A Phase 3 Study to Evaluate Safety and Biomarkers of Resmetirom (MGL-3196) in Non Alcoholic Fatty Liver Disease Patients

NCT ID: NCT04197479

Last Updated: 2023-09-05

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

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

1343 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-16

Study Completion Date

2023-01-06

Brief Summary

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A double-blind placebo controlled randomized Phase 3 study to evaluate the safety and tolerability of once-daily, oral administration of 80 or 100 mg resmetirom versus matching placebo. At least 100 patients will be enrolled in a 100 mg open-label arm and will include a special safety population (eg, patients with compensated NASH cirrhosis).

Detailed Description

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Conditions

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Non-Alcoholic Fatty Liver Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Open label: resmetirom

100 mg daily

Group Type EXPERIMENTAL

Resmetirom

Intervention Type DRUG

Tablet

Double blinded: matching placebo

Placebo daily

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching tablets

Double blinded: resmetirom 80 mg

80 mg daily

Group Type EXPERIMENTAL

Resmetirom

Intervention Type DRUG

Tablet

Double blinded: resmetirom 100 mg

100 mg daily

Group Type EXPERIMENTAL

Resmetirom

Intervention Type DRUG

Tablet

Interventions

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Placebo

Matching tablets

Intervention Type DRUG

Resmetirom

Tablet

Intervention Type DRUG

Other Intervention Names

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MGL-3196

Eligibility Criteria

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Inclusion Criteria

* Must be willing to participate in the study and provide written informed consent.
* Male and female adults ≥18 years of age.
* Suspected or confirmed diagnosis of NASH or NAFLD (presumed NASH):

* Fibroscan with kPa ≥5.5 and \<8.5; CAP ≥280 dB.m-1 OR
* MRE ≥2 and \<4.0; MRI-PDFF ≥8% liver fat consistent with steatosis and fibrosis stage ≥1 and \<4. OR
* Recent liver biopsy (within past 2 years) documenting NASH/NAFLD with steatosis showing one of the following:

* NAS ≥4, steatosis ≥1, fibrosis stage 0 or F1A/1C with PRO-C3 \<14
* NAS \<4, steatosis ≥1, with fibrosis stage ≤3
* NAS ≥4, steatosis ≥1, fibrosis stage ≤3 without ballooning

* NOTE: Since the completion of enrollment of the double-blind arms, patients meeting all other criteria who have a liver biopsy result from MGL-3196-11 with the following may be enrolled in the open-label active treatment arm of MGL-3196-14 (100 mg dose):

* NAS = 3, steatosis 1, ballooning 1, inflammation 1 with F2 or F3
* NAS = 3, ballooning 0 with F2 or F3
* For the compensated NASH cirrhosis arm, eligible patients must have compensated NASH cirrhosis diagnosed by liver biopsy showing NASH with F4 stage fibrosis (either historic or recent biopsy) or a historic biopsy with NASH F2-F3 fibrosis with subsequent progression to NASH cirrhosis as diagnosed by an expert hepatologist/gastroenterologist.
* Compensated NASH cirrhosis at screening and baseline includes

* Child Pugh-A (score 5-6) ( may have either mild hepatic encephalopathy OR mild diuretic responsive ascites OR albumin \< 3.5 and ≥ 3.2 (not any two of these, unless explained by Gilbert's Syndrome or non-hepatic causes)).
* MELD \< 12 at screening/baseline unless MELD ≥ 12 based on non-cirrhotic parameters (e.g., elevated INR due to anticoagulation, bilirubin elevation due to documented Gilbert's Syndrome, elevated creatine due to renal disease (non-hepatic)).
* Albumin ≥ 3.2.
* Bilirubin \< 2 (unless documented Gilbert's Syndrome).
* MRI-PDFF fat fraction ≥8% obtained during the Screening Period (baseline MRI-PDFF) or a historic MRI-PDFF ≤8 weeks old at the time of randomization.
* Stable dyslipidemia therapy for ≥30 days prior to randomization.

Exclusion Criteria

* History of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to Screening.
* Regular use of drugs historically associated with NAFLD.
* History of bariatric surgery or intestinal bypass surgery within the 5 years prior to randomization or planned during the conduct of the study.
* Weight gain or loss ≥5% total body weight within 12 weeks prior to randomization.
* HbA1c \>9.0%.
* Glucagon-like peptide 1 \[GLP-1\] agonist therapy or high dose vitamin E (\>400 IU/day) unless stable for 24 weeks prior to biopsy.
* Presence of cirrhosis on liver biopsy defined as stage 4 fibrosis.
* Diagnosis of hepatocellular carcinoma (HCC).
* Model for End-stage Liver Disease (MELD) score ≥12, as determined at Screening, unless due to therapeutic anti coagulation or Gilbert syndrome.
* Hepatic decompensation.
* Chronic liver diseases.
* Has an active autoimmune disease.
* Serum ALT \>250 U/L.
* History of biliary diversion.
* Uncontrolled hypertension (either treated or untreated).
* Active, serious medical disease with a likely life expectancy \<2 years.
* Participation in an investigational new drug trial in the 60 days or 5 half-lives, whichever is longer, prior to randomization.
* Any other condition which, in the opinion of the Investigator, would impede compliance, hinder completion of the study, compromise the well-being of the patient, or interfere with the study outcomes.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Madrigal Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rebecca Taub, MD

Role: STUDY_DIRECTOR

Madrigal Pharmaceuticals, Inc.

Locations

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East-West Medical Research Institute

Honolulu, Hawaii, United States

Site Status

Chicago Research Center

Chicago, Illinois, United States

Site Status

Northwestern Memorial Physicians Group

Chicago, Illinois, United States

Site Status

Iowa Diabetes Research

West Des Moines, Iowa, United States

Site Status

Central Research Associates

Birmingham, Alabama, United States

Site Status

Arizona Liver Health - Chandler

Chandler, Arizona, United States

Site Status

East Valley Family Physicians

Chandler, Arizona, United States

Site Status

The Institute For Liver Health - Glendale

Glendale, Arizona, United States

Site Status

Arizona - Desert Clinical Research

Mesa, Arizona, United States

Site Status

The Institute For Liver Health - Tucson

Tucson, Arizona, United States

Site Status

Adobe Gastroenterology

Tucson, Arizona, United States

Site Status

Arkansas Gastroenterology

North Little Rock, Arkansas, United States

Site Status

Fresno Clinical Research Center

Fresno, California, United States

Site Status

National Research Institute - Huntington Park

Huntington Park, California, United States

Site Status

Ruane Clinical Research Group

Los Angeles, California, United States

Site Status

National Research Institute - Los Angeles

Los Angeles, California, United States

Site Status

Catalina Research Institute

Montclair, California, United States

Site Status

National Research Institute - Panorama City

Panorama City, California, United States

Site Status

Alliance Clinical Research

Poway, California, United States

Site Status

San Fernando Valley Health Institute

West Hills, California, United States

Site Status

South Denver Gastroenterology - Swedish Medical Center Office

Englewood, Colorado, United States

Site Status

Excel Medical Clinical Trials

Boca Raton, Florida, United States

Site Status

Velocity Clinical Research, Hallandale Beach (MD Clinical)

Hallandale, Florida, United States

Site Status

Floridian Clinical Research

Hialeah, Florida, United States

Site Status

Nature Coast Clinical Research - Inverness

Inverness, Florida, United States

Site Status

Jacksonville Center for Clinical Research

Jacksonville, Florida, United States

Site Status

Florida Research Institute

Lakewood Rch, Florida, United States

Site Status

Miami Dade Medical Research Institute

Miami, Florida, United States

Site Status

Orlando Research Center

Orlando, Florida, United States

Site Status

Progressive Medical Research

Port Orange, Florida, United States

Site Status

Covenant Research

Sarasota, Florida, United States

Site Status

The Villages Research Center

The Villages, Florida, United States

Site Status

Gastrointestinal Specialists of Georgia

Marietta, Georgia, United States

Site Status

Kansas Medical Clinic - Gastroenterology

Topeka, Kansas, United States

Site Status

L-MARC Research Center

Louisville, Kentucky, United States

Site Status

Digestive Health Center of Louisiana

Baton Rouge, Louisiana, United States

Site Status

Tandem Clinical Research - New Orleans Area Site

Marrero, Louisiana, United States

Site Status

Clinical Trials of America

West Monroe, Louisiana, United States

Site Status

Huron Gastroenterology

Ypsilanti, Michigan, United States

Site Status

Gastrointestinal Associates & Endoscopy Center - Flowood

Flowood, Mississippi, United States

Site Status

Southern Therapy and Advanced Research

Jackson, Mississippi, United States

Site Status

Kansas City Research Institute

Kansas City, Missouri, United States

Site Status

Henderson Research Center

Henderson, Nevada, United States

Site Status

Clarity Clinical Research

East Syracuse, New York, United States

Site Status

Mount Sinai Health System

New York, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Cumberland Research Associates

Fayetteville, North Carolina, United States

Site Status

Diabetes and Endocrinology Consultants

Morehead City, North Carolina, United States

Site Status

TMA - Wilmington Gastroenterology Accociates

Wilmington, North Carolina, United States

Site Status

Platinum - Sterling Research Group - Springdale

Cincinnati, Ohio, United States

Site Status

Aventiv Research Columbus

Columbus, Ohio, United States

Site Status

Awasty Research Network

Marion, Ohio, United States

Site Status

Northeast Clinical Research Center

Bethlehem, Pennsylvania, United States

Site Status

Premier Medical Group - Clarksville - Dunlop Lane

Clarksville, Tennessee, United States

Site Status

Gastro One - Germantown Office - Wolf Park Drive

Germantown, Tennessee, United States

Site Status

Pinnacle Clinical Research - Austin

Austin, Texas, United States

Site Status

The Liver Institute At Methodist Dallas

Dallas, Texas, United States

Site Status

Dallas Research Center

Dallas, Texas, United States

Site Status

Liver Center of Texas

Dallas, Texas, United States

Site Status

Texas Digestive Disease Consultants - Dallas - Baylor University Medical Center Gaston Ave

Dallas, Texas, United States

Site Status

South Texas Research Institute

Edinburg, Texas, United States

Site Status

Texas Digestive Disease Consultants - Forth Worth - Downtown

Fort Worth, Texas, United States

Site Status

Liver Associates of Texas

Houston, Texas, United States

Site Status

Doctor's Hospital at Renaissance

McAllen, Texas, United States

Site Status

Plano Research Center

Plano, Texas, United States

Site Status

Texas Liver Institute/American Research Corporation

San Antonio, Texas, United States

Site Status

Pinnacle Clinical Research - San Antonio

San Antonio, Texas, United States

Site Status

San Antonio Research Center

San Antonio, Texas, United States

Site Status

Texas Digestive Disease Consultants - San Marcos

San Marcos, Texas, United States

Site Status

Texas Digestive Disease Consultants - Bay Area Houston Endoscopy Center

Webster, Texas, United States

Site Status

Wasatch Peak Family Practice

Layton, Utah, United States

Site Status

Salt Lake City Research Center

Murray, Utah, United States

Site Status

Bon Secours Liver Institute of Richmond

Richmond, Virginia, United States

Site Status

National Clinical Research - Richmond

Richmond, Virginia, United States

Site Status

Virginia Commonwealth University School of Medicine

Richmond, Virginia, United States

Site Status

Liver Institute Northwest

Seattle, Washington, United States

Site Status

Fundacion de Investigacion de Diego

San Juan, , Puerto Rico

Site Status

Countries

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United States Puerto Rico

References

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Alkhouri N, McRae MP, Taub R, Hill B, Imperial JC, Kittelson J, Moussa SE, Everson GT. The Cholate Challenge Test Quantified Baseline Functional Heterogeneity and Improvement in Response to Resmetirom in MASH-related Child-Pugh A Cirrhosis. Gastro Hep Adv. 2025 Aug 29;4(10):100785. doi: 10.1016/j.gastha.2025.100785. eCollection 2025.

Reference Type DERIVED
PMID: 41142528 (View on PubMed)

Harrison SA, Taub R, Neff GW, Lucas KJ, Labriola D, Moussa SE, Alkhouri N, Bashir MR. Resmetirom for nonalcoholic fatty liver disease: a randomized, double-blind, placebo-controlled phase 3 trial. Nat Med. 2023 Nov;29(11):2919-2928. doi: 10.1038/s41591-023-02603-1. Epub 2023 Oct 16.

Reference Type DERIVED
PMID: 37845512 (View on PubMed)

Harrison SA, Ratziu V, Anstee QM, Noureddin M, Sanyal AJ, Schattenberg JM, Bedossa P, Bashir MR, Schneider D, Taub R, Bansal M, Kowdley KV, Younossi ZM, Loomba R. Design of the phase 3 MAESTRO clinical program to evaluate resmetirom for the treatment of nonalcoholic steatohepatitis. Aliment Pharmacol Ther. 2024 Jan;59(1):51-63. doi: 10.1111/apt.17734. Epub 2023 Oct 2.

Reference Type DERIVED
PMID: 37786277 (View on PubMed)

Other Identifiers

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MGL-3196-14

Identifier Type: -

Identifier Source: org_study_id

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