- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07763015
Study of Resmetirom in Children and Adolescents With MASH
10. september 2026 oppdatert av: 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.
Studieoversikt
Status
Rekruttering
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Antatt)
61
Fase
- Fase 2
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Tom Hare
- Telefonnummer: 267-406-0611
- E-post: info@madrigalpharma.com
Studiesteder
-
-
Indiana
-
Indianapolis, Indiana, Forente stater, 46402
- Rekruttering
- Riley Hospital for Children at IU Health
-
-
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
Tar imot friske frivillige
Nei
Beskrivelse
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.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomisert
- Intervensjonsmodell: Sekvensiell tildeling
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
|
Eksperimentell: 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.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Safety and tolerability of multiple ascending doses of resmetirom
Tidsramme: 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)
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Pharmacokinetic parameters of resmetirom (MGL-3196) and its metabolite (MGL-3623)
Tidsramme: 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
Tidsramme: 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
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
1. august 2026
Primær fullføring (Antatt)
1. juli 2029
Studiet fullført (Antatt)
1. juli 2029
Datoer for studieregistrering
Først innsendt
10. august 2026
Først innsendt som oppfylte QC-kriteriene
10. august 2026
Først lagt ut (Faktiske)
13. august 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
11. september 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
10. september 2026
Sist bekreftet
1. september 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- MGL-3196-29
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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