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Hepatocellular CarcinomaPhase 2NCT03785210

Nivolumab (Anti-PD1), Tadalafil and Oral Vancomycin in People With Refractory Primary Hepatocellular Carcinoma or Liver Dominant Metastatic Cancer From Colorectal or Pancreatic Cancers

A Phase 2 study testing nivolumab, tadalafil and oral vancomycin in people with Hepatocellular Carcinoma and Hepatocellular Cancer, run by National Cancer Institute (NCI) at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

Background: A most common liver cancer in adults is hepatocellular carcinoma. Other kinds of liver cancer happen when colorectal or pancreatic cancer spreads to the liver. Researchers want to study if a combination of drugs helps people with these cancers. The drugs are nivolumab, tadalafil, and vancomycin. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
nivolumab, tadalafil and oral vancomycin
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
1 siteUnited States
Study dates
5 Jun 2019to 31 Dec 2022
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have
  • histopathological confirmation of Hepatocellular Carcinoma (HCC) (Cohort 1) OR
  • histopathological confirmation of carcinoma by in the setting of clinical and radiological characteristics which, together with the pathology, are highly suggestive of a diagnosis of HCC (Cohort 1) OR
  • histopathological confirmation of advanced colorectal or pancreatic malignancy with liver involvement as dominant site of metastasis (Per multidiscipline tumor board review and approval) (Cohort 2).
  • Patients must have disease that is not amenable to potentially curative resection, transplantation or ablation.
  • Patients must have progressed on, been intolerant to, or refused prior sorafenib/lenvatinib and/or atezolizumab/bevacizumab therapy (Cohort 1 only).
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  • Subjects must have progressed on or after standard systemic chemotherapy (at least one line of chemotherapy for patients with liver metastasis from pancreatic ductal adenocarcinoma (PDAC), at least two lines of chemotherapy for patients with liver metastasis from colorectal cancer (CRC) (Cohort 2 only).
  • Patients must have evaluable or measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
  • Patients must have lesion accessible for biopsy and be willing to undergo pre- and posttreatment biopsies.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1
  • If liver cirrhosis is present, patient must have a Child-Pugh score less than or equal to 7
  • Active chronic hepatitis B virus (HBV) infected subjects must be on antivirals and have HBV deoxyribonucleic acid (DNA) <100IU/mL. hepatitis C virus (HCV) infected subjects can be enrolled with close HCV RNA level monitoring.
  • Age greater than or equal to 18 years. Because no dosing or adverse event data are currently available on the use of nivolumab in combination with tadalafil and vancomycin in patients less than 18 years of age, children are excluded from this study, but will be eligible for future pediatric trials
  • Adequate hematological function defined by:
  • white blood cell (WBC) count greater than or equal to 3x10^9/L
  • absolute neutrophil count (ANC) greater than or equal to 1.5x10^9/L,
  • lymphocyte count greater than or equal to 0.5x10^9/L,
  • platelet count greater than or equal to 60x10^9/L, and
  • Hemoglobin (Hgb) greater than or equal to 9 g/ dL (more than 48 hours post-completion of blood transfusion)
  • Adequate hepatic function defined by:
  • a total bilirubin level less than or equal to 1.5 X Upper limit of normal (ULN),
  • an aspartate transaminase (AST) level <5xULN,
  • an alanine aminotransferase (ALT) level <5xULN.
  • Adequate renal function defined by:
  • Creatinine clearance (CrCl) greater than or equal to 50 mL/min/1.73 m^2 by 24 hours urine collection or as predicted by the Cockcroft-Gault formula:
  • CrCl = (140 age (y)) x (weight in kg) x (0.85, if female) x1.73 m^2/72 x Serum Creatinine (mg/dL) x patient's body surface area (BSA) (m^2) Or
  • The effects of nivolumab on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation and up to 5 months (women) and 7 months (men) after the last dose of the drug. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
  • Patients with a history of cardiovascular disease may be enrolled per cardiology consultation and approval with echocardiogram and troponin level in normal range at the time of enrollment.
  • Patient must be able to understand and willing to sign a written informed consent document.

Who cannot join

  • Patients who have had standard-of-care anti-cancer therapy or therapy with investigational agents (e.g., chemotherapy, immunotherapy, endocrine therapy, targeted therapy, biologic therapy, tumor embolization, monoclonal antibodies or other investigation agents), large field radiotherapy, or major surgery within 4 weeks prior to enrollment.
  • Therapy with antibiotics within 30 days prior to enrollment.
  • Therapy with nitrates, alpha-blockers, or cytochrome P450 (CYP3A4) inhibitors within 7- days prior to enrollment and for whom stopping is unsafe and/or a safe substitute is not medically recommended. Use of PDE5 inhibitors such as vardenafil (Levitra), tadalafil (Cialis), and sildenafil citrate (Viagra) less than or equal to 15-days prior to enrollment.
  • The patient must not be currently on a corticosteroid dose greater than physiologic replacement dosing defined as 10 mg of cortisone per day or its equivalent.
  • For PDAC patients with liver metastases, primary PDAC has not been resected (unless the primary is in the tail of the pancreas).
  • Patients with known brain metastases will be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events.
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  • Have signs of liver failure, e.g., clinical significant ascites, encephalopathy, or variceal bleeding within 6 months prior to enrollment.
  • Prior major liver resection: remnant liver <50% of the initial liver volume. Patients with a biliary stent can be included.
  • Patients with active autoimmune disease or history of autoimmune disease that might recur, which may affect vital organ function or require immune suppressive treatment including systemic corticosteroids. These include but are not limited to patients with a history of immune related neurologic disease, multiple sclerosis, autoimmune (demyelinating) neuropathy, Guillain-Barre syndrome or Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), myasthenia gravis; systemic autoimmune disease such as Systemic lupus erythematosus (SLE), connective tissue diseases, scleroderma, inflammatory bowel disease (IBD), Crohn's, ulcerative colitis, hepatitis; and patients with a history of toxic epidermal necrolysis (TEN), Stevens-Johnson syndrome, or phospholipid syndrome. Such diseases should be excluded because of the risk of recurrence or exacerbation of disease.
  • Of note, patients with vitiligo, endocrine deficiencies including thyroiditis managed with replacement hormones including physiologic corticosteroids are eligible. Patients with rheumatoid arthritis and other arthropathies, Sjogren's syndrome and psoriasis controlled with topical medication and patients with positive serology, such as antinuclear antibodies (ANA), anti-thyroid antibodies should be evaluated for the presence of target organ involvement and potential need for systemic treatment but should otherwise be eligible.
  • Have history of idiopathic pulmonary fibrosis (including bronchiolitis obliterans with organizing pneumonia) or evidence of active pneumonitis on screening chest computed tomography (CT) scan.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Patients with myocardial infarction or myocarditis within 12 months prior to enrollment.
  • History of severe or unstable cerebrovascular disease.
  • Sustained hypotension (<90/50 mmHg) or uncontrolled hypertension (>160/100 mmHg)
  • Stroke within 6 months prior to enrollment.
  • Human immunodeficiency virus (HIV)-positive patients are excluded because HIV causes complicated immune deficiency and study treatment can possess more risks for these patients.
  • Have had prior transplant of any kind.
  • Have ascites.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to nivolumab, tadalafil or vancomycin.
  • History of severe hypersensitivity reaction to any monoclonal antibody.
  • Prior invasive malignancy (except non-melanomatous skin cancer) unless disease free for a minimum of 3 years prior to enrollment.
  • Pregnant women are excluded from this study because nivolumabs potential for teratogenic or abortifacient effects is unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with nivolumab, breastfeeding should be discontinued if the mother is treated with nivolumab

Trial locations and contacts

1 site in 1 country.

United States1 site
  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Phase II Study of Nivolumab (Anti-PD1), Tadalafil and Oral Vancomycin in Patients With Refractory Primary Hepatocellular Carcinoma or Liver Dominant Metastatic Cancer From Colorectal or Pancreatic Cancers

Background:

A most common liver cancer in adults is hepatocellular carcinoma. Other kinds of liver cancer happen when colorectal or pancreatic cancer spreads to the liver. Researchers want to study if a combination of drugs helps people with these cancers. The drugs are nivolumab, tadalafil, and vancomycin.

Read more

Objective:

To investigate if nivolumab given with tadalafil and vancomycin causes liver cancer to shrink.

Eligibility:

Adults ages 18 years and older with hepatocellular carcinoma or metastases to the liver from colorectal or pancreatic cancer for which standard treatment has not worked

Design:

Participants will be screened with:

Medical and cancer history

Review of symptoms and ability to perform normal activities

Physical exam

Heart test. Some participants may meet with a cardiologist and/or have another heart test.

Scan of the chest, abdomen, and pelvis

Blood and urine tests

Tumor sample review. This can be from a previous procedure.

Participants will receive the study drugs in 4-week cycles. In each cycle participants will:

Get nivolumab through a small plastic tube in the arm on Day 1.

Take tadalafil by mouth 1 time every day.

Take vancomycin by mouth 4 times a day. They will take it every day for weeks 1 3, then not take it for week 4.

Complete a medicine diary of dates, times, missed doses and symptoms.

Throughout the study, participants will repeat screening tests and will give stool samples or rectal swabs.

After their last cycle, participants will have 3 follow-up visits over 3 months. Then they will be contacted every 6 months by phone or email and asked about their general well-being.

...

Read the detailed description

Background:

  • Current treatment options for patients with liver cancers, including hepatocellular carcinoma (HCC) and advanced liver cancers are limited and take no account of the known biological and genetic heterogeneity in these diseases. Median survival for advanced disease remains poor at approximately 1 year.
  • Nivolumab is a fully human monoclonal immunoglobulin G4 (IgG4) antibody that is specific for human programmed death-1 (PD-1, cluster of differentiation 279 [CD279]) cell surface membrane receptor. Nivolumab has been approved by the Food and Drug Administration (FDA) for the treatment of HCC and other solid tumors.
  • Tadalafil is a phosphodiesterase type 5 (PDE5) inhibitor which have been approved by the FDA for the treatment of pulmonary arterial hypertension, benign prostatic hyperplasia and erectile dysfunction, with a relative safe clinical profile. PDE5 inhibitors have been examined in multiple malignancies and cancer cell lines for their direct anticancer activities, for their efficacy as chemo-sensitizers and for cancer chemoprevention.
  • Oral vancomycin is antibiotic that has effect on altering gut commensal bacteria subsequently inducing a liver-selective anti-tumor effect.
  • The aim of the study is to evaluate whether the immunomodulatory effect induced by PDE5 inhibitor and oral vancomycin can be enhanced by immune checkpoint inhibition in advanced liver cancer.

Objective:

-To determine the Best Overall Response (BOR) according to Response Evaluation Criteria (RECIST 1.1) to combined treatment of nivolumab, oral vancomycin and tadalafil in patients with refractory primary HCC or liver dominant metastatic cancer from colorectal cancer (CRC) or pancreatic adenocarcinoma (PDAC).

Eligibility:

  • Histologically confirmed, hepatocellular carcinoma (HCC) Or
  • Histologically confirmed carcinoma highly suggestive of a diagnosis of HCC Or
  • Histologically confirmed advanced colorectal or pancreatic malignancy with liver involvement as dominant site of metastasis
  • Measurable lesion, accessible for biopsy.
  • Age greater than or equal to 18 years
  • ECOG less than or equal to 1
  • Acceptable renal, bone marrow and liver function.
  • Willingness to undergo two mandatory tumor biopsies.

Design:

  • The proposed study is a phase II study of combined nivolumab, oral vancomycin and tadalafil treatment in patients with HCC or liver dominant metastatic cancer from colorectal or pancreatic cancers.
  • Treatment will be delivered in cycles consisting of 4 weeks (+/- 3 days) until progression or unacceptable toxicity.
  • Patients will be seen in Clinical Center on monthly basis with disease status evaluation every 8 (+/-1) weeks after start of study therapy.
Being tested
nivolumab, tadalafil, oral vancomycin
Keywords
Anticancer Activities, Altering Gut Commensal Bacteria, Immunomdulatory Effect, Checkpoint Inhibition
Medical subject headings
Carcinoma, Hepatocellular, Liver Neoplasms, Pancreatic Neoplasms, Colorectal Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • nivolumabDrug

    Also called Opdivo

    480 mg intravenous (IV) every 4 weeks

  • tadalafilDrug

    Also called Cialis

    10 mg by mouth (PO) daily

  • oral vancomycinDrug

    Also called Vancocin

    125 mg every 6-hour (500 mg total daily dose) by mouth (PO) from week 1 to week 3, week 4 off.

Study groups in NCT03785210
Study groupRoleWhat they receive
1/Arm 1 - Nivolumab, Tadalafil and Oral VancomycinExperimentalNivolumab, tadalafil and oral vancomycin
Receives: Drug: nivolumab; Drug: tadalafil; Drug: oral vancomycin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Best Overall Response (BOR)

    Best overall response is defined as the best response recorded from the start of the treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started) assessed by the Response Evaluation Criteria in Solid Tumors (RECIST)v1.1. Complete Response (CR) is disappearance of all target lesions. Partial Response (PR) is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). The appearance of one or more new lesions is also considered progressions). Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study.

    Measured: Every 2 months and then every 6 months after discontinuation of treatment, for survival purposes, up to 3 years

Secondary outcomes (2)
  1. Serious Adverse Events Possibly, Probably, and/or Definitely Related to Treatment

    Adverse events were assessed by the by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

    Measured: From first study treatment, approximately 90 days

  2. Overall Survival (OS) of Nivolumab Combined With Oral Vancomycin and Tadalafil in Participants With Refractory Hepatocellular Carcinoma (HCC) or Liver Dominant Metastatic Cancer From Colorectal Cancer or Pancreatic Ductal Adenocarcinoma (PDAC)

    Overall survival is calculated from the on-study date to the day of death using the Kaplan-Meier method.

    Measured: Participants were followed from the on-study date to post discontinuation of treatment until the day of death, an average of 8 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Dec 2018
  2. Study start5 Jun 2019
  3. Primary completion31 Oct 2022
  4. Study completion31 Dec 2022

Why it stopped: Study was terminated due to poor accrual.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03785210
Sponsor's protocol ID
190033
Other IDs
19-C-0033
Status last verified
Feb 2026
First submitted
20 Dec 2018
Last updated
23 Feb 2026
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request. In addition, all large-scale genomic sequencing data will be shared with subscribers to the Database of Genotypes and Phenotypes (dbGaP).

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing nivolumab, tadalafil and oral vancomycin in people with Hepatocellular Carcinoma and Hepatocellular Cancer, run by National Cancer Institute (NCI) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 23 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Best Overall Response (BOR) (time frame: Every 2 months and then every 6 months after discontinuation of treatment, for survival purposes, up to 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2026.

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