Enavogliflozin for the Management of Patients With Amyloid CardiomyopaThy - A Randomized, Double-Blind, Placebo-Controlled, Crossover, Multicenter Trial
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 68
- 试验地点
- 4
- 主要终点
- Change in KCCQ-CSS from baseline to Week 12
研究概览
简要总结
This study aims to evaluate the safety and effectiveness of Enavogliflozin 0.3 mg, an SGLT2 inhibitor, in patients with amyloid cardiomyopathy. Participants will take both the study drug and a placebo in two separate periods, with a wash-out period in between. The goal is to determine whether Enavogliflozin is safe and effective for treating amyloid cardiomyopathy.
详细描述
Patients with a confirmed diagnosis of cardiac amyloidosis and symptomatic heart failure (NYHA class II-III) will be recruited.
Participants will be randomized to receive either the study drug or placebo and will undergo baseline and 12-week assessments, including the Kansas City Cardiomyopathy Questionnaire (KCCQ), 6-minute walk test, and cardiopulmonary exercise testing. Following a 4-week washout period, participants will cross over to the alternate treatment arm (study drug or placebo) and repeat the same assessments at baseline and 12 weeks.
The primary endpoint will be the change in KCCQ Clinical Summary Score (KCCQ-CSS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults aged 19 years or older
- •Diagnosed with amyloid cardiomyopathy confirmed by cardiac biopsy or non-invasive imaging
- •Presence of heart failure symptoms corresponding to NYHA functional class II-III
- •Patients on stable oral diuretic use, without dose changes exceeding 50% of the previous dose, for at least 2 weeks prior to study enrollment
- •Ambulatory (able to walk)
- •Able to provide written informed consent for study participation
排除标准
- •Pregnant or breastfeeding women
- •Active infection
- •Major cardiovascular events (e.g., myocardial infarction, stroke) within the past 6 months
- •Scheduled for coronary revascularization, CRT-D implantation, atrial flutter/fibrillation ablation, or valve surgery
- •History of heart transplantation
- •Estimated glomerular filtration rate (eGFR) < 60 ml/min/1.73 m² (calculated by CKD-EPI formula)
- •No history of cancer within the past 5 years at screening (except borderline cancers without recurrence for 2-3 years; multiple myeloma with cardiac involvement is classified as cardiac amyloidosis and is exempt)
- •Heart failure primarily caused by severe left sided valvular disease or ischemic heart disease (except if valvular disease is corrected)
- •Type 1 diabetes mellitus or insulin-dependent diabetes
- •History of ketoacidosis, complicated urinary or genital infections, or kidney stones
- •Systolic blood pressure < 80 mmHg or symptomatic hypotension
- •Major surgery within 90 days prior to enrollment
- •Known hypersensitivity or allergic reaction to the study drug or its components
- •Moderate to severe liver impairment
- •Chronic alcohol or substance abuse
- •Residing in long-term care facilities (e.g., nursing homes)
- •Unable to understand or comply with study drug, procedures, or follow-up, or who are deemed by the investigator to be unlikely to complete the study
研究组 & 干预措施
Enavogliflozin
Administer Enavogliflozin 0.3 mg
干预措施: Enavogliflozin 0.3mg (Drug)
Placebo
Administer placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change in KCCQ-CSS from baseline to Week 12
时间窗: Baseline and Week 12
Change in the Clinical Summary Score of the Kansas City Cardiomyopathy Questionnaire (KCCQ-CSS) from baseline to 12 weeks after treatment initiation. The KCCQ-CSS is a patient-reported outcome measure that assesses symptom frequency and physical limitation in patients with heart failure. Scores range from 0 to 100, with higher scores indicating better symptom status and physical function (i.e., better health status). A higher change from baseline reflects an improvement in clinical status.
次要结局
- Change in 6-Minute Walk Distance(Baseline and Week 12)
- Incidence of Worsening Heart Failure Events or Death(From baseline to Week 12 during each treatment period (excluding the 4-week washout period))
- Change in log-transformed NT-proBNP levels(Baseline and Week 12)
- Change in Body Weight(Baseline and Week 12)
- Change in Body Fat Percentage(Baseline and Week 12)
- Change in Extracellular Water Ratio(Baseline and Week 12)
研究者
Jong-Chan Youn, MD, PhD
Professor
Seoul St. Mary's Hospital