A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis

Resumen

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3). The study will enroll subjects in two cohorts for a total samples size of 1650 subjects.

Elegibilidad

Diseño del estudio

Variables de resultado

1. Variable de resultado principal

   Cohort 1 Only: Resolution of NASH/MASH and a ≥ 1 stage improvement in fibrosis

   Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)

   Plazo 52 Weeks

2. Variable de resultado principal

   Event-free survival

   Based on time from randomization to the first clinical event including evidence of disease progression, liver decompensation events, liver transplantation or eligibility for liver transplantation, and all-cause mortality.

   Plazo 240 Weeks

3. Variable de resultado secundaria

   Change from baseline of non-invasive markers of liver fibrosis: ELF score components (TIMP-1, HA, PIIINP, Pro-C3)

   Tissue inhibitor of metalloproteinase-1 \[TIMP-1\], hyaluronic acid \[HA\], amino terminal pro-peptide of type 3 procollagen \[PIIINP\]), and propeptide of type 3 procollagen (Pro-C3)

   Plazo 52 Weeks

4. Variable de resultado secundaria

   Cohort 1 Only: Resolution of NASH/MASH and no worsening of fibrosis

   Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)

   Plazo 52 Weeks

5. Variable de resultado secundaria

   Cohort 1 Only: ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis

   Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)

   Plazo 52 Weeks

6. Variable de resultado secundaria

   Change from baseline of non-invasive markers of liver fibrosis: ELF score

   The ELF score measures the severity of liver fibrosis and estimates the risk of disease progression to cirrhosis or liver-related events, with higher scores indicating higher liver fibrosis level and higher risk of disease progression.

   Plazo 52 Weeks

7. Variable de resultado secundaria

   Change from baseline in non-invasive markers of liver fibrosis: ELF score

   The ELF score measures the severity of liver fibrosis and estimates the risk of disease progression to cirrhosis or liver-related events, with higher scores indicating higher liver fibrosis level and higher risk of disease progression.

   Plazo 96 Weeks, 240 Weeks

8. Variable de resultado secundaria

   Change from baseline in non-invasive markers of liver fibrosis: ELF score components: TIMP-1, HA, PIIINP, Pro-C3

   The ELF score measures the severity of liver fibrosis and estimates the risk of disease progression to cirrhosis or liver-related events, with higher scores indicating higher liver fibrosis level and higher risk of disease progression.

   Plazo Week 240

9. Variable de resultado secundaria

   Change from baseline of non-invasive markers of liver fibrosis: Fibroscan

   Liver stiffness assessed by transient elastography (FibroScan)

   Plazo 52 Weeks

10. Variable de resultado secundaria

    Change from baseline of non-invasive markers of liver fibrosis: Fibroscan

    Liver stiffness assessed by transient elastography (FibroScan)

    Plazo 96 Weeks, 240 Weeks

11. Variable de resultado secundaria

    Change from baseline of markers of liver injury: ALT, AST, GGT

    ALT (U/L), AST (U/L), GGT (U/L)

    Plazo 52 Weeks, 240 Weeks

12. Variable de resultado secundaria

    Change from baseline of markers of liver injury: Uric Acid

    Uric acid (mg/dL)

    Plazo 52 Weeks, 240 Weeks

13. Variable de resultado secundaria

    Change from baseline of lipoproteins: Total cholesterol, TG, Non-HDL-C, HDL-C, and LDL-C

    Total cholesterol (mg/dL), TG (mg/dL), Non-HDL-C (mg/dL), HDL-C (mg/dL), and LDL-C (mg/dL)

    Plazo 52 Weeks, 240 Weeks

14. Variable de resultado secundaria

    Change from baseline of markers of insulin sensitivity and glycemic control: HbA1c

    HbA1c (%)

    Plazo 52 Weeks, 240 Weeks

15. Variable de resultado secundaria

    Change from baseline of markers of insulin sensitivity and glycemic control: Adiponectin

    Adiponectin (mg/L)

    Plazo 52 Weeks, 240 Weeks

16. Variable de resultado secundaria

    Change from baseline of body weight (kg)

    Plazo 52 Weeks, 240 Weeks

17. Variable de resultado secundaria

    To assess the safety and tolerability of EFX through the reporting of extent of exposure (weeks)

    Plazo 52 Weeks, 240 Weeks

18. Variable de resultado secundaria

    To assess the safety and tolerability of EFX through the reporting of adverse events (severity of events)

    Plazo 52 Weeks, 240 Weeks

19. Variable de resultado secundaria

    To assess the safety and tolerability of EFX through the reporting of adverse events (frequency of events)

    Plazo 52 Weeks, 240 Weeks

20. Variable de resultado secundaria

    To assess the safety and tolerability of EFX through the reporting of abnormal clinical laboratory tests, ECGs, ultrasounds, vital sign assessments (number of patients)

    Plazo 52 Weeks, 240 Weeks

21. Variable de resultado secundaria

    To assess the immunogenicity of EFX through the reporting of antidrug antibodies (number of patients)

    Plazo 52 Weeks, 240 Weeks

Fechas

Real significa que el evento ocurrió. Estimada significa que el patrocinador lo prevé. Ambas cosas significan algo distinto.

Ubicaciones

319 centros están en reclutamiento

En el registro figuran 306 centros más.

Documentos del estudio

No hay documentos enlazados en este registro.

Cambios a lo largo del tiempo

No se han registrado cambios desde que incorporamos este registro por primera vez.

Se registra un cambio cada vez que el patrocinador actualiza el registro. El estado, las fechas, la inscripción y los centros aparecen aquí a medida que cambian.