背景:代谢功能障碍相关的脂肪变性肝病(MASLD)是全球主要的慢性肝病,与肥胖密切相关,糖尿病,和血脂异常。必需磷脂(EPL)被推荐作为管理肝脏疾病的支持治疗,包括MASLD或代谢功能障碍相关的脂肪性肝炎,肝硬化,和病毒性肝炎。虽然EPL在MASLD治疗中作为辅助治疗的疗效已经较早确立,其使用的某些方面,如护理标准参数的影响,EPL对MASLD患者的生活质量(QoL)和症状评估变化的影响仍未研究。拟议的试验旨在评估EPL的有效性和安全性以及与2型糖尿病(T2DM)和/或高脂血症和/或肥胖相关的MASLD患者的后续QoL。
方法:这是一个多中心,跨国公司,双盲,随机化,双臂,安慰剂对照,平行组,IV期临床试验。该试验在大约190名患者中进行,这些患者以1:1的比例随机分配到EPL组(Essentiale®1800mg/天口服+标准护理)或安慰剂组(安慰剂+标准护理)。主要结果是评估EPL对肝脏脂肪变性的疗效,通过瞬时弹性成像测量,从基线到6个月。次要结果包括QoL参数的变化,根据慢性肝病问卷-代谢功能障碍相关的脂肪性肝病/代谢功能障碍相关的脂肪性肝炎和症状评估的变化(使用全球总体症状量表)从基线到6个月的症状,包括虚弱,感到沮丧,腹痛/不适,或疲劳。
结论:当前的方案设计将允许通过评估各种结果指标,全面探索将EPL添加到治疗标准中对肝脏脂肪变性和QoL的疗效及其在患有与T2DM和/或高脂血症和/或肥胖相关的MASLD患者中的安全性。
背景:欧盟临床试验注册,EudraCT,2021-006069-39。2022年3月13日注册。
BACKGROUND: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a predominant chronic liver condition globally and is strongly associated with obesity, diabetes mellitus, and dyslipidemia. Essential
phospholipids (EPL) are recommended as supportive treatment for managing liver conditions, including MASLD or metabolic dysfunction-associated steatohepatitis, cirrhosis, and viral hepatitis. While efficacy of EPL as an adjunctive therapy in MASLD treatment has been established earlier, certain aspects of its usage such as the impact of standard-of-care parameters, effect of EPL on quality of life (QoL) and change in symptoms evaluation in patients with MASLD remain unexplored. The proposed
trial aims to assess the efficacy and safety of EPL and the subsequent QoL of patients with MASLD associated with type 2 diabetes mellitus (T2DM) and/or hyperlipidemia and/or obesity.
METHODS: This is a multicenter, multinational, double-blind, randomized, two-arm, placebo-controlled, parallel-group, phase IV clinical
trial. The
trial is being conducted in approximately 190 patients who are randomized on a 1:1 basis either to the EPL arm (Essentiale® 1800 mg/day orally + standard of care) or placebo arm (placebo + standard of care). The primary outcome is to assess the efficacy of EPL on hepatic steatosis, as measured by transient elastography, from baseline to 6 months. The secondary outcomes include change in QoL parameters, as measured by the Chronic Liver Disease Questionnaire-metabolic dysfunction-associated steatotic liver disease/ metabolic dysfunction-associated steatohepatitis and change in symptom evaluation (using the Global Overall Symptom scale) from baseline to 6 months for symptoms, including asthenia, feeling depressed, abdominal pain/discomfort, or fatigue.
CONCLUSIONS: The current protocol design will allow to comprehensively explore the efficacy of EPL added to the standard of care on hepatic steatosis and QoL and its safety in patients with MASLD associated with T2DM and/or hyperlipidemia and/or obesity by assessing various outcome measures.
BACKGROUND: European Union Clinical Trials Register, EudraCT, 2021-006069-39. Registered on March 13, 2022.