61 Participants NeededMy employer runs this trial

Resmetirom for Fatty Liver Disease

TH
Overseen ByTom Hare
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

What You Need to Know Before You Apply

What is the purpose of this trial?

This study will evaluate the safety, pharmacokinetics (how the body absorbs, distributes, metabolizes, and eliminates the drug), and pharmacodynamics (how the drug affects the body) of resmetirom in children and adolescents with metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis. Participants will receive oral resmetirom once daily for approximately 14 days at one of several dose levels. The information from this study will help determine appropriate dosing and further evaluate the safety and biological effects of resmetirom in pediatric participants with MASH.

Are You a Good Fit for This Trial?

This trial is for children and teens diagnosed with metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis. Participants should be generally healthy aside from their liver condition, as determined by the study doctors.

Inclusion Criteria

Hepatic fat fraction ≥8% by MRI-PDFF obtained during the Screening period
Able to swallow study tablets
I am between 6 and 17 years old.
See 3 more

Exclusion Criteria

Known hypersensitivity to resmetirom or any excipient
I have not had any cancer in the past 5 years, except for treated non-melanoma skin cancer.
History or presence of clinically significant cardiovascular, pulmonary, renal, gastrointestinal, neurologic, hematologic, endocrine, psychiatric, or other medical conditions that could interfere with study participation or interpretation of results
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral resmetirom once daily for approximately 14 days at one of several dose levels

2 weeks
Daily dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week
1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • Resmetirom

Trial Overview

The study tests different doses of resmetirom, a daily oral medication, to see how safe it is and how children's bodies process it. The goal is to find the right dose for kids with MASH over about two weeks.

How Is the Trial Designed?

9

Treatment groups

Experimental Treatment

Group I: Child Dose-Escalation Cohort 9Experimental Treatment1 Intervention
Group II: Child Dose-Escalation Cohort 8Experimental Treatment1 Intervention
Group III: Child Dose-Escalation Cohort 7Experimental Treatment1 Intervention
Group IV: Child Dose-Escalation Cohort 6Experimental Treatment1 Intervention
Group V: Adolescent Dose-Escalation Cohort 5Experimental Treatment1 Intervention
Group VI: Adolescent Dose-Escalation Cohort 4Experimental Treatment1 Intervention
Group VII: Adolescent Dose-Escalation Cohort 3Experimental Treatment1 Intervention
Group VIII: Adolescent Dose-Escalation Cohort 2Experimental Treatment1 Intervention
Group IX: Adolescent Dose-Escalation Cohort 1Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Madrigal Pharmaceuticals, Inc.

Lead Sponsor

Trials
16
Recruited
5,400+