Liver Cirrhosis Network Rosuvastatin Efficacy and Safety for Cirrhosis in the United States

NCT ID: NCT05832229

Last Updated: 2026-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

256 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-07

Study Completion Date

2029-08-31

Brief Summary

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This is a double-blind, phase 2 study to evaluate safety and efficacy of rosuvastatin in comparison to placebo after 2 years in patients with compensated cirrhosis.

Detailed Description

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This study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial, of 20mg (or 10mg for participants of East Asian ancestry) of rosuvastatin by mouth, once daily. Participants will be randomized (1:1) to either once daily placebo or once daily rosuvastatin.

Patients meeting all eligibility criteria will be assigned to a randomization arm prior to initiation of a 4-week lead-in phase of the study. All participants will undergo a 4-week, open-label active run-in phase to evaluate initial safety and adherence to rosuvastatin. During this active run-phase, all participants will receive target dose rosuvastatin-- 20 mg daily (10 mg daily for participants of East-Asian ancestry). After the active run-in phase, all participants will continue with their pre-assigned randomization (1:1) treatment of rosuvastatin 20 mg daily (10 mg daily for participants of East-Asian ancestry) or matching placebo.

The total duration of the study will be 96 weeks in the assigned treatment arm plus the 4-week lead-in period. The primary outcome will be the mean change in liver stiffness from the baseline measurement to the end of study liver stiffness, as measured by ultrasound-based vibration-controlled transient elastography (VCTE).

There are 10 participating clinical centers, and we anticipate a total of 256 patients will be recruited for the initial lead-in as we estimate 20% of participants may dropout after the lead-in (256 x 0.8 = 204 for randomization into the study drug treatment phase).

Conditions

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Cirrhosis Cirrhosis, Liver Cirrhosis Early Cirrhosis Due to Hepatitis B Cirrhosis Advanced Cirrhosis Infectious Cirrhosis Alcoholic Cirrhosis Due to Hepatitis C

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial, of 20mg (or 10mg for participants of East Asian ancestry) of rosuvastatin by mouth, once daily. Participants will be randomized (1:1) to either once daily placebo or once daily rosuvastatin.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
The drug manufacturer and bottler will be unblinded, as will a few members of the Scientific and Data Coordinating Center. All other study team members will remain blinded.

Study Groups

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Active

Open-label lead-in period of 4 weeks on 20 mg (10 mg for participants of East ancestry or on a protease inhibitor) rosuvastatin by mouth once daily, followed by a period of 96 weeks rosuvastatin 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor).

Group Type EXPERIMENTAL

Rosuvastatin

Intervention Type DRUG

Patients meeting all eligibility criteria will be assigned to a randomization arm prior to initiation of a 4-week lead-in phase of the study. All participants will undergo a 4-week, open-label active run-in phase to evaluate initial safety and adherence to rosuvastatin. During this active run-phase, all participants will receive target dose rosuvastatin-- 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor). After the active run-in phase, all participants will continue with their pre-assigned randomization (1:1) treatment of rosuvastatin 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor) or matching placebo.

Placebo

Open-label lead-in period of 4 weeks on 20 mg (10 mg for participants of East ancestry or on a protease inhibitor) rosuvastatin by mouth once daily, followed by a period of 96 weeks placebo.

Group Type PLACEBO_COMPARATOR

Rosuvastatin

Intervention Type DRUG

Patients meeting all eligibility criteria will be assigned to a randomization arm prior to initiation of a 4-week lead-in phase of the study. All participants will undergo a 4-week, open-label active run-in phase to evaluate initial safety and adherence to rosuvastatin. During this active run-phase, all participants will receive target dose rosuvastatin-- 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor). After the active run-in phase, all participants will continue with their pre-assigned randomization (1:1) treatment of rosuvastatin 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor) or matching placebo.

Interventions

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Rosuvastatin

Patients meeting all eligibility criteria will be assigned to a randomization arm prior to initiation of a 4-week lead-in phase of the study. All participants will undergo a 4-week, open-label active run-in phase to evaluate initial safety and adherence to rosuvastatin. During this active run-phase, all participants will receive target dose rosuvastatin-- 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor). After the active run-in phase, all participants will continue with their pre-assigned randomization (1:1) treatment of rosuvastatin 20 mg daily (10 mg daily for participants of East-Asian ancestry or on a protease inhibitor) or matching placebo.

Intervention Type DRUG

Other Intervention Names

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Rosuvastatin 20 mg Rosuvastatin 10 mg

Eligibility Criteria

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Inclusion Criteria

1. Age 18-75 years
2. Cirrhosis due to nonalcoholic steatohepatitis, alcohol-associated liver disease, or chronic viral hepatitis (treated hepatitis B virus or hepatitis C virus)
3. Clinical diagnosis of cirrhosis as defined investigator confirmation and the following:

1. At least one liver biopsy within 5 years prior to consent showing either: Metavir stage 4 fibrosis; Ishak Stage 5-6 fibrosis, OR
2. At least 2 of the following:

i. Evidence on imaging: Nodular liver with either splenomegaly or recanalized umbilical vein within the past 48 weeks ii. Liver stiffness: vibration-controlled transient elastography within 48 weeks prior to consent or during Screening ≥15 kilopascal or magnetic resonance elastography within 48 weeks prior to consent or during Screening ≥5 kilopascal iii. Evidence of varices demonstrated on imaging or endoscopy within 3 years prior to consent or during Screening iv. Either: Fibrosis-4\>2.67 or platelets \<150/mL within 6 months prior to consent or during Screening
4. Two measures of vibration-controlled transient elastography: one at screening and one at the randomization study visit, meeting the following criteria:

1. The first measure must be ≥ 15 kilopascal.
2. The two measures must be at least 2 hours apart and no more than 60 days apart from one another.
3. The mean of two measurements must be ≥ 15 kilopascal.
4. Additionally, both screening and open-label dispense liver stiffness measures must be ≤50 kPa
5. Compensated defined by:

1. Absence of ascites/hydrothorax, hepatic encephalopathy or variceal bleeding currently or in the last 48 weeks, as determined clinically by investigator.
2. If prior history of decompensation, must be without current symptoms of decompensation and no longer requiring treatment of complications for the last 48 weeks, including the use of diuretics for the treatment of ascites, and/or rifaximin or lactulose for the treatment of hepatic encephalopathy. Use of non-selective beta blockers will be allowed.
3. Child-Pugh score \<8
6. Provision of written informed consent.

Exclusion Criteria

1. Currently on a statin or any statin exposure within 24 weeks prior to consent.
2. Known indication for statin therapy, defined as:

1. Prior peripheral vascular, cardiovascular or cerebrovascular event for which statins are indicated for secondary prevention, OR
2. Documented familial hypercholesterolemia, heterozygous familial hypercholesterolemia, OR
3. Fasting LDL-C ≥ 190 mg/dL
3. Myocardial infarction, Unstable angina, transient ischemic events, or stroke within 24 weeks of screening.
4. Alcohol Use Disorder Identification Test (AUDIT) total score of ≥8 at screening.
5. Patients with limitations in attending study visits.
6. Prisoners.
7. Known prior or current hepatocellular carcinoma (HCC) or cholangiocarcinoma.
8. Known transjugular intrahepatic portosystemic shunt (TIPS), balloon retrograde transvenous obliteration (BRTO) or porto-systemic shunt surgery regardless of time of occurrence.
9. Current (in past 24 weeks prior to consenting) use of medications known to cause hepatic fibrogenesis or confound endpoint assessment, defined as:

1. amiodarone
2. methotrexate
3. warfarin
10. Current (in past 24 weeks prior to consenting) use of medications which may increase risk for rosuvastatin-related myositis or DILI, defined as:

1. fenofibrate
2. erythromycin
3. gemfibrozil
4. niacin (500 mg or more)
5. HIV protease inhibitors (darunivar, indinavir, nelfinavir, amprenavir) in patients of East Asian descent
6. colchicine
7. cyclosporin
8. Additional medications that will be excluded:

atazanavir/ritonavir capmatinib darolutamide dasabuvir/ombitasvir/paritaprevir/ritonavir ledipasvir/sofosbuvir elbasvir/grazoprevir erythromycin glecaprevir/pibrentasvir lopinavir/ritonavir regorafenib ritonavir, in any combination simeprevir sofbuvir/velpatasvir/voxilaprevir sofosbuvir/velpatasvir tafamidis teriflunomide

\*If exposure was for 7 or less days for one of these medications can consider enrollment after 28 days from final dose.
11. Presence of portal or hepatic vein thrombosis
12. Diagnosis of untreated hypothyroidism or on unstable treatment regimen for hypothyroidism
13. Receiving an elemental diet or parenteral nutrition
14. Chronic pancreatitis or pancreatic insufficiency
15. Etiology of cirrhosis other than ALD, NAFLD, or viral hepatitis (excluded diagnoses include cryptogenic immune-mediated such as AIH, PSC and PBC, cardiac cirrhosis or Fontan-associated liver disease, A1AT, Wilson's disease, etc.)
16. Conditions which may confound study outcome:

1. Unstable or active inflammatory bowel disease
2. Active infection
3. Any malignant disease (other than squamous or basal cell carcinoma of the skin) within previous 3 years
4. Prior solid organ or hematopoietic cell transplant
5. Bariatric surgery in the last 24 weeks prior to consent or planned bariatric surgery within the next 96 weeks
6. Current liver-unrelated end-stage organ failures such as end-stage renal disease on dialysis, stage 3-4 congestive heart failure (CHF), current chronic obstructive pulmonary disease (COPD) on home oxygen.
17. Known current medical or psychiatric conditions which, in the opinion of the investigator, would make the participant unsuitable for the study for safety reasons or interfere with or prevent adherence to the protocol.
18. The following laboratory abnormalities within 90 days of screening:

1. Hemoglobin \<10 g/dL
2. Albumin \<3.0 g/dL
3. Prolonged international normalized ratio (INR) \>1.5
4. Total bilirubin ≥ 2.0 mg/dl (unless due to Gilbert's syndrome or hemolysis as denoted by normal direct bilirubin fraction)
5. Direct bilirubin ≥ 0.9
6. Uncontrolled diabetes (HbA1c ≥ 9.5%) within past 90 days.
19. Kidney function abnormalities including:

1. Dialysis
2. Baseline eGFR \< 30 cc/min with CKD-Epi equation
3. Known nephrotic proteinuria, defined as 3g or greater of protein in 24-hour urine collection
20. Recent (within 48 weeks) or present hepatic decompensation with ascites/hydrothorax, hepatic encephalopathy or variceal bleeding
21. Untreated chronic hepatitis B or C infection

1. HCV eligible for enrollment if HCV RNA negative at baseline or documentation of prior SVR12
2. HBV eligible if an HBV DNA \<100 IU/mL within the last 48 weeks and on treatment
22. Serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥ 200 U/L, or alkaline phosphatase (ALP) ≥ 300 within the past 24 weeks.
23. Documented history of intolerance to statins
24. Serious comorbid medical disease which in the investigator's opinion renders a life-expectancy less than 96 weeks
25. Active illicit substance use (other than THC), including inhaled or injected drugs, in the 24 weeks prior to screening
26. Pregnancy, planned pregnancy or breastfeeding
27. Current participation in active medication treatment trials (within 24 weeks prior to randomization) or planned participation in active medication treatment trials simultaneous to participation in present trial.
28. Significant existing muscle pain or tenderness or prior history of myasthenia gravis as determined by a site physician.
29. Failure or inability to provide informed consent.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The Cleveland Clinic

OTHER

Sponsor Role collaborator

Columbia University

OTHER

Sponsor Role collaborator

Weill Medical College of Cornell University

OTHER

Sponsor Role collaborator

Duke University

OTHER

Sponsor Role collaborator

Mayo Clinic

OTHER

Sponsor Role collaborator

University of Miami

OTHER

Sponsor Role collaborator

University of Michigan

OTHER

Sponsor Role collaborator

University of California, San Diego

OTHER

Sponsor Role collaborator

University of California, San Francisco

OTHER

Sponsor Role collaborator

LAC+USC Medical Center

OTHER

Sponsor Role collaborator

Virginia Commonwealth University

OTHER

Sponsor Role collaborator

National Institute on Alcohol Abuse and Alcoholism (NIAAA)

NIH

Sponsor Role collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

University of Southern California

OTHER

Sponsor Role collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Abigail Smith, PhD

Role: PRINCIPAL_INVESTIGATOR

Northwestern University

Locations

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University of California San Diego NAFLD Research Center

La Jolla, California, United States

Site Status RECRUITING

Keck Medical Center of USC

Los Angeles, California, United States

Site Status RECRUITING

LAC + USC Medical Center

Los Angeles, California, United States

Site Status RECRUITING

UCSF/Zuckerberg San Francisco General Hospital and Trauma Center

San Francisco, California, United States

Site Status RECRUITING

UCSF Medical Center

San Francisco, California, United States

Site Status RECRUITING

University of Miami Health System

Miami, Florida, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

New York Presbyterian/Weill Cornell

New York, New York, United States

Site Status RECRUITING

Columbia University Iriving School of Medicine

New York, New York, United States

Site Status RECRUITING

Duke Liver Center

Durham, North Carolina, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Crystal Santillanes, MS

Role: CONTACT

312-503-5536

Mary Beth Tull, MBA, CCRP

Role: CONTACT

312-503-4746

Facility Contacts

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Rohit Loomba

Role: primary

Norah Terrault

Role: primary

Norah Terrault

Role: primary

Mandana Khalili

Role: primary

Bilal Hameed

Role: primary

David Goldberg

Role: primary

Elliot Tapper

Role: primary

Doug A. Simonetto, MD

Role: primary

(507) 284-2511

Robert Brown

Role: primary

Elizabeth Verna

Role: primary

Cynthia Ann Moylan, MD, MHS, MS

Role: primary

919-681-4044

Mariko Kopping

Role: backup

Srinivasan Dasarathy

Role: primary

Arun Sanyal

Role: primary

Related Links

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http://www.lcnstudy.org

Information about the study

Other Identifiers

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5U24DK130164-04

Identifier Type: NIH

Identifier Source: secondary_id

View Link

Pro00070580

Identifier Type: -

Identifier Source: org_study_id

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