

~1160

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Cholangiocarcinoma treatments often target specific pathways to inhibit cancer cell growth and metastasis. Targeted therapies, like pemigatinib, inhibit fibroblast growth factor receptor (FGFR) alterations, which are common in cholangiocarcinoma, thereby blocking cancer cell proliferation. Chemotherapy regimens, such as gemcitabine combined with cisplatin, disrupt DNA replication and repair, leading to cancer cell death. Immunotherapies, like pembrolizumab, enhance the immune system's ability to recognize and destroy cancer cells by targeting immune checkpoints. These mechanisms are crucial for cholangiocarcinoma patients as they offer tailored approaches to halt disease progression and improve survival outcomes.