2024-515057-19-00招募中3 期
The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled study
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 144
- 试验地点
- 2
- 主要终点
- The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.
研究概览
简要总结
The primary objective of this study is to evaluate whether metoprolol succinate in addition to standard of care cardiac treatment introduced before the onset of echocardiography detectable left ventricular dysfunction, can slow the rate of progression left ventricular ejection fraction drop that leads to cardiomyopathy in males with DMD compared to placebo.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (>16 years old)
- •Stated willingness to comply with all study procedures and availability for the duration of the study
- •Male, ≥ 8 years and <17 years of age at time of enrolment in the study
- •Ability to take oral medication and be willing to adhere to the study intervention regimen
- •Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and: • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or • Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-offrame' or • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication leading to a downstream stop codon)
- •Taking ACEi treatment at minimum required doses as outlined in the protocol for at least 30 days
排除标准
- •Current or previous permanent use of any beta-blocker medication
- •Treatment with another investigational drug or other intervention within 3 months prior to screening
- •Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds
- •Presence of pacemaker or ICD
- •Clinical signs or symptoms of heart failure
- •Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz echocardiography)
- •Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety)
- •Known allergic reactions to components of the IMPs
结局指标
主要结局
The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.
The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.
次要结局
- • Disease Free Survival (DFS) i.e., the time to develop cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method. • prevalence of patients with cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method • Disease Free Survival (DFS) i.e., the time to develop clinically evident Heart Failure (HF)
研究者
Chief Medical Officer; Principal Investigator
Scientific
Medical University Of Gdansk
研究点 (2)
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