Primary Idiopathic Dilated Cardiomyopathy Clinical Trial
Official title:
Phase I/II Randomized Clinical Trial to Assess the Safety and Feasibility of Transendocardial Injection of Bone Marrow Autologous Mesenchymal Stem Cells in Patients With Idiopathic Dilated Cardiomyopathy.
The purpose of this study is to assess the safety, the feasibility and the efficacy of transendocardial injection of bone marrow-derived mesenchymal stem cells (MSCs) in patients with dilated idiopathic cardiomyopathy.
Chronic congestive heart failure (CHF) is a public health problem that entails high rates of
morbidity and mortality, and enormous costs for health systems worldwide. In the United
States there are 5 million people living with CHF, and each year 60.000 people reach
terminal phases of the disease, with mortality rates of 70-80% at two years. Although the
first cause of CHF in developed countries is atherosclerotic coronary artery disease (CAD),
dilated idiopathic cardiomyopathy (DCM) represents almost half of the cases of newly
diagnosed CHF. Treatment of CHF includes pharmacological and non-pharmacological strategies,
including implantable cardioverter defibrillators, cardiac resynchronization therapy and
heart transplantation. Despite all these advances, CHF prognosis remains poor. Cardiac stem
cell therapy emerged more than ten years ago as a new hope for CHF patients.
Although the most extensive evidence of the benefits of stem cell therapy for cardiovascular
diseases refers to ischemic heart disease (CAD), initial experiences with stem cells for
other conditions such as DCM are encouraging.
This randomized clinical trial will include 70 patients with DCM, left ventricular ejection
fraction (LVEF) between 20% and 45%, and who are symptomatic in New York Heart Association
(NYHA) functional class II-III/IV. In a first-in-man pilot phase, 10 patients will be
treated with transendocardial injections of bone marrow-derived MSCs after cardiac
catheterization and NOGA XPTM mapping of the left ventricle. A Data and Safety Monitoring
Board (DSMB) will analyse the safety and feasibility of this first phase of the trial, and
then 60 patients more will be randomized to receive MSCs or placebo (ratio 3:1).
Primary objectives include safety and feasibility variables, and secondary objectives
include efficacy variables. All patients will be studied with a complete cardiac imaging
protocol that includes: electrocardiography, echocardiography, treadmill tests with oxygen
consumption, holter, laboratory analyses, magnetic resonance imaging (MRI), single photon
emission computed tomography (SPECT), electromechanical mapping (NOGA XPTM) and quality of
life questionnaires.
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment