An Open-label Extension Study to Assess the Long-term Safety and Efficacy of Danicamtiv in Adults With Symptomatic Genetic and Familial Dilated Cardiomyopathy
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 332
- 试验地点
- 156
- 主要终点
- Frequency and severity of serious adverse events (SAEs)
研究概览
简要总结
Danicamtiv is an investigational medication, which means it is still being studied and has not been approved. The purpose of this study is to learn how well danicamtiv works, how safe it is, how well people can take it when used in patients with genetic or familial dilated cardiomyopathy (DCM). There are currently no approved medicines made specifically to treat genetic or familial DCM.
详细描述
The Sponsor is studying an investigational medication called danicamtiv to understand its long-term benefits and risks in patients with symptomatic genetic or familial DCM. This study will examine the durability of treatment effects with continued exposure and evaluate appropriateness of chronic dosing over time.
Participants will:
- Take the investigational medicine, danicamtiv, twice daily throughout the study, which is up to 5 years
- Visit the clinic about 16 times for clinical evaluations and diagnostic tests
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed 24 weeks of blinded study treatment (corresponding to the Week 26 Visit in DAN-301) and planned assessments in Part 1 or Part 2 of the DAN-301 study.
- •If sexually active, must use a highly effective birth control method from the Screening Visit through 3 months after the last dose.
- •Is able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to local and institutional guidelines before the first study-specific procedure.
排除标准
- •Acute coronary syndrome or hemodynamically significant epicardial coronary disease, or coronary revascularization (percutaneous coronary intervention [PCI] or coronary artery bypass graft [CABG]) performed within 90 days of DAN-302 Screening Visit.
- •Recent (<90 days) hospitalization for heart failure (HF) or use of IV diuretic.
- •Other recent (<90 days) cardiovascular events (e.g., cerebrovascular accident).
- •Hospitalization for sepsis or other systemic infections within 30 days of Screening. A systemic infection includes suspected or confirmed infection associated with fever, hemodynamic instability, bacteremia, or organ dysfunction typically requires urgent evaluation and/or acute care.
- •Any malignancy requiring treatment after enrollment in DAN-301 that could limit patient's survival.
研究组 & 干预措施
Danicamtiv
干预措施: Danicamtiv (Drug)
结局指标
主要结局
Frequency and severity of serious adverse events (SAEs)
时间窗: 61 months
Frequency and severity of disease-related events (DREs)
时间窗: 61 months
Frequency of abnormal findings from physical examination
时间窗: 61 months
Frequency of abnormal findings from vital signs
时间窗: 61 months
Frequency of abnormal findings from 12-lead electrocardiograms (ECGs)
时间窗: 61 months
Frequency of abnormal findings from clinical laboratory assessments
时间窗: 61 months
Frequency and severity of treatment-emergent adverse events (TEAEs)
时间窗: 61 months
Frequency and severity of adverse events of special interest (AESIs)
时间窗: 61 months
次要结局
- Change from Baseline (Day 1) to Week 26, Months 18, 30, 42, 54, 60, and 61 in cardiac structure, function, and size using Transthoracic Echocardiogram (TTE)(61 months)
- Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS)(61 months)
- Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L) Utility Score(61 months)
- Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in EuroQol Visual Analogue Scale (EQ-VAS) Score(61 months)
- Proportion of participants from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 who achieve at least a one-class improvement in New York Heart Association (NYHA) class(61 months)
- Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentrations(61 months)