NCT07218887招募中1 期
A Phase 1/2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 11
- 主要终点
- Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pathogenic or likely pathogenic mutation in BAG3
- •Medical history of diagnosis of DCM
- •Stable combination of HF SoC medications
- •Adequate acoustic windows for echocardiography
- •LVEF > 15% and ≤ 45% at Screening by echocardiography
- •Diagnosis of chronic HF for at least 3 months
- •NYHA Class I/II/III at Screening
排除标准
- •Presence of antibodies to AAV9
- •Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
- •Decompensated HF
- •Cardiac ventricular arrhythmia that requires treatment
- •History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
- •NYHA Class IV
- •History of substance abuse that could contribute to cardiac dysfunction
- •Note: Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
ALXN2350
Experimental
Participants will receive one of three dose levels of ALXN2350 depending on the cohort.
干预措施: ALXN2350 (Drug)
结局指标
主要结局
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
时间窗: Baseline up to Week 78
次要结局
- CFB in cardiac serum biomarker NT-pro-BNP(Weeks 52 and 78)
- Part B: Number of Participants With TEAEs and SAEs(Baseline up to Week 78)
- Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78(Weeks 52 and 78)
- Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death(Baseline up to Week 78)
研究者
研究点 (11)
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