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

• Status: Recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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. 100 patients will be enrolled in a 100 mg open-label arm.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Madrigal Pharmaceuticals, Inc.
• Treatments: Hormones

Criteria

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 1A/1C with PRO-C3 <14

• NAS <4, steatosis ≥1, with fibrosis stage ≤3

• NAS ≥4, steatosis ≥1, fibrosis stage ≤3 without ballooning

• 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.