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Resmetirom for nonalcoholic fatty liver disease: a randomized, double-blind, placebo-controlled phase 3 trial.
Harrison, Stephen A; Taub, Rebecca; Neff, Guy W; Lucas, K Jean; Labriola, Dominic; Moussa, Sam E; Alkhouri, Naim; Bashir, Mustafa R.
Afiliación
  • Harrison SA; Pinnacle Clinical Research, San Antonio, TX, USA. Stephenharrison87@gmail.com.
  • Taub R; Madrigal Pharmaceuticals, Conshohocken, PA, USA.
  • Neff GW; Covenant Metabolic Specialists, Sarasota, FL, USA.
  • Lucas KJ; Lucas Research, Morehead City, NC, USA.
  • Labriola D; Madrigal Pharmaceuticals, Conshohocken, PA, USA.
  • Moussa SE; University of Arizona for Medical Sciences, Tucson, AZ, USA.
  • Alkhouri N; Arizona Liver Health, Tucson, AZ, USA.
  • Bashir MR; Duke University Medical Center, Durham, NC, USA.
Nat Med ; 29(11): 2919-2928, 2023 Nov.
Article en En | MEDLINE | ID: mdl-37845512
Nonalcoholic steatohepatitis (NASH) is a progressive liver disease with no approved treatment. MAESTRO-NAFLD-1 was a 52-week randomized, double-blind, placebo-controlled phase 3 trial evaluating the safety of resmetirom in adults with nonalcoholic fatty liver disease and presumed NASH. Patients were randomized to three double-blind arms (100 mg resmetirom (n = 325), 80 mg resmetirom (n = 327) or placebo (n = 320)) or open-label 100 mg resmetirom (n = 171). The primary end point was incidence of treatment-emergent adverse events (TEAEs) over 52 weeks and key secondary end points were LDL-C, apoB, triglycerides (over 24 weeks), hepatic fat (over 16 and 52 weeks) and liver stiffness (over 52 weeks). Resmetirom was safe and well tolerated. TEAEs occurred in 86.5% (open-label 100 mg resmetirom), 86.1% (100 mg resmetirom), 88.4% (80 mg resmetirom) and 81.8% (placebo) of patients. TEAEs in excess of placebo included diarrhea and nausea at the initiation of treatment. Key secondary end points included least square means difference from placebo at 80 mg, 100 mg resmetirom: LDL-C (-11.1%, -12.6%), apoB (-15.6%, -18.0%), triglycerides (-15.4%, -20.4%), 16-week hepatic fat (-34.9%, -38.6%), (P < 0.0001) and liver stiffness (-1.02, -1.70) and 52-week hepatic fat (-28.8, -33.9). These findings demonstrate resmetirom was safe and well tolerated in adults with presumed NASH, supporting a role for further clinical development. (ClinicalTrials.gov identifier NCT04197479 ).
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad del Hígado Graso no Alcohólico Límite: Adult / Humans Idioma: En Revista: Nat Med Asunto de la revista: BIOLOGIA MOLECULAR / MEDICINA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad del Hígado Graso no Alcohólico Límite: Adult / Humans Idioma: En Revista: Nat Med Asunto de la revista: BIOLOGIA MOLECULAR / MEDICINA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos