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- Klinische proef NCT07763015
Study of Resmetirom in Children and Adolescents With MASH
10 september 2026 bijgewerkt door: 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.
Studie Overzicht
Toestand
Werving
Interventie / Behandeling
Studietype
Ingrijpend
Inschrijving (Geschat)
61
Fase
- Fase 2
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studiecontact
- Naam: Tom Hare
- Telefoonnummer: 267-406-0611
- E-mail: info@madrigalpharma.com
Studie Locaties
-
-
Indiana
-
Indianapolis, Indiana, Verenigde Staten, 46402
- Werving
- Riley Hospital for Children at IU Health
-
-
Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
Accepteert gezonde vrijwilligers
Nee
Beschrijving
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Sequentiële toewijzing
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
|
Experimenteel: 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.
|
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Safety and tolerability of multiple ascending doses of resmetirom
Tijdsspanne: 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)
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)
Tijdsspanne: 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
Tijdsspanne: 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
|
Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
1 augustus 2026
Primaire voltooiing (Geschat)
1 juli 2029
Studie voltooiing (Geschat)
1 juli 2029
Studieregistratiedata
Eerst ingediend
10 augustus 2026
Eerst ingediend dat voldeed aan de QC-criteria
10 augustus 2026
Eerst geplaatst (Werkelijk)
13 augustus 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
11 september 2026
Laatste update ingediend die voldeed aan QC-criteria
10 september 2026
Laatst geverifieerd
1 september 2026
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- MGL-3196-29
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
NEE
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Ja
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
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