DCM II Trial
A Phase IIB Randomized, Placebo-Controlled, Multicenter Study of the Comparative Efficacy and Safety of Transendocardial Injection of Allogeneic-MSC versus Placebo in Patients with Non-Ischemic Dilated Cardiomyopathy
What is DCM?
Cardiomyopathy is a medical term meaning, ‘heart muscle disease’. Dilated cardiomyopathy (DCM) is the most common type and is classified as either Ischemic (lack of blood supply) or Non-Ischemic. In patients with Non-Ischemic Dilated Cardiomyopathy (NIDCM), the heart becomes enlarged and cannot pump blood effectively1. As the heart stretches, the walls of the heart chambers become thinner and weaker, which over time can lead to heart failure and death. Common symptoms include shortness of breath, fatigue and swelling of the ankles, feet, legs, abdomen and veins in the neck2. DCM also can lead to other problems such as heart valve disease, arrhythmias (irregular heartbeats) and blood clots in the heart1. DCM occurs mostly in adults age 20 to 60, is more common in men, and accounts for 10,000 deaths and 46,000 hospitalizations in the United States annually3.
Oftentimes the cause of DCM is unknown, but one-third of patients with DCM inherit it from their parents2. Other causes include alcohol and drug abuse, viral infection of the heart, after pregnancy, side-effect of cancer chemotherapy or radiation, and diseases of the endocrine system (thyroid, adrenal glands).
Treatments currently available for DCM include medications to reduce stress on the heart, implantable devices, a heart transplant4.
3 - CDC website
Non-Ischemic Dilated Cardiomyopathy (DCM) is a common and problematic condition; definitive therapy in the form of heart transplantation is available to only a small minority of eligible patients. Cellular therapy for chronic heart failure represents a potentially important alternative for this disease.
Stem cells are cells that do not yet have a specific function in the body. Human mesenchymal stem cells (hMSCs) are a type of stem cell that can be grown from human bone marrow. Stem cells can develop into other types of more mature cells, such as blood and muscle cells. When cells are taken from a healthy donor (who is not the patient) it is called allogeneic (or allo for short). It is hoped that by placing these cells in the heart, they will allow the heart to work better.
This research study is being conducted to determine whether giving allo hMSCs to patients with heart muscle damage is safe. We will also examine whether this study drug improves heart function and if an individual’s genotype (genetics) plays a role in determining his/her response to allo hMSC therapy.
136 subjects are expected to participate in this study at 4 research sites across the United States. They will be randomized (like flipping a coin) to receive allo hMSCs or placebo. Study products are administered during a cardiac catherization requiring an overnight stay in the hospital.
Follow-up visits occur at the following timepoints after study product administration: 1 week, 1 month, 3 months, 6 months, and 12 months.
See additional information regarding this trial at www.clinicaltrials.gov.