NCT05836259招募中1 期
First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM)
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 20
- 主要终点
- Number and severity of Adverse Events over the course of the study.
研究概览
简要总结
This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.
详细描述
The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •MYBPC3 mutation
- •Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- •Left Ventricular Ejection Fraction ≥45%
- •NYHA Functional Class II or III symptoms
- •NT-proBNP ≥160pg/ml
排除标准
- •High AAV9 neutralizing antibody titer
研究组 & 干预措施
Cohort 2
Experimental
Dose for Cohort 2 will be 6E13 vg/kg
干预措施: TN-201 (Genetic)
Cohort 1
Experimental
Dose for Cohort 1 will be 3E13 vg/kg
干预措施: TN-201 (Genetic)
结局指标
主要结局
Number and severity of Adverse Events over the course of the study.
时间窗: 5 Years
Number of Serious Adverse Events related to study drug.
时间窗: 5 Years
次要结局
- Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).(52 Weeks)
研究者
研究点 (20)
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相关资讯
Tenaya Therapeutics Doses First Patient in Phase 1b Trial of TN-401 Gene Therapy for ARVC• Tenaya Therapeutics has dosed the first patient in the RIDGE-1 Phase 1b trial evaluating TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).
• TN-401 is an AAV9-based gene therapy designed to deliver a functional copy of the PKP2 gene directly to heart cells, addressing the underlying genetic cause of ARVC.
• The RIDGE-1 trial is a multicenter study enrolling approximately 15 patients with ARVC across sites in the US, UK, and Europe, with initial data expected next year.
• Preclinical data demonstrated TN-401's ability to normalize heart rhythms, reverse disease progression, and extend survival in animal models, supporting its clinical development.last yearTenaya Therapeutics' TN-201 Receives Clearance for Higher Dose Cohort in Hypertrophic Cardiomyopathy Trial• Tenaya Therapeutics received DSMB clearance to escalate the TN-201 dose to 6x10^13 vg/kg in the MyPeak-1 trial for hypertrophic cardiomyopathy (HCM).
• The decision follows positive early safety data from the first cohort (3x10^13 vg/kg) showing no unexpected adverse events related to the gene therapy.
• The MyPeak-1 protocol was modified to include baseline biopsies, broaden eligibility, and increase the potential participant number to 24.
• Initial findings from the first cohort, including safety, tolerability, biopsy analyses, and biomarker data, are expected in December 2024.last year