NCT06092034招募中2 期
Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 14
- 试验地点
- 9
- 主要终点
- Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
研究概览
简要总结
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
详细描述
The study is a single arm Phase 2 clinical trial to characterize the safety and efficacy of RP-A501, a recombinant adeno-associated serotype 9 (rAAV9 capsid containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene) in male patients with Danon Disease.
Male subjects ≥8 years of age will receive a single intravenous infusion of RP-A501.
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
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- •Age ≥8 years.
- •Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
- •Abnormal thickening of Left ventricular wall,
- •Left ventricular ejection fraction (LVEF) ≥ 50%.
- •New York Heart Association (NYHA) Class II to III.
- •High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
- •Ability to comply with study procedures including investigational therapy and follow-up evaluations.
排除标准
- •Anti-AAV9 neutralizing antibody titer >1:
- •Severe heart failure or requirement for advanced therapies.
- •History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- •Prior cardiac or other organ (lung, liver, other) transplantation.
研究组 & 干预措施
RP-A501
Experimental
One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease.
干预措施: RP-A501 (Genetic)
结局指标
主要结局
Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
时间窗: 12 Months post-infusion
Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).
次要结局
- Evaluation of efficacy via components of the primary endpoint - LAMP2(12 months post infusion)
- Evaluation of efficacy via components of the primary endpoint - Left Ventricular Mass Index (LVMI)(12 months post infusion)
- Evaluation of efficacy via biomarker evidence of myocardial injury - High Sensitivity Troponin I (hsTnI)(12 months post infusion)
- Evaluation of efficacy via biomarker evidence of myocardial injury - N-Terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)(12 months post infusion)
- Evaluation of efficacy via assessment of event-free survival(60 months post infusion)
- Evaluation of safety(60 months post infusion)
研究者
研究点 (9)
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相关资讯
Rocket Pharmaceuticals Advances Gene Therapy Pipeline for Rare Disorders- Rocket Pharmaceuticals completed enrollment in the Phase 2 pivotal study of RP-A501 for Danon disease, with updated Phase 1 data expected at AHA 2024.
- Enrollment finished in the low dose cohort of the Phase 1 study of RP-A601 for PKP2-ACM, with preliminary data anticipated in the first half of 2025.
- The company initiated a rolling BLA submission for RP-L102 for Fanconi Anemia and anticipates FDA approval of KRESLADI for severe LAD-I in 2025.
- Rocket Pharmaceuticals reported a cash position of $235.7 million, expected to fund operations into 2026, and appointed Mikael Dolsten to its Board of Directors.last yearGene and Cell Therapies Show Promise in Treating Various Cardiac Diseases- Rocket Pharmaceuticals completed enrollment in a Phase 2 trial of RP-A501 gene therapy for Danon disease, targeting LAMP2 expression and left ventricular mass reduction.
- LX2006 gene therapy from Lexeo Therapeutics demonstrated improved biomarkers in patients with Friedreich Ataxia cardiomyopathy in early-stage trials.
- Immix Biopharma dosed the first patient in a Phase 1b/2 trial of NXC-201 CAR-T therapy for relapsed/refractory light chain amyloidosis.
- Verve Therapeutics dosed the first patient in a Phase 1b trial of VERVE-102, an in vivo base editing therapy targeting PCSK9 for heterozygous familial hypercholesterolemia.last yearRocket Pharma Completes Enrollment in Phase 2 Trial of RP-A501 for Danon Disease- Rocket Pharmaceuticals has completed enrollment in its Phase 2 pivotal trial of RP-A501 for male patients with Danon disease.
- The trial enrolled 12 patients across the U.S. and EU, evaluating RP-A501's efficacy and safety with a dose of 6.7 x 10^13 GC/kg.
- RP-A501 targets LAMP2 protein expression and left ventricular mass, with potential for accelerated approval based on biomarker endpoints.
- Rocket plans to pursue simultaneous regulatory filings in the U.S. and other regions, addressing the unmet need for Danon disease treatment.last year