NCT07426419招募中1 期
A Phase 1/2 Multicenter, Open-Label, Dose-Escalation and Dose-Expansion Trial to Assess the Safety, Tolerability, Pharmacodynamics, and Preliminary Efficacy of AFTX-201 Administered to Adult Participants With BCL2-Associated Athanogene 3 (BAG3) Mutation-Associated Dilated Cardiomyopathy: UPBEAT Trial
Affinia Therapeutics4 个研究点 分布在 2 个国家目标入组 22 人开始时间: 2026年9月1日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 22
- 试验地点
- 4
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
研究概览
简要总结
This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation
详细描述
Participants will receive a single intravenous infusion of AFTX-201 and will be followed for safety and clinical assessments. The study includes short-term safety monitoring and long-term follow-up for up to 60 months
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
- •Male or female, 18 to 70 years of age
- •Truncating mutation in BAG3
- •Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) < 45%
- •NYHA Class II or III heart failure symptoms
- •NT-proBNP ≥ 300 pg/mL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg/mL for subjects in atrial fibrillation
- •Willing and able to sign informed consent and comply with study procedures
排除标准
- •Prior myocardial infarction, heart transplant, or presence/requirement of a left ventricular assist device (LVAD)
- •IV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment
- •Positive neutralizing antibodies to ATC-0187
- •Any condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results
研究组 & 干预措施
AFTX-201
Experimental
Participants will receive a single intravenous dose of AFTX-201
干预措施: AFTX-201 (Genetic)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: Baseline through 12 months
Safety and tolerability of AFTX-201 over 12 months.
次要结局
- Change from baseline in pharmacodynamic biomarkers(Baseline through 12 months)
- Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)(Baseline through 12 months)
- Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)(Baseline through 12 months)
- Change from baseline in New York Heart Association (NYHA) functional class(Baseline through 12 months)
- Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)(Baseline through 12 months)
- Number of participants with adverse events during long-term follow-up(Baseline through 60 months)
研究者
研究点 (4)
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