Simvastatin in Preventing Liver Cancer in Patients With Liver Cirrhosis

NCT ID: NCT02968810

Last Updated: 2025-09-17

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

52 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-06-21

Study Completion Date

2025-12-23

Brief Summary

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This phase II trial studies how well simvastatin works in preventing liver cancer in patients with liver cirrhosis. Simvastatin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Detailed Description

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PRIMARY OBJECTIVE:

I. To evaluate the effect of a simvastatin intervention versus placebo on the change in serum AFP-L3% from baseline to 6 months following treatment initiation in patients with liver cirrhosis who have a current model for end-stage liver disease (MELD) =\< 20.

SECONDARY OBJECTIVES:

I. To evaluate the effect of a simvastatin intervention versus placebo at 6 months from baseline on the change in:

Ia. Serum AFP; Ib. Serum IL-6; Ic. Serum deoxycholic acid; Id. Liver stiffness; Ie. Fibrosis 4 index (FIB-4) score; If. MELD score.

EXPLORATORY OBJECTIVES:

I. To evaluate the effect of a simvastatin intervention versus placebo at 6 months from baseline on the change in other:

Ia. serum bile acid levels; Ib. serum immune markers.

OUTLINE: Patients are randomized to 1 of 2 groups.

GROUP I: Patients receive simvastatin orally (PO) once daily (QD). Patients also undergo collection of blood on study and computed tomography (CT) scans/magnetic resonance imaging (MRI) throughout the trial.

GROUP II: Patients receive placebo PO QD. Patients also undergo collection of blood on study and CT/MRI throughout the trial.

In both groups, treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30, 60, and 90 days.

Conditions

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Cirrhosis Hepatocellular Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Group I (simvastatin)

Patients receive simvastatin PO QD. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity. Patients also undergo collection of blood on study and CT/MRI throughout the trial.

Group Type EXPERIMENTAL

Biospecimen Collection

Intervention Type PROCEDURE

Undergo collection of blood

Computed Tomography

Intervention Type PROCEDURE

Undergo CT

Magnetic Resonance Imaging

Intervention Type PROCEDURE

Undergo MRI

Questionnaire Administration

Intervention Type OTHER

Ancillary studies

Simvastatin

Intervention Type DRUG

Given PO

Group II (placebo)

Patients receive placebo PO QD. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity. Patients also undergo collection of blood on study and CT/MRI throughout the trial.

Group Type PLACEBO_COMPARATOR

Biospecimen Collection

Intervention Type PROCEDURE

Undergo collection of blood

Computed Tomography

Intervention Type PROCEDURE

Undergo CT

Magnetic Resonance Imaging

Intervention Type PROCEDURE

Undergo MRI

Placebo Administration

Intervention Type OTHER

Given PO

Questionnaire Administration

Intervention Type OTHER

Ancillary studies

Interventions

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Biospecimen Collection

Undergo collection of blood

Intervention Type PROCEDURE

Computed Tomography

Undergo CT

Intervention Type PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

Intervention Type PROCEDURE

Placebo Administration

Given PO

Intervention Type OTHER

Questionnaire Administration

Ancillary studies

Intervention Type OTHER

Simvastatin

Given PO

Intervention Type DRUG

Other Intervention Names

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Biological Sample Collection Biospecimen Collected Specimen Collection CAT CAT Scan Computed Axial Tomography Computerized Axial Tomography Computerized axial tomography (procedure) Computerized Tomography Computerized Tomography (CT) scan CT CT Scan Diagnostic CAT Scan Diagnostic CAT Scan Service Type tomography Magnetic Resonance Magnetic Resonance Imaging (MRI) Magnetic resonance imaging (procedure) Magnetic Resonance Imaging Scan Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance MR MR Imaging MRI MRI Scan MRIs NMR Imaging NMRI Nuclear Magnetic Resonance Imaging sMRI Structural MRI MK 733 Synvinolin Zocor

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of liver cirrhosis assessed by the presence of clinical signs, symptoms, body imaging (ultrasound, computed tomography \[CT\], or magnetic resonance imaging \[MRI\]), or liver biopsy
* Age \>= 18 years
* Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (Karnofsky \>= 70%)
* Leukocytes \>= 2,500/microliter
* Absolute neutrophil count \>= 1,500/microliter
* Platelets \>= 50,000/microliter
* Hemoglobin \>= 8 g/dL
* Total bilirubin =\< 3 x institutional upper limit of normal (ULN)
* Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 5 x institutional ULN
* Creatinine =\< 1.5 x institutional ULN
* Women who are able to become pregnant must have a confirmed negative pregnancy test result prior to enrollment; women \>= 50 years of age who have not had a menstrual period in the past year; and women who have had a hysterectomy, both ovaries removed, or a tubal ligation; will not be required to have a pregnancy test
* The effects of simvastatin on the developing human fetus at the recommended therapeutic dose are unknown; for this reason, women who are able to become pregnant 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; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her study physician immediately
* Ability to understand and the willingness to sign a written informed consent document and medical release
* Willing and able to comply with trial protocol and follow-up
* Have had an abdominal imaging test (CT, MRI, or ultrasound) within the past 18 months

* 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
* History of chronic myopathy
* Prior germ cell cancer
* History of active malignancy within the past 5 years (excluding basal/squamous cell skin cancer or prostate cancer with a Gleason score 6 or less)
* Known active infection with HIV
* Medical contraindications to blood draw (e.g., hemophilia)
* Concurrent illness which in the opinion of the investigators would compromise either the patient or the integrity of the data
* Current excessive alcohol consumption (average alcohol consumption of more than 5 drinks per day)

Exclusion Criteria

* Prior or current use of statin medication
* Current systemic use of medications known to interact with statins and potentially increase toxicity, including gemfibrozil, cyclosporine, danazol, lomitapide, verapamil, diltiazem, dronedarone, amiodarone, amlodipine, ranolazine, strong CYP3A4 inhibitors (e.g., itraconazole, ketoconazole, posaconazole, voriconazole, human immunodeficiency virus \[HIV\] protease inhibitors, boceprevir, telaprevir, erythromycin, clarithromycin, telithromycin, nefazodone, or cobicistat-containing products), or strong CYP3A4 inducers (e.g., carbamazepine, phenytoin, rifampin, St. John's wort, bosentan, efavirenz, etravirine, modafinil, nafcillin)
* History of adverse effects, intolerance, or allergic reactions attributed to compounds of similar chemical or biologic composition to simvastatin (i.e., other statin medications)
* Current use of any other investigational agents
* Women who are pregnant or breastfeeding; pregnant women are excluded from this study because simvastatin is a lipid-lowering agent with the potential for teratogenic or abortifacient effects; it is not known whether simvastatin is excreted into human milk; however, a small amount of another drug in this class does pass into breast milk; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with simvastatin, breastfeeding should be discontinued if the mother is treated with simvastatin
* Prior liver transplant
* Prior known or suspected hepatocellular carcinoma
* Prior cholangiocarcinoma
* Model for end-stage liver disease (MELD) \> 20
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc T Goodman

Role: PRINCIPAL_INVESTIGATOR

Northwestern University

Locations

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Cedars Sinai Medical Center

Los Angeles, California, United States

Site Status

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Centro Comprensivo de Cancer de UPR

San Juan, , Puerto Rico

Site Status

University of Puerto Rico

San Juan, , Puerto Rico

Site Status

Countries

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United States Puerto Rico

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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NCI-2016-01719

Identifier Type: REGISTRY

Identifier Source: secondary_id

N01-CN-2012-00035

Identifier Type: -

Identifier Source: secondary_id

NCI2015-06-03

Identifier Type: OTHER

Identifier Source: secondary_id

NWU2015-06-03

Identifier Type: OTHER

Identifier Source: secondary_id

N01CN00035

Identifier Type: NIH

Identifier Source: secondary_id

View Link

P30CA060553

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NCI-2016-01719

Identifier Type: -

Identifier Source: org_study_id

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