Department of Internal Medicine IV, University Hospital Tübingen, Tübingen, Germany.
Department of Gastroenterology and Hepatology, Faculty of Medicine, Medical University of Silesia, Katowice, Poland.
Trials. 2024 Jun 11;25(1):374. doi: 10.1186/s13063-024-08208-4.
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.
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.
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.
European Union Clinical Trials Register, EudraCT, 2021-006069-39. Registered on March 13, 2022.
代谢相关脂肪性肝病（MASLD）是一种主要的慢性肝脏疾病，在全球范围内普遍存在，与肥胖、糖尿病和血脂异常密切相关。必需磷脂（EPL）被推荐作为治疗肝脏疾病的辅助治疗方法，包括 MASLD 或代谢相关脂肪性肝炎、肝硬化和病毒性肝炎。虽然 EPL 作为 MASLD 治疗的辅助治疗的疗效早已得到证实，但关于其使用的某些方面，例如标准治疗参数的影响、EPL 对生活质量（QoL）的影响以及 MASLD 患者症状评估的变化等，仍有待探讨。拟议的试验旨在评估 EPL 的疗效和安全性，以及随后患有 2 型糖尿病（T2DM）和/或血脂异常和/或肥胖的 MASLD 患者的 QoL。
这是一项多中心、多国、双盲、随机、两臂、安慰剂对照、平行组、四期临床试验。该试验正在大约 190 名患者中进行，他们以 1:1 的比例随机分为 EPL 组（Essentiale® 1800mg/天口服+标准治疗）或安慰剂组（安慰剂+标准治疗）。主要结局是通过瞬时弹性成像从基线评估 EPL 对肝脂肪变性的疗效，评估时间为 6 个月。次要结局包括通过慢性肝脏疾病问卷-代谢相关脂肪性肝病/代谢相关脂肪性肝炎和从基线到 6 个月的症状评估（使用全球总体症状量表）评估 QoL 参数的变化，包括乏力、抑郁感、腹痛/不适或疲劳等症状。
本试验方案设计将通过评估各种结局指标，全面探讨 EPL 联合标准治疗对 T2DM 和/或血脂异常和/或肥胖的 MASLD 患者肝脂肪变性和 QoL 的疗效及其安全性。
欧洲联盟临床试验注册处，EudraCT，2021-006069-39。于 2022 年 3 月 13 日注册。
