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Study of Resmetirom in Children and Adolescents With MASH

10 settembre 2026 aggiornato da: Madrigal Pharmaceuticals, Inc.

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.

Panoramica dello studio

Stato

Reclutamento

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

61

Fase

  • Fase 2

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

Luoghi di studio

    • Indiana
      • Indianapolis, Indiana, Stati Uniti, 46402
        • Reclutamento
        • Riley Hospital for Children at IU Health

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino

Accetta volontari sani

No

Descrizione

Inclusion Criteria:

  1. Male or female participants 6 to 17 years of age, inclusive.
  2. Parent(s) or legal guardian(s) able to provide written informed consent (as required by local regulations), with participant assent obtained as applicable.
  3. Diagnose of MASH with fibrosis stage F1-F3 based on a liver biopsy obtained within 24 months before Screening.
  4. Hepatic fat fraction ≥8% by magnetic resonance imaging-proton density fat fraction (MRI-PDFF) obtained during the Screening period.
  5. Able to swallow study tablets.
  6. 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.

Exclusion Criteria:

  1. Previous exposure to resmetirom.
  2. 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.
  3. Clinically significant thyroid disease or use of thyroid replacement therapy, triiodothyronine, thyroxine, or other prohibited thyroid medications.
  4. 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.
  5. Use of glucagon-like peptide-1 (GLP-1) receptor agonists unless on a stable dose for at least 24 weeks before screening.
  6. Clinically significant alcohol or substance abuse, or regular use of tobacco/nicotine products within 6 months before screening.
  7. Active or clinically significant infection, including chronic hepatitis B or hepatitis C infection, HIV infection, or other immunocompromising conditions.
  8. 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.
  9. History of malignancy within the past 5 years (except adequately treated non-melanoma skin cancer or other protocol-permitted exceptions).
  10. History of organ transplantation or known immunocompromised status.
  11. Participation in another investigational study within 60 days or 5 half-lives (whichever is longer) before screening, unless permitted by the protocol.
  12. Major surgery within 6 weeks before screening.
  13. Known hypersensitivity to resmetirom or any excipient.
  14. Females who are pregnant or breastfeeding.
  15. Any condition that, in the opinion of the Investigator, would compromise participant safety, compliance, or the integrity of the study.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione sequenziale
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Adolescent Dose-Escalation Cohort 1
Adolescents participants 12 to 17 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days
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.
Sperimentale: Adolescent Dose-Escalation Cohort 2
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.
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.
Sperimentale: Adolescent Dose-Escalation Cohort 3
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.
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.
Sperimentale: Adolescent Dose-Escalation Cohort 4
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.
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.
Sperimentale: Adolescent Dose-Escalation Cohort 5
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.
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.
Sperimentale: Child Dose-Escalation Cohort 6
Children participants 6 to 11 years of age receive the first planned dose level of oral resmetirom once daily for approximately 14 days.
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.
Sperimentale: Child Dose-Escalation Cohort 7
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.
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.
Sperimentale: Child Dose-Escalation Cohort 8
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.
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.
Sperimentale: Child Dose-Escalation Cohort 9
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.
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.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Safety and tolerability of multiple ascending doses of resmetirom
Lasso di tempo: 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.
Baseline through approximately Day 21 (or the protocol-defined safety follow-up period)

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)
Lasso di tempo: Day 1 through Day 14
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.
Day 1 through Day 14
Pharmacodynamic biomarkers associated with resmetirom exposure
Lasso di tempo: Baseline through Day 21
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.
Baseline through Day 21

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

1 agosto 2026

Completamento primario (Stimato)

1 luglio 2029

Completamento dello studio (Stimato)

1 luglio 2029

Date di iscrizione allo studio

Primo inviato

10 agosto 2026

Primo inviato che soddisfa i criteri di controllo qualità

10 agosto 2026

Primo Inserito (Effettivo)

13 agosto 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

11 settembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

10 settembre 2026

Ultimo verificato

1 settembre 2026

Maggiori informazioni

Termini relativi a questo studio

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Sì

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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