A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

Rocket Pharmaceuticals Inc.3 个研究点分布在 1 个国家目标入组 9 人2023年8月29日

适应症PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

干预措施RP-A601

概览

阶段: 1 期
干预措施: RP-A601
疾病 / 适应症: PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
发起方: Rocket Pharmaceuticals Inc.
入组人数: 9
试验地点: 3
主要终点: Evaluation of safety associated with RP-A601
状态: 招募中
最后更新: 16天前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验相关资讯

概览

简要总结

This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.

注册库

clinicaltrials.gov

开始日期

2023年8月29日

结束日期

2029年9月1日

最后更新

16天前

研究类型

Interventional

研究设计

Sequential

性别

All

研究者

发起方

Rocket Pharmaceuticals Inc.

责任方

Sponsor

入排标准

入选标准

•Male or female ≥18 years at the time of signing the informed consent
•Capable and willing to provide signed informed consent
•Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
•Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
•History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
•PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
•Left ventricular ejection fraction by echocardiogram or CMR ≥50%

排除标准

•Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
•Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
•Previous participation in a study of gene transfer or gene editing
•Severe Right Ventricular (RV) dysfunction
•New York Heart Association (NYHA) Class IV heart failure.

研究组 & 干预措施

RP-A601

Single ascending dose of RP-A601 in 2 consecutive cohorts

干预措施: RP-A601

结局指标

主要结局

Evaluation of safety associated with RP-A601

时间窗: 12 months post-infusion

Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)

次要结局

Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression(12 months post-infusion)
Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia(12 months post-infusion)
Preliminary efficacy of RP-A601 - Cardiac biomarkers(12 months post-infusion)