Advertisement

Topics

Cyclosporine A to Treat Hypertrophic Cardiomyopathy (HCM)

2014-08-27 03:59:19 | BioPortfolio

Summary

This study will examine the effectiveness of the drug cyclosporine in treating hypertrophic cardiomyopathy (HCM), a condition in which the heart muscle thickens. The thickened muscle can impair the heart's pumping action or decrease its blood supply, or both. Various symptoms, such as chest pain, shortness of breath, fatigue, and palpitations, may result. In animal studies, cyclosporine prevented heart muscle from thickening in mice that had been engineered to develop thick hearts.

Patients with HCM 18 to 75 years old are screened for this study under protocol 98-H-0102 and this protocol. Screening tests include blood tests, echocardiogram to measure heart thickness, Holter monitor to record heartbeats, treadmill exercise test, and various imaging tests including a thallium scan, radionuclide angiography, magnetic resonance imaging (MRI), and cardiac catheterization to examine heart function and blood supply.

Patients admitted to the study will be randomly assigned to take either cyclosporine tablets or a placebo (a look-alike tablet with no active ingredient) twice a day for 6 months. During a brief hospital stay at the start of the study, blood samples will be taken to measure cyclosporine levels. After discharge, heart rate and blood pressure will be checked and blood tests done during follow-up visits once a week for 2 weeks and then every two weeks until the end of the 6-month treatment period. At that time, patients will be hospitalized a second time for repeat tests to determine the effects of the drug on the heart condition. They include thallium scan, radionuclide angiogram, MRI, treadmill exercise test, cardiac catheterization, and echocardiogram. An echocardiogram and MRI will be repeated 1 year after the start of the study to evaluate long term effects of the drug, if any.

Description

Hypertrophic cardiomyopathy (HCM) is a genetic cardiac disease characterized by marked increase in cardiac mass caused by proliferation/hypertrophy of several cell types (myocytes, fibroblasts, smooth muscle cells, and endothelial cells). There is often associated left ventricular (LV) diastolic dysfunction and myocardial ischemia. The severity of the LV hypertrophy, diastolic dysfunction, and myocardial ischemia are important determinants of clinical course. In several animal models of LV hypertrophy, calcineurin has been implicated in the development of myocardial hypertrophy, leading to cardiac dilatation and failure. Inhibitors of calcineurin (Cyclosporin A and FK506) have been shown to prevent the development of cardiac hypertrophy in these animal models, where cardiac hypertrophy is related to sarcomeric dysfunction. We propose to study the ability of Cyclosporin A (CsA) to reduce LV mass, and to improve symptoms, LV diastolic function, and myocardial perfusion in HCM caused by sarcomeric gene mutations.

Study Design

Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment

Conditions

Cardiomyopathy, Hypertrophic

Intervention

Cyclosporine A

Location

National Heart, Lung and Blood Institute (NHLBI)
Bethesda
Maryland
United States
20892

Status

Completed

Source

National Institutes of Health Clinical Center (CC)

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:59:19-0400

Clinical Trials [504 Associated Clinical Trials listed on BioPortfolio]

Sleep Apnea and Hypertrophic Cardiomyopathy (HCM)

The investigators are trying to find out how common sleep apnea is in hypertrophic cardiomyopathy. The purpose of this study is to see if sleep apnea is common in hypertrophic cardiomyopat...

Evaluating Microvascular Dysfunction in Symptomatic Patients With HypertroPhic CaRdiomyopathy

Patients with hypertrophic cardiomyopathy are being compared to a control group. IMR will be assessed with a pressure wire. Clinical f/u at 3 months and 6 months and a 48 hour holter monit...

Multicenter Exploratory Study of Accelerometry in Hypertrophic Cardiomyopathy

Study evaluate the relationships between daily physical activity levels (PAL) and functional capacity (VO2peak) in patients with hypertrophic cardiomyopathy (HCM)

Hypertrophic Cardiomyopathy Pilot Study

This study evaluates mechanisms of arrhythmogenicity in hypertrophic cardiomyopathy, in comparison to patients with well-understood arrhythmogenic substrate (ischemic cardiomyopathy), as w...

The Chinese Hypertrophic Cardiomyopathy Study(CHCS)

Hypertrophic cardiomyopathy (HCM) is one of the most common inherited cardiac diseases, with a prevalence of ∼0.2%. The disease affects all age groups, with marked clinical heterogeneity...

PubMed Articles [713 Associated PubMed Articles listed on BioPortfolio]

Genetic Pathogenesis of Hypertrophic and Dilated Cardiomyopathy.

Sarcomere cardiomyopathies are genetic diseases that perturb contractile function and lead to hypertrophic or dilated myocardial remodeling. Identification of preclinical mutation carriers has yielded...

E/e' ratio and left atrial area are predictors of atrial fibrillation in patients with hypertrophic cardiomyopathy.

Atrial fibrillation (AF) occurs in about 20%-25% of patients with hypertrophic cardiomyopathy and is associated with increased risk of cardioembolism and heart failure impacting on patients' morbidity...

Left ventricular apical aneurysm resection in hypertrophic cardiomyopathy.

Apical aneurysms are outpouchings of the left ventricular apex that are relatively common in patients with apical hypertrophic cardiomyopathy or hypertrophic cardiomyopathy with midventricular obstruc...

Effect of Spironolactone on Myocardial Fibrosis and Other Clinical Variables in Patients with Hypertrophic Cardiomyopathy: a Prospective, Randomized Trial.

Myocardial fibrosis has proved to be an important marker and determinant in the pathogenesis of hypertrophic cardiomyopathy . In particular, scar formation, if substantial, can promote ventricular tac...

Clinical Presentation and Natural History of Hypertrophic Cardiomyopathy in RASopathies.

RASopathies are a heterogeneous group of genetic syndromes characterized by mutations in genes that regulate cellular processes, including proliferation, differentiation, survival, migration, and meta...

Medical and Biotech [MESH] Definitions

An autosomal dominant inherited form of HYPERTROPHIC CARDIOMYOPATHY. It results from any of more than 50 mutations involving genes encoding contractile proteins such as VENTRICULAR MYOSINS; cardiac TROPONIN T; ALPHA-TROPOMYOSIN.

A group of diseases in which the dominant feature is the involvement of the CARDIAC MUSCLE itself. Cardiomyopathies are classified according to their predominant pathophysiological features (DILATED CARDIOMYOPATHY; HYPERTROPHIC CARDIOMYOPATHY; RESTRICTIVE CARDIOMYOPATHY) or their etiological/pathological factors (CARDIOMYOPATHY, ALCOHOLIC; ENDOCARDIAL FIBROELASTOSIS).

A form of CARDIAC MUSCLE disease, characterized by left and/or right ventricular hypertrophy (HYPERTROPHY, LEFT VENTRICULAR; HYPERTROPHY, RIGHT VENTRICULAR), frequent asymmetrical involvement of the HEART SEPTUM, and normal or reduced left ventricular volume. Risk factors include HYPERTENSION; AORTIC STENOSIS; and gene MUTATION; (FAMILIAL HYPERTROPHIC CARDIOMYOPATHY).

An autosomal recessively inherited glycogen storage disease caused by GLUCAN 1,4-ALPHA-GLUCOSIDASE deficiency. Large amounts of GLYCOGEN accumulate in the LYSOSOMES of skeletal muscle (MUSCLE, SKELETAL); HEART; LIVER; SPINAL CORD; and BRAIN. Three forms have been described: infantile, childhood, and adult. The infantile form is fatal in infancy and presents with hypotonia and a hypertrophic cardiomyopathy (CARDIOMYOPATHY, HYPERTROPHIC). The childhood form usually presents in the second year of life with proximal weakness and respiratory symptoms. The adult form consists of a slowly progressive proximal myopathy. (From Muscle Nerve 1995;3:S61-9; Menkes, Textbook of Child Neurology, 5th ed, pp73-4)

Isoforms of MYOSIN TYPE II, specifically found in the ventricular muscle of the HEART. Defects in the genes encoding ventricular myosins result in FAMILIAL HYPERTROPHIC CARDIOMYOPATHY.

More From BioPortfolio on "Cyclosporine A to Treat Hypertrophic Cardiomyopathy (HCM)"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topics

Drug Discovery
Clinical Approvals Clinical Trials Drug Approvals Drug Delivery Drug Discovery Generics Drugs Prescription Drugs In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which drugs are dis...

Cardiology
Cardiology is a specialty of internal medicine.  Cardiac electrophysiology : Study of the electrical properties and conduction diseases of the heart. Echocardiography : The use of ultrasound to study the mechanical function/physics of the h...


Searches Linking to this Trial