NCT06109181进行中（未招募）1 期
A Phase 1/2 Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 试验地点
- 10
- 主要终点
- Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).
研究概览
简要总结
This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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 meeting the 2010 revised Task Force Criteria (TFC)
- •Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2
- •Frequent premature ventricular complexes (PVCs)
- •Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI
- •Left ventricular ejection fraction ≥ 40%
排除标准
- •Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM
- •Other cardiac abnormalities as specified in the protocol
- •New York Heart Association Functional Class IV at the time of consent
- •History of prior gene transfer therapy
结局指标
主要结局
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).
时间窗: 12 months
Evaluation of Safety and Tolerability of LX2020
次要结局
未报告次要终点
研究者
研究点 (10)
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