Randomised Controlled Trial to Compare the Effects of G-CSF (Granocyte™) and Autologous Bone Marrow Progenitor Cells on Quality of Life and Left Ventricular Function in Patients With Idiopathic Dilated Cardiomyopathy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography
研究概览
简要总结
A randomised, double-blind, placebo-controlled trial to evaluate the role of intracoronary injection of progenitor cells compared to placebo injection in patients with Dilated Cardiomyopathy who have been pre-treated with G-CSF (Granocyte™) injections for 5 days, and patients treated with a 5 day course of G-CSF (Granocyte™) injection only compared to placebo injection
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Symptomatic patients with a confirmed diagnosis of dilated cardiomyopathy (NYHA II-III) attending their local 'Heart Failure clinic' who are on optimal heart failure treatment, under supervision from their physician or heart failure nurse specialist, and have no other treatment options
- •Patients who are NYHA II that have been hospitalised with a dilated cardiomyopathy related condition
- •Coronary angiography will be performed where necessary to confirm the diagnosis and ensure no other conventional treatment options are indicated
- •Prior to recruitment to the study patients at risk of ventricular arrhythmia will have undergone electrophysiological assessment and appropriate clinical management (including implantable defibrillator insertion) where indicated (as per NICE guidelines)
排除标准
- •Referral hospitals most recent documented ejection fraction of >45% (any imaging modality)
- •The presence of cardiogenic shock
- •The presence of acute left and/or right sided pump failure as judged by the presence of pulmonary oedema and/or new peripheral oedema
- •Known severe pre-existent left ventricular dysfunction (with a documented ejection fraction of <10% from referral hospital) prior to randomisation
- •Congenital cardiac disease
- •Cardiomyopathy secondary to 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
- •Contra-indication for bone marrow aspiration
- •Known active infection
- •Known infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), HTLV or syphilis.
- •Chronic inflammatory disease requiring ongoing medication
- •Serious known concomitant disease with a life expectancy of less than one year
- •Follow-up impossible (no fixed abode, etc)
- •Patients with an irregular heart rhythm (AF allowed if paced in a regular rhythm)
- •Patients with renal impairment (Creatinine >200mmol/L)
- •Neoplastic disease without documented remission within the past 5 years
- •Weight>140kg
- •Subjects of childbearing potential
研究组 & 干预措施
Peripheral
Half the patients will be randomised to the non-interventional part of the trial. In this subgroup of patients will be randomised 1:1 to 5 day course of subcutaneous placebo injections or a 5 day course of G-CSF(Granocyte™) subcutaneous injections
干预措施: granulocyte colony stimulating factor (GCSF) (Drug)
Interventional arm
In the subgroup of the interventional arm patients will be randomised 1:1 to receive a 5 day course of subcutaneous G-CSF (Granocyte™) injections and bone marrow aspiration at day 5, they will then receive either stem cells or placebo via intracoronary injection
干预措施: bone marrow mononuclear cells (Procedure)
结局指标
主要结局
Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography
时间窗: 3 months
次要结局
- Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)(3 months and 12 months)
- Changes in V02 max (exercise capacity)(3 months and 12 months)
- Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography(3 months and 12 months)
- The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death(3 months and 12 months)
- Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires(3 months and 12 months)
- Occurrence of a Major Adverse Cardiac Event (MACE) defined as cardiac death, myocardial infarction (CK / CK-MB over 2 times the upper limit of normal)(3 months and 12 months)
- Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death(3 months and 12 months)
研究者
Anthony Mathur
Clinical and Research Lead (Cardiology)
Barts & The London NHS Trust