NCT06976606招募中不适用
An Observational Study to Assess Real-world Patient Characteristics, Clinical Course, and Treatment Patterns for Symptomatic Patients With Arrhythmogenic Cardiomyopathy (ACM) Due To a PlaKoPhilin-2 Pathogenic Variant (PKP2)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 6
- 主要终点
- Premature ventricular contractions (PVC)
研究概览
简要总结
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
详细描述
SNAPSHOT-PKP2 is an observational, multicenter study that consists of 2 parts, a retrospective EMR review (up to 2 years) and a prospective observational study (1 year), to evaluate the clinical burden of illness of patients with PKP2-ACM, and to prospectively evaluate changes in key cardiac parameters and patient-reported outcome measures (PROMs) associated with PKP2-ACM progression.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults with a clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
- •Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
- •Frequent premature ventricular contractions (PVCs)
- •Patients must have an ICD placed prior to enrollment
- •Left ventricular ejection fraction (LVEF) ≥ 50% for Part A participants. Left ventricular ejection fraction (LVEF) ≥40% for Part B participants.
排除标准
- •Evidence of variant(s) in addition to PKP2 that meet standard criteria to be considered pathogenic or likely pathogenic for an arrhythmogenic cardiomyopathy.
- •A history of other cardiac abnormalities as specified in the protocol.
- •New York Heart Association symptoms of heart failure of Class IV at the time of consent.
- •A history of prior gene transfer therapy.
结局指标
主要结局
Premature ventricular contractions (PVC)
时间窗: 12 months
Change from baseline
次要结局
- Ventricular arrhythmias and associated clinical events(12 months)
- ECG parameters(12 months)
- Cardiac structure and function(12 months)
- Patient reported outcomes(12 months)
- Cardiac Biomarkers(12 months)
- Health resource utilization(12 months)
研究者
研究点 (6)
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