A PILOT TRIAL OF CELL THERAPY IN HEART FAILURE WITH PRESERVED EJECTION FRACTION
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Change in left ventricular filling pressures (E/e') assessed by echocardiography
研究概览
简要总结
The primary objective of the study is to investigate safety and efficacy of transendocardial CD34+ cell therapy in patients with HFpEF by evaluating changes in myocardial structure and function, patient exercise capacity and clinical outcome.
The safety end-points include serious adverse events (SAEs), defined as any serious event that may result in persistent or significant disability or incapacity and included death, heart transplantation, sustained ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation), and heart failure exacerbation requiring hospitalization.
详细描述
In all patients, peripheral blood stem cells will be mobilized by daily subcutaneous injections of G-CSF (10 mcg/kg, divided b.i.d) for 5 days. Peripheral blood stem cells will then be collected with Miltenyi cell separator (Miltenyi Biotech, Germany) and the magnetic cell separator Isolex 300i (Nexell Therapeutics Inc., California, USA) will be used for the immunomagnetic positive selection of the CD34+ cells.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
盲法说明
The echocardiographer will be blinded for the timing of echocardiographic recordings.
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
- •Preserved left ventricular systolic function on echocardiography (LVEF>50%)
- •Evidence of diastolic dysfunction by echocardiography (E/e'>15)
- •Symptoms of heart failure
- •NT-proBNP levels >300 pg/ml
- •absence of permanent atrial fibrillation
排除标准
- •acute multi-organ failure
- •history of any malignant disease within 5 years
- •diminished functional capacity due to non-cardiac co-morbidities (COPD, PAOD, morbid obesity)
- •pregnancy
研究组 & 干预措施
SC Group
In Phase 1 of the study, all patients will be treated with optimization of medical therapy for 6 months. Thereafter, all patients will cros over to Phase 2 of the study, where they will receive transendocardial CD34+ cell therapy. Follow-up of Phase 2 will last for 6 months. At the time of enrollment (6 months before cell therapy), at time of cell therapy, and 6 months thereafter we will perform detailed clinical evaluation, laboratory assays, echocardiography, 6-minute walk test, and measure plasma levels of N-terminal pro B-type natriuretic peptide (NT-proBNP).
干预措施: Cell Therapy (Biological)
结局指标
主要结局
Change in left ventricular filling pressures (E/e') assessed by echocardiography
时间窗: Baseline, 6 months and 1 year
The E/e' ratio will be calculated from early transmitral velocity divided by peak left ventricular relaxation velocity for estimation of the left ventricular filling pressure.
次要结局
- Change in diastolic dysfunction grade(Baseline, 6 months and 1 year)
- Change in exercise capacity(Baseline, 6 months and 1 year)
- Change in NT-proBNP levels(Baseline, 6 months and 1 year)
- Change in systolic strain(Baseline, 6 months and 1 year)
研究者
Bojan Vrtovec
Medical Director, Advanced Heart Failure and Transplantation Programme
University Medical Centre Ljubljana