Active clinical trials for "Cardiomyopathy, Hypertrophic"

Human induced pluripotent stem cells (hiPSCs) have driven a paradigm shift in the modeling of human disease; the ability to reprogram patient-specific cells holds the promise of an enhanced understanding of disease mechanisms and phenotypic variability, with applications in personalized predictive pharmacology/toxicology, cell therapy and regenerative medicine. This research will collect blood or skin biopsies from patients and healthy controls for the purpose of generating cell and tissue models of Mendelian heritable forms of heart disease focusing on cardiomyopathies, channelopathies and neuromuscular diseases. Cardiomyocytes derived from hiPSCs will provide a ready source of disease specific cells to study pathogenesis and therapeutics.

To demonstrate the efficacy of ranolazine in improving coronary microvascular and diastolic dysfunction in patients affected by HCM evaluating changes in maximum (i.e. during dipyridamole-induced coronary vasodilatation) myocardial blood flow (MBF) measured by PET at baseline and after 4 months of treatment with ranolazine in patients with non obstructive HCM.

The overall aim of this project is to establish potential benefits of a novel lifestyle (physical activity and dietary nitrate) and pharmacological (angiotensin receptor neprilysin inhibitor) interventions in patients with hypertrophic cardiomyopathy (HCM). HCM is the most common genetic cardiovascular disease with a broad spectrum of disease severity. Angiotensin receptor neprilysin inhibitor reduces death, hospitalisation, and may improve cardiac function and exercise tolerance in heart failure. Exercise training is associated with a significant increase in exercise tolerance, but appear to have limited effect on measures of cardiac morphology or function in patients with HCM. Dietary supplementation with inorganic nitrate (i.e. concentrated nitrate-rich beetroot juice) improves exercise capacity, vasodilatation and cardiac output reserves while reduces arterial wave reflections, which are linked to left ventricular diastolic dysfunction and remodelling. Using a five-centre, open label, three-arm, pilot design, the present study will evaluate the effect of lifestyle (physical activity and dietary supplementation with inorganic nitrate) and pharmacological (angiotensin receptor neprilysin inhibitor sacubitril / valsartan) interventions in patients with HCM. The Aim is to examine whether these interventions improve functional capacity, clinical phenotypic characteristics, and quality of life in patients with HCM.

The purpose of the sudy is to conduct a small study to gather the preliminary data for future lage scale clinical studies that will be designed test the potential beneficial effect of over-the counter study anti-oxidant drug called N-acetylcysteine (NAC) in patients with a heart muscle condition called Hypertrophic Cardiomyopathy (HCM). The present study is a pilot feasibility study, the investigators want to find out whether the investigators can recruit and retain patients with HCM in the study and whether these patients can tolerate this drug and can stay on one year. Likewise, the investigators want to find out any potential side effects that this drug might have and estimate whether it has any beneficial effects.

Paroxysmal or chronic atrial fibrillation (AF) develops in about 20- 25% of adult patients with hypertrophic cardiomyopathy (HCM) and represents an important complication in the clinical course of the disease, with adverse long-term consequences on functional status and outcome. Therefore, aggressive therapeutic strategies are indicated to restore and maintain sinus rhythm (SR) in patients with HCM. Nevertheless, pharmacologic prevention of AF recurrence is challenging because of the limited long-term efficacy and potentially hazardous side effects of available treatment options. Currently radiofrequency catheter ablation (RFCA) of AF is successfully used in clinical practice. However, comparison of the efficacy and safety of these two therapeutic options has not been done up till now in randomized manner in this group of patients. Thus, the aim of the present study is to compare the efficacy and safety of RFCA vs. antiarrhythmic drug therapy in patients with HCM and AF.

This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty properly. This can lead to heart failure, breathlessness and excessive fatigue. The heart's inability to relax may be due to scarring, or fibrosis, in the muscle wall. This study will test whether pirfenidone can reduce fibrosis, improve heart relaxation and reduce abnormal heart rhythms. Men and women 20 to 75 years old with HCM may be eligible for this study. Participants will undergo a physical examination, blood tests, and other tests and procedures, described below, to assess heart function. When the tests are completed, patients will be randomly assigned to one of two treatment groups. One group will take a pirfenidone capsule and the other will take a placebo (a look-alike pill with no active ingredient) twice a day with meals for 6 months. For the pirfenidone group, the dose of drug will be increased gradually from 400 to 800 milligrams. At the end of 6 months, all patients will repeat the physical examination and heart tests that were done before starting medication. These include: Electrocardiogram (ECG) - electrodes are attached to the heart to record the heart's electrical activity, providing information on the heartbeat. Echocardiogram - a probe held against the chest wall uses sound waves to produce images of the heart, providing information on the function of the heart chambers. 24-hour Holter monitor - a 24-hour recording of the electrical activity of the heart monitors for abnormal heartbeats or conduction abnormalities. Magnetic resonance imaging (MRI) - Radiowaves and a strong magnetic field are used to produce images of the heart, providing information on the thickness and movement of the heart muscle. Radionuclide angiogram - a radioactive tracer is injected into a vein and a special camera is used to scan the heart, providing information on the beating motion of the heart. Scans are obtained at rest and after exercise. Cardiac (heart) catheterization - a catheter (thin plastic tube) is inserted into a blood vessel in the groin and advanced to the heart to record pressures and take pictures inside the heart. Electrophysiology study - a catheter is inserted into a blood vessel in the groin and advanced to the heart to record electrical activity, providing information on abnormal heart rhythms. This procedure is done at the time of the heart catheterization. Cardiac biopsy - a catheter is inserted into a blood vessel in the groin and advanced to the heart to remove a small sample of heart muscle for microscopic examination. This procedure is done at the end of the heart catheterization.

To assess the safety and effectiveness of the SAPIEN XT transcatheter heart valve in patients with a failing surgical aortic bioprosthetic valve.

The purpose of this phase 2 open-label pilot study is to evaluate the efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of MYK-461 in subjects with symptomatic HCM and LVOT obstruction aged 18-70 years.

The purpose of this trial is to determine whether treatment with valsartan will have beneficial effect in early hypertrophic cardiomyopathy (HCM) by assessing many domains that reflect myocardial structure, function and biochemistry.

A heart condition called hypertrophic cardiomyopathy (HCM) causes abnormal thickening of the heart muscle, which obstructs the flow of blood out of the heart. The thickened muscle and the obstruction of blood flow are believed to cause chest discomfort, breathlessness, fainting, and a sensation of heart pounding. Treatment options for children with HCM include medicine, heart operation, and cardiac transplantation. However, there is no evidence that medicine prevents further thickening of heart muscle; operations carry the risk of death; and donor hearts are not always available. Several studies have shown that pacemaker treatment reduces the obstruction and improves heart complaints in patients with HCM. This study investigates further the efficacy of pacemaker treatment in children. Patients will have exercise tests after treatment with beta blocker and verapamil and will be eligible for the study if heart complaints or reduced exercise performance continue. A pacemaker that treats slow heart rhythms will be inserted. The patient will be sedated and local anesthesia will be administered to numb the area. The procedure takes about an hour. The study will last two years. Patients will be placed on one of two pacemaker programs for the first year and another the second year. At 3- and 6-month follow-up visits, a pacemaker check and echocardiogram will be performed. After 1 year, patients will be admitted to NIH for 2 to 3 days for exercise tests, echocardiogram, and cardiac catheterization. Also, the pacemaker will be changed to the second program. At 15- and 18-month follow-up visits, a pacemaker check and echocardiogram will be performed. After 2 years, patients will again be admitted for 2 to 3 days for exercise tests, echocardiogram, and cardiac catheterization. A pregnancy test will be given to females of child-bearing age before each cardiac catheterization and electrophysiology study. At the end of the study, the pacemaker will be set to the program that worked better. Risks of pacemaker insertion include lung collapse, infection, blood vessel damage, bleeding, heart attack, and death. Risks of cardiac catheterization include infection, bleeding, blood clots, abnormal heart rhythms, perforation of the heart, need for surgery, and death. However, the safety record for both these procedures at NIH has been excellent. The radiation exposure exceeds the NIH radiation guidelines for children, but this exposure in adults has not been associated with any definite adverse effects.