MASHPhase 2NCT07763015
Study of Resmetirom in Children and Adolescents With MASH
A Phase 2 study testing Resmetirom in people with MASH and MASH - Metabolic Dysfunction-Associated Steatohepatitis, run by Madrigal Pharmaceuticals, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- MASH, MASH - Metabolic Dysfunction-Associated Steatohepatitis
- What is being tested?
- Resmetirom
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2026to Jul 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46402
About this study
Official title: A Phase 2a, Multicenter, Open-label, Multiple Ascending Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Resmetirom in Pediatric Subjects Ages 6-17 Years With MASH With Fibrosis Stage F1-F3
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.
- Being tested
- Resmetirom
- Keywords
- MASH, MASLD, Pediatric, Resmetirom, Liver Disease, Fibrosis
- Medical subject headings
- Liver Diseases, Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ResmetiromDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: Drug: Resmetirom |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Pharmacokinetic parameters of resmetirom and MGL-3623 following single and repeated dosing, including CL/F (parent only), Vz/F (parent only), Cmax, AUC0-24, and AUC0-∞ after the first dose, and CL/F (parent only), Cmax, and AUC0-24 following repeated dosing, as applicable.
Measured: Day 1 through Day 14
Pharmacodynamic biomarkers associated with resmetirom exposure
Changes in pharmacodynamic biomarkers, including thyroid axis markers, sex hormone-binding globulin, and lipid parameters and their relationship to dose and/or plasma concentrations of resmetirom.
Measured: Baseline through Day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2026
- Study start1 Aug 2026
- Primary completionJul 2029 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MASHStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 61 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Madrigal Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07763015
- Sponsor's protocol ID
- MGL-3196-29
- Status last verified
- Sep 2026
- First submitted
- 10 Aug 2026
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Multicenter, Open-label, Multiple Ascending Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Resmetirom in Pediatric Subjects Ages 6-17 Years With MASH With Fibrosis Stage F1-F3
- ClinicalTrials.gov ID
- NCT07763015
- Acronym / sponsor ID
- MGL-3196-29
- Keywords
- MASH, MASLD, Pediatric, Resmetirom, Liver Disease, Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 61 participants (estimated)
- Lead sponsor
- Madrigal Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Aug 2026 (actual)
- Primary completion
- Jul 2029 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 13 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Adolescent Dose-Escalation Cohort 1 | Experimental | Drug: ResmetiromAdolescents participants 12 to 17 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days |
| Adolescent Dose-Escalation Cohort 2 | Experimental | Drug: ResmetiromAdolescent 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. |
| Adolescent Dose-Escalation Cohort 3 | Experimental | Drug: ResmetiromAdolescent 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. |
| Adolescent Dose-Escalation Cohort 4 | Experimental | Drug: ResmetiromAdolescent 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. |
| Adolescent Dose-Escalation Cohort 5 | Experimental | Drug: ResmetiromAdolescent 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. |
| Child Dose-Escalation Cohort 6 | Experimental | Drug: ResmetiromChildren participants 6 to 11 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days. |
| Child Dose-Escalation Cohort 7 | Experimental | Drug: ResmetiromChildren 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. |
| Child Dose-Escalation Cohort 8 | Experimental | Drug: ResmetiromChildren 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. |
| Child Dose-Escalation Cohort 9 | Experimental | Drug: ResmetiromChildren 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. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ResmetiromResmetirom (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. | Drug | 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 |
Primary outcome
Safety and tolerability of multiple ascending doses of resmetirom
Time frame: Baseline through approximately Day 21 (or the protocol-defined safety follow-up period)
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.
2 secondary outcomes
Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)
Time frame: Day 1 through Day 14
Pharmacodynamic biomarkers associated with resmetirom exposure
Time frame: Baseline through Day 21
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Resmetirom in people with MASH and MASH - Metabolic Dysfunction-Associated Steatohepatitis, run by Madrigal Pharmaceuticals, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tom Hare (267-406-0611, info@madrigalpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of multiple ascending doses of resmetirom (time frame: Baseline through approximately Day 21 (or the protocol-defined safety follow-up period)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2026.