Идёт набор NCT06215716

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

Фаза III С лечением NASH With Fibrosis MASH With Fibrosis

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Efruxifermin, Placebo.
Кому может быть актуально: Состояния в реестре: NASH With Fibrosis, MASH With Fibrosis. Базовые параметры: 18 лет — 80 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США, Аргентина, Австралия, Канада, Франция +13
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis

Обзор

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.

Подробное описание

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of efruxifermin (EFX) in subjects with non-cirrhotic nonalcoholic steatohepatitis (NASH)/metabolic dysfunction-associated steatohepatitis (MASH) and fibrosis stage 2 or 3 (F2 or F3).

Approximately 1,650 subjects will be enrolled into 2 cohorts.

Cohort 1 will enroll approximately 750 subjects with biopsy-confirmed NASH/MASH and fibrosis stage F2 or F3. Subjects in Cohort 1 will undergo evaluation of histologic efficacy endpoints at Week 52.

Cohort 2 will enroll approximately 900 subjects with biopsy-confirmed fibrosis stage F3. Subjects in Cohort 2 may enroll regardless of NAFLD Activity Score (NAS). Subjects in Cohort 2 will undergo liver biopsy assessment at Week 96.

Eligible subjects will be randomized in a 1:1:1 ratio to receive:

* EFX 28 mg administered subcutaneously once weekly * EFX 50 mg administered subcutaneously once weekly * Placebo administered subcutaneously once weekly

Subjects will participate in:

* a screening period of up to 12 weeks, * a 52-week primary histology endpoint treatment period (Cohort 1), * a 96-week secondary histology endpoint period (Cohort 2), * long-term treatment and clinical outcomes follow-up for up to approximately -240 weeks total treatment duration, and * a follow-up visit approximately 30 days after the last dose of study drug.

The study will evaluate the effects of EFX compared with placebo on histologic improvement in NASH/MASH, fibrosis regression, noninvasive markers of liver fibrosis, biochemistry markers of lipidic and glycemic metabolism, liver-related clinical outcomes, and long-term safety.

Clinical outcomes assessments include evaluation of liver-related events and all-cause mortality. Key secondary and long-term outcome assessments include evaluation of fibrosis progression, liver stiffness by FibroScan, and Enhanced Liver Fibrosis (ELF) score at prespecified time points including Weeks 96 and 240.

Subjects who discontinue study drug may continue study assessments according to the protocol schedule to support long-term efficacy and safety evaluations.

Вмешательства

Препарат Efruxifermin
Administered by subcutaneous injection
Препарат Placebo
Administered by subcutaneous injection

Первичные конечные точки

Cohort 1 Only: Resolution of NASH/MASH and a ≥ 1 stage improvement in fibrosis [Срок оценки: 52 Weeks]
Event-free survival [Срок оценки: 240 Weeks]

Вторичные конечные точки (12)

Cohort 1 Only: Resolution of NASH/MASH and no worsening of fibrosis [Срок оценки: 52 Weeks]
Cohort 1 Only: ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis [Срок оценки: 52 Weeks]
Change from baseline of non-invasive markers of liver fibrosis: ELF score [Срок оценки: 52 Weeks]
Change from baseline of non-invasive markers of liver fibrosis: ELF score components (TIMP-1, HA, PIIINP, Pro-C3) [Срок оценки: 52 Weeks]
Change from baseline in non-invasive markers of liver fibrosis: ELF score [Срок оценки: 96 Weeks, 240 Weeks]
Change from baseline in non-invasive markers of liver fibrosis: ELF score components: TIMP-1, HA, PIIINP, Pro-C3 [Срок оценки: Week 240]
Change from baseline of non-invasive markers of liver fibrosis: Fibroscan [Срок оценки: 52 Weeks]
Change from baseline of non-invasive markers of liver fibrosis: Fibroscan [Срок оценки: 96 Weeks, 240 Weeks]
Change from baseline of markers of liver injury: ALT, AST, GGT [Срок оценки: 52 Weeks, 240 Weeks]
Change from baseline of markers of liver injury: Uric Acid [Срок оценки: 52 Weeks, 240 Weeks]
Change from baseline of lipoproteins: Total cholesterol, TG, Non-HDL-C, HDL-C, and LDL-C [Срок оценки: 52 Weeks, 240 Weeks]
Change from baseline of markers of insulin sensitivity and glycemic control: HbA1c [Срок оценки: 52 Weeks, 240 Weeks]

Критерии участия

Критерии включения

Males and non-pregnant, non-lactating females between 18 - 80 years of age inclusive, based on the date of the screening visit.
Previous history or presence of 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose) or type 2 diabetes.
Cohort 1: Biopsy-proven NASH/MASH. Must have had a liver biopsy obtained ≤ 180 days prior to screening with fibrosis stage 2 or 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components:

Steatosis (scored 0 to 3),
Ballooning degeneration (scored 0 to 2), and
Lobular inflammation (scored 0 to 3).
Cohort 2: Biopsy-proven fibrosis stage 3. Must have had a liver biopsy obtained ≤ 180 days prior to screening. Subjects with NAS <4 may be enrolled and are not required to meet 1 point in each of the components of NAS.

Критерии исключения

Other causes of liver disease based on medical history and/or liver histology and/or central laboratory results.
Presence of cirrhosis on liver biopsy (fibrosis stage 4).
Type 1 or uncontrolled Type 2 diabetes.

Other inclusion and exclusion criteria may apply.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение: Рандомизированное
Модель: Параллельные группы
Маскирование: Четверное слепое
Основная цель: Лечение