Heart failure is caused by a cardiomyopathy, which is defined as a disorder of the heart muscle. Dilated cardiomyopathy (DCM) is characterised by enlargement of the ventricles and impaired systolic function of one or both ventricles in the absence of significant coronary artery disease. MedDRA version: 20.0 Level: LLT Classification code 10056419 Term: Dilated cardiomyopathy System Organ Class: 100000004849
Conditions
Interventions
Product Name: Autologous Bone Marrow Derived Mononuclear Cells
Product Code: aBMC
Pharmaceutical Form:
INN or Proposed INN: Autologous Bone Marrow Deriv
Sponsors
Queen Mary Innovation Centre, Queen Mary University of London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patients with a confirmed diagnosis of dilated cardiomyopathy under the supervision of a physician or a heart failure nurse specialist. • NYHA class III or IV symptoms despite having received optimal medical therapy and appropriate device therapy, as per clinical guidelines for an interval of at least 3 months. • No other treatment options available as part of current best standard care. • LVEF =30% on the cardiac CT scan performed as part of the screening phase. • Patient between 18-85 years old. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: • NYHA I-II. • Documented latest ejection fraction >30% (any imaging modality) • Congenital heart disease. • Clinically significant valvular heart disease. • Patients who are not suitable for a Percutaneous Mechanical Support Device (E.g. unsuitable femoral artery anatomy, unable able to lie flat for prolonged time to accommodate the stem cell infusion & presence of LV thrombus) • Weight of patient that exceeds the maximum limit of the cardiac catheter laboratory table / CT scanner. • Cardiomyopathy 2o 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 at time of randomisation. • Positive virology tests. • Chronic inflammatory disease requiring on-going medication. • Concomitant disease with a life expectancy of less than one year • Follow-up impossible (no fixed abode, etc.) • Neoplastic disease without documented remission within the past 5 years. • Patients on renal replacement therapy. • Subjects of childbearing potential unless ßHCG negative and are on adequate contraception during the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The principle research question of this trial is to determine if the injection of autologous bone marrow derived mononuclear cells (cells obtained from the patient’s own bone marrow) into the coronary arteries with co - current G-CSF and a percutaneous mechanical support device is feasible and improves left ventricular ejection fraction (pumping of the main heart chamber). The percutaneous mechanical support device is essentially a pump that is inserted into the femoral artery (the main artery in the groin) and ultimately positioned in the heart. It helps the heart pump blood around the body and takes some of the strain off the heart. Importantly, this device is removed at the end of the procedure and therefore does not remain in long-term. ; Secondary Objective: Assessment of major adverse cardiac events (MACE; death, Q wave myocardial infarction, need for repeat revascularisation) and changes in New York Heart Association (NYHA) class status. NYHA classification is a simple way of assessing patient’s heart failure symptoms. It places patients into one of four categories (I-IV). ;Primary end point(s): Change in Left Ventricular Ejection Fraction as measured with advanced cardiac imaging (Cardiac CT) at 3 months. Although this is a feasibility study with no control group, a comparator group is available from our recently published REGENERATE DCM Support Trial. ;Timepoint(s) of evaluation of this end point: The primary end point will be assessed at 3 months. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: The secondary end points will be assessed at 12 months. ; Secondary end point(s): • Change in LVEF from baseline measured by advanced cardiac imaging (cardiac CT) at 12 months. • Procedural safety as assessed by in hospital procedural related morbidity/mortality. • Change in NT-proBNP, Troponin T, renal function and inflammatory profile at 3 and 12 months. • Assessment of MACE endpoints at 3 months and 12 months. • Assessment of arrhythmia burden through follow up. • Change in exercise capacity and NYHA class at 3 months and 12 months as assessed by 6 minute walk test. | — |
Countries
United Kingdom
Contacts
Public ContactProfessor Anthony Mathur
Cardiac Research Department (Bart's Health NHS Trust)
Outcome results
None listed