Clinical trial NCT01302171: Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dilated Cardiomyopathy
Keywords: dilated cardiomyopathy
adult stem cells
bone marrow progenitor cells
bone marrow stem cells
autologous
granulocyte-colony stimulating factor
intracoronary injection
left ventricular function
To determine if patient's own bone marrow derived stem cells can be used to improve cardiac function
Lead sponsor: Barts & The London NHS Trust
Sponsor class: OTHER
Collaborators: name: Royal Brompton & Harefield NHS Foundation Trust
name: University College London Hospitals
First posted: 2011-02-24
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: Inclusion Criteria:

* 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)

Exclusion Criteria:

* NYHA I
* 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

Primary outcomes:
measure: Change in left ventricular ejection fraction as measured by cardiac magnetic resonance imaging or computerised tomography
timeFrame: 3 months
Secondary outcomes:
measure: Change in: Concentrations of N-terminal prohormone of brain natriuretic peptide (cardiac enzyme)
timeFrame: 3 months and 12 months
measure: Changes in V02 max (exercise capacity)
timeFrame: 3 months and 12 months
measure: Changes in left ventricular ejection fraction, ventricular dimensions as measured by cardiac magnetic resonance imaging or computerised tomography
timeFrame: 3 months and 12 months
measure: Functional class changes according to NYHA and quality of life (QoL - EQ-5D & Kansas City) questionnaires
timeFrame: 3 months and 12 months
measure: 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)
timeFrame: 3 months and 12 months
measure: Hospitalization for Heart failure & the occurrence of major arrhythmias defined as symptomatic ventricular tachycardia or survived sudden death
timeFrame: 3 months and 12 months
measure: The occurrence of major arrhythmias defined by symptomatic ventricular tachycardia or survived sudden death
timeFrame: 3 months and 12 months
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: London Chest Hospital
Study officials:
affiliation: Barts & The London NHS Trust
name: Anthony Mathur, MD FRCP FESC
role: PRINCIPAL_INVESTIGATOR
References:
citation: Arnous S, Mozid A, Mathur A. The Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy (REGENERATE-DCM) trial: study design. Regen Med. 2011 Jul;6(4):525-33. doi: 10.2217/rme.11.29.