CTIS2023-506480-34-00
招募中
1 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 - RP-A501-0123
概览
- 阶段
- 1 期
- 干预措施
- 未指定
- 疾病 / 适应症
- 未指定
- 发起方
- Rocket Pharmaceuticals Inc.
- 入组人数
- 12
- 状态
- 招募中
- 最后更新
- 去年
概览
简要总结
暂无简介。
研究者
入排标准
入选标准
- •Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene., Male gender., Age \=8 years., Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: a. For subjects \< 18 years, z\-score of the left ventricular posterior wall or interventricular septum at end diastole \=\+ 2, and for subjects \=18 years, left ventricular posterior wall or interventricular septum at end diastole \>13 mm (\>12 mm if family history of clinically significant Danon disease), b. Left ventricular ejection fraction (LVEF) \= 50%., New York Heart Association (NYHA) Class II to III., hsTnI \=20% above the ULN, Ability to comply with study procedures including investigational therapy and follow\-up evaluations.
排除标准
- •Anti\-AAV9 neutralizing antibody titer \>1:40\., Intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment., Presence or requirement for mechanical circulatory support (MCS)., Presence or requirement for mechanical ventilation., 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.
结局指标
主要结局
未指定
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