Study of Resmetirom in Children and Adolescents With MASH

NCT07763015 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MGL-3196-29
• Study Type: interventional
• Target: 61
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

MASH; MASH - Metabolic Dysfunction-Associated Steatohepatitis

Keywords

MASH; MASLD; Pediatric; Resmetirom; Liver Disease; Fibrosis

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of multiple ascending doses of resmetirom

  Safety and tolerability will be assessed by the incidence and severity of adverse events, serious adverse events, clinical laboratory evaluations, vital signs, 12-lead electrocardiograms, physical examinations, and other protocol-defined safety assessments.

  Baseline through approximately Day 21 (or the protocol-defined safety follow-up period)

Secondary Outcomes (2)

• Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)

  Day 1 through Day 14

• Pharmacodynamic biomarkers associated with resmetirom exposure

  Baseline through Day 21

Study Arms (9)

Adolescent Dose-Escalation Cohort 1

EXPERIMENTAL

Adolescents participants 12 to 17 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days

Drug: Resmetirom

Adolescent Dose-Escalation Cohort 2

EXPERIMENTAL

Adolescent participants 12 to 17 years of age receive the second planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Adolescent Dose-Escalation Cohort 3

EXPERIMENTAL

Adolescent participants 12 to 17 years of age receive the third planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Adolescent Dose-Escalation Cohort 4

EXPERIMENTAL

Adolescent participants 12 to 17 years of age receive the fourth planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Adolescent Dose-Escalation Cohort 5

EXPERIMENTAL

Adolescent participants 12 to 17 years of age receive the fifth planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Child Dose-Escalation Cohort 6

EXPERIMENTAL

Children participants 6 to 11 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days.

Drug: Resmetirom

Child Dose-Escalation Cohort 7

EXPERIMENTAL

Children participants 6 to 11 years of age receive the second planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Child Dose-Escalation Cohort 8

EXPERIMENTAL

Children participants 6 to 11 years of age receive the third planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Child Dose-Escalation Cohort 9

EXPERIMENTAL

Children participants 6 to 11 years of age receive the fourth planned dose level of oral resmetirom once daily for approximately 14 days following review of safety and PK data.

Drug: Resmetirom

Interventions

Resmetirom DRUG

Resmetirom (MGL-3196) : Oral resmetirom administered once daily. Participants receive one of several protocol-defined dose levels assigned according to the sequential multiple ascending-dose study design. Dose escalation proceeds following review of available safety, pharmacokinetic, and pharmacodynamic data.

Adolescent Dose-Escalation Cohort 1; Adolescent Dose-Escalation Cohort 2; Adolescent Dose-Escalation Cohort 3; Adolescent Dose-Escalation Cohort 4; Adolescent Dose-Escalation Cohort 5; Child Dose-Escalation Cohort 6; Child Dose-Escalation Cohort 7; Child Dose-Escalation Cohort 8; Child Dose-Escalation Cohort 9

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female participants 6 to 17 years of age, inclusive.
• Parent(s) or legal guardian(s) able to provide written informed consent (as required by local regulations), with participant assent obtained as applicable.
• Diagnose of MASH with fibrosis stage F1-F3 based on a liver biopsy obtained within 24 months before Screening.
• Hepatic fat fraction ≥8% by magnetic resonance imaging-proton density fat fraction (MRI-PDFF) obtained during the Screening period.
• Able to swallow study tablets.
• Females of childbearing potential must have a negative pregnancy test at screening, must not be pregnant or breastfeeding, and must agree to use a highly effective method of contraception during the study and for at least 30 days after the last dose of study drug. Premenarchal participants and those not of childbearing potential are eligible without contraception.

You may not qualify if:

• Previous exposure to resmetirom.
• Clinically significant liver disease other than MASH or evidence of cirrhosis (F4), decompensated liver disease, or other hepatic conditions that may interfere with study participation or interpretation of results.
• Clinically significant thyroid disease or use of thyroid replacement therapy, triiodothyronine, thyroxine, or other prohibited thyroid medications.
• Use of prohibited concomitant medications, including medications known to affect hepatic steatosis or liver function, lipid-lowering therapies, CYP2C8 inhibitors, OATP1B1/OATP1B3/BCRP inhibitors, protease inhibitors, St. John's Wort, or other medications prohibited by the protocol.
• Use of glucagon-like peptide-1 (GLP-1) receptor agonists unless on a stable dose for at least 24 weeks before screening.
• Clinically significant alcohol or substance abuse, or regular use of tobacco/nicotine products within 6 months before screening.
• Active or clinically significant infection, including chronic hepatitis B or hepatitis C infection, HIV infection, or other immunocompromising conditions.
• History or presence of clinically significant cardiovascular, pulmonary, renal, gastrointestinal, neurologic, hematologic, endocrine, psychiatric, or other medical conditions that, in the opinion of the Investigator, could interfere with study participation or interpretation of study results.
• History of malignancy within the past 5 years (except adequately treated non-melanoma skin cancer or other protocol-permitted exceptions).
• History of organ transplantation or known immunocompromised status.
• Participation in another investigational study within 60 days or 5 half-lives (whichever is longer) before screening, unless permitted by the protocol.
• Major surgery within 6 weeks before screening.
• Known hypersensitivity to resmetirom or any excipient.
• Females who are pregnant or breastfeeding.
• Any condition that, in the opinion of the Investigator, would compromise participant safety, compliance, or the integrity of the study.

Sponsors & Collaborators

• Madrigal Pharmaceuticals, Inc.: lead

Study Sites (1)

Riley Hospital for Children at IU Health

Indianapolis, Indiana, 46402, United States

Location

MeSH Terms

Conditions

Liver Diseases; Fibrosis

Interventions

resmetirom

Condition Hierarchy (Ancestors)

Digestive System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Tom Hare

CONTACT

267-406-0611; info@madrigalpharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2026
• First Posted: August 13, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: August 13, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)