NCT03797092Unknown1 期
Stem Cell Therapy in Non-IschEmic Non-treatable Dilated CardiomyopathiEs II: a Pilot Study
JKastrup2 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2019年10月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Left ventricle end-systolic volume
研究概览
简要总结
The overall aim of the project is to test the feasibility and safety of allogeneic adipose-derived stromal cells (CSCC_ASC) investigational medicinal product, to improve myocardial function in patients with non-ischemic dilated cardiomyopathies (NIDCM) and heart failure.
详细描述
Study design
The primary objective of the study is to investigate safety and regenerative capacity of direct intra-myocardial injection of 100 million allogeneic CSCC_ASCs in NIDCM patients with reduced left ventricular EF (≤ 40%) and heart failure.
It is a proof of concept study enrolling a total of 30 NIDCM patients with heart failure who will be randomly allocated in a 2:1 ratio to either CSCC_ASC cell therapy (Stem Cell Group) or no cell therapy (Control Group). The treatment period is estimated to be 6 months (efficacy end-point) with a 12-month follow-up period for safety end-points.
Patient treatment and follow-up
- The cell IMP will be delivered by a courier service to from REGIONH to UKCL using validated portable dry liquid nitrogen shipping containers. It will then be stored in nitrogen vapour containers until treatment.
- The preparation of the IMP will be performed as described in the treatment manual. The IMP will be thawed and prepared for injection immediately before treatment.
- A 3-D map of the left ventricle will be created using the NOGA XP® system (Biological Delivery System, Cordis, Johnson & Johnson, USA). The delivery of the IMP (100 million ASCs) into the myocardium will be performed by 10-15 injections of 0.2 cc, as described in the treatment manual.
- The post cell therapy surveillance will include clinical and laboratory safety follow-up with monitoring of cardiac enzymes, hemodynamic and rhythm stability. The clinical follow-up will be obtained at pre-defined time points. The presence of allogeneic HLA anti-bodies will be monitored three and six months after treatment. End-points will be monitored continuously and reported as occurring throughout the 12 months follow-up period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The patients will be randomized to either IMP or control in a 2:1 randomization.
The outcome ECHO investigations will be analyzed blinded by an independent core lab.
入排标准
- 年龄范围
- 30 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •30 to 80 years of age
- •Signed informed consent
- •Patients with non-ischemic dilated cardiomyopathy
- •NYHA ≥ II in spite of optimal heart failure treatment and have no other treatment options
- •Heart failure medication unchanged two months prior to inclusion/signature of informed consent. Changes in diuretics accepted
- •LVEF ≤ 405%
- •Plasma NT-pro-BNP > 300 pg/ml (> 35 pmol/L)
- •Patients cannot be included until three months after implantation of a cardiac resynchronisation therapy device (CRTD) and until 1 month after an ICD unit
排除标准
- •Heart Failure NYHA I
- •Moderate to severe aortic stenosis (valve area < 1.3 cm2) or valvular disease with option for surgery or interventional therapy.
- •Heart failure caused by cardiac valve disease or untreated hypertension.
- •If the patient is expected to be candidate for MitraClip therapy of mitral regurgitation in the 12 months follow-up period.
- •Cardiomyopathy with a reversible cause that has not been treated e.g. thyroid disease, alcohol abuse, hypophosphataemia, hypocalcaemia, cocaine abuse, selenium toxicity & chronic uncontrolled tachycardia
- •Cardiomyopathy in association with a neuromuscular disorder e.g. Duchenne's progressive muscular dystrophy
- •Previous cardiac surgery
- •Diminished functional capacity for other reasons such as: obstructive pulmonary disease (COPD) with forced expiratory volume (FEV) <1 L/min, moderate to severe claudication or morbid obesity
- •Clinical significant anaemia (haemoglobin < 6 mmol/L), leukopenia (leucocytes < 2 109/L), leucocytosis (leucocytes > 14 109/L) or thrombocytopenia (thrombocytes < 50 109/L)
- •Reduced kidney function (eGFR < 30 ml/min)
- •Left ventricular thrombus
- •Anticoagulation treatment that cannot be paused during cell injections.
- •Patients with reduced immune response
- •History with malignant disease within five years of inclusion or suspected malignity - except treated skin cancer other than melanoma
- •Pregnant women
- •Woman of childbearing potential unless βHCG negative and they should be on contraception during the trial
- •Other experimental treatment within four weeks of baseline tests
- •Participation in another intervention trial
- •Life expectancy less than one year
结局指标
主要结局
Left ventricle end-systolic volume
时间窗: 6 months after treatment
Measured using echocardiography
次要结局
- 6 min walking test(6 months)
- Kansas City Cardiomyopathy Questionnaire(6 months after treatment)
- EQ-5D3L Questionnaire(6 months after treatment)
- NYHA(6 months after treatment)
- Allogeneic antibodies(Up to 12 months after treatment)
- Left ventricular ejection fraction(6 months after treatment)
- Myocardial mass of left ventricle(6 months after treatment)
研究者
JKastrup
Professor, Doctor
Rigshospitalet, Denmark
研究点 (2)
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