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New treatment option for Advanced Cholangiocarcinoma
Official title AU409 for the Treatment of Advanced Primary Liver Cancers or Solid Tumor With Liver Metastatic Disease
ClinicalTrials.gov ID: NCT05791448
What this study is testing
What is RNA Transcription Modulator AU-409?
RNA Transcription Modulator AU-409 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for advanced cholangiocarcinoma.
Also referred to as AU 409, AU-409.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age \>= 18 years old
- Patients must have histopathologically /cytologically confirmed advanced solid tumor, which is refractory to standard therapeutic options, or for...
- During the dose-escalation portion, patients must have primary liver malignancy (including hepatocellular carcinoma or cholangiocarcinoma) OR a solid...
- Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Patient must have recovered from any toxic effects of previous chemotherapy, targeted therapy or radiotherapy as judged by the Investigator to =\<...
You likely can't join if
- Patients who have had hypersensitivity to pentamidine or any excipients of AU409
- Treatment with other anticancer therapies (including surgery, radiation therapy, chemotherapy, anti-angiogenic therapy, targeted therapy, or...
- Hepatocellular carcinoma patients with a Child Pugh score \>= B7
- Patients with known central nervous system metastases which are untreated or symptomatic; patients with treated brain metastases (completed \>= 30...
- Patient with a history of the following within 6 months prior to cycle 1 day 1: a myocardial infarction, severe/unstable angina pectoris...
- Patients who have corrected QT (QTc) interval to \> 470 msec (Fredericia's equation) on 2 out of 3 electrocardiogram (ECG)'s (if first ECG has QTc \...
See the full eligibility criteria
Who can join
- Age \>= 18 years old
- Patients must have histopathologically /cytologically confirmed advanced solid tumor, which is refractory to standard therapeutic options, or for which there are no standard therapeutic options. Failure of all approved...
- During the dose-escalation portion, patients must have primary liver malignancy (including hepatocellular carcinoma or cholangiocarcinoma) OR a solid tumor with liver dominant disease; liver dominant disease is defined...
- Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Patient must have recovered from any toxic effects of previous chemotherapy, targeted therapy or radiotherapy as judged by the Investigator to =\< grade 1 (except for alopecia). Residual sensory neuropathy =\< grade 2...
- Previous chemotherapy/radiotherapy/targeted/immunotherapy therapy should have been completed at least 4 weeks prior to start of AU409 administration, or five half-lives, whichever is shorter (except for palliative...
- Patients must have an estimated life expectancy of at least 3 months
- Women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation. A male...
- Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
- A female of child-bearing potential is any woman (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: Has not undergone a hysterectomy...
- Patients must agree, as part of the informed consent, to undergo liver biopsy (for a subset of patients enrolled at and above dose level 4) and to provide blood for pharmacokinetics analysis
- Absolute neutrophil count (ANC) \>= 1500/mm\^3
- Platelet count \>= 100,000/mm\^3
- Hemoglobin \>= 8 g/dL (prior transfusion is allowed if completed 2 weeks prior to screening and hemoglobin remains \>= 8 g/dL)
- For patients with HCC with splenic sequestration: ANC \>= 1000/mm\^3
- For patients with HCC with splenic sequestration: Platelets \>= 70,000
- Calculated clearance \>= 60 mL/min/1.73 m\^2. Actual body weight should be used for calculating creatinine clearance (e.g., using the Cockroft-Gault formula). For people with a Body Mass Index (BMI) \> 30 kg/m\^2, lean...
- Total bilirubin =\< 1.5 X upper limit of normal (ULN) (people with known Gilbert's hepatic function disease can have bilirubin of up to 2 X ULN)
- Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =\< 3 X ULN; or AST/ALT =\< 5 X ULN if patient has liver tumors
- Prothrombin time (PT)/international normalized ratio (INR) =\< 1.8 times upper limit of normal (unless patient is on anticoagulation)
What rules you out
- Patients who have had hypersensitivity to pentamidine or any excipients of AU409
- Treatment with other anticancer therapies (including surgery, radiation therapy, chemotherapy, anti-angiogenic therapy, targeted therapy, or radiofrequency ablation therapy, etc.) or investigational therapy within 28...
- Hepatocellular carcinoma patients with a Child Pugh score \>= B7
- Patients with known central nervous system metastases which are untreated or symptomatic; patients with treated brain metastases (completed \>= 30 days prior to screening) are allowed provided they are asymptomatic and...
- Patient with a history of the following within 6 months prior to cycle 1 day 1: a myocardial infarction, severe/unstable angina pectoris, coronary/peripheral artery bypass graft, New York Heart Association (NYHA) Class...
- Patients who have corrected QT (QTc) interval to \> 470 msec (Fredericia's equation) on 2 out of 3 electrocardiogram (ECG)'s (if first ECG has QTc \ 470 repeat twice for a total of 3 ECG's)
- Patients who are on therapeutic anticoagulation with warfarin; however, patients on therapeutic doses of with low molecular weight heparins or Factor Xa inhibitors are eligible
- Patient with history of gastrointestinal surgery or malabsorptive conditions that may change the absorption of drugs and/or cause rapid transit (such as total gastrectomy, small bowel resection, etc.)
- Patients who have known active hepatitis B. Patients with chronic hepatitis B who are on anti-viral therapy and have a hepatitis B viral load of =\< 500 IU/mL are allowed on the study. Patients with chronic Hepatitis C...
- Patients who have active infection requiring treatment (except hepatitis B and C as noted above) including known human immunodeficiency virus (HIV) infection
- Patients who have concurrent conditions resulting in immune compromise, including chronic treatment with corticosteroids or other immunosuppressive agents
- Patients who have any other condition, including mental illness or substance abuse, deemed by the Investigator to be likely to interfere with a patient's ability to sign informed consent, cooperate and participate in...
- Patients must not be pregnant or nursing due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants
- Patients who are on medications that are considered to be strong inducers or inhibitors of the cytochrome P450 isoenzymes should have such medications discontinued or replaced. Such medications should be avoided for one...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.