Double Blind Placebo Controlled Study of Cyclosporin A in Patients With Left Ventricular Hypertrophy Caused by Sarcomeric Gene Mutations
Cyclosporine A
Enfermedad de la Válvula Aórtica+8
+ Estenosis Aórtica Subvalvular
+ Estenosis de la válvula aórtica
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de diciembre de 1999
Fecha en la que se inscribió al primer participante.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.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 32 pacientes
Número total de participantes que el ensayo clínico espera reclutar.Estudio de Tratamiento
Elegibilidad
Los investigadores buscan pacientes que cumplan ciertos criterios, conocidos como criterios de elegibilidad: estado general de salud o tratamientos previos.Cualquier sexo
Sexo biológico de los participantes elegibles para inscribirse.Voluntarios sanos no permitidos
Indica si personas sanas, sin la condición que se estudia, pueden participar.Condiciones
Patología
Criterios
Patients of either gender, aged 18-75 years, with HCM caused by sarcomeric gene mutations determined by existing protocols. LV wall thickness of greater than or equal to 20 mm measured in any LV segment by MRI. Severe symptoms refractory to medical treatment (New York Heart Association functional class III or IV). No LV outflow tract obstruction at rest greater than 30 mm Hg as determined by cardiac catheterization. No coronary artery disease (greater than 50% arterial luminal narrowing of a major epicardial vessel). No chronic atrial fibrillation. No bleeding disorder (PTT greater than 35 sec, pro time greater than 14 sec, platelet count less than 154 k/mm(3). No anemia (Hb less than 12.7 g/dl in males and less than 11.0 g/dl in females). No renal impairment (serum creatinine greater than 1.3 mg/dl). No hepatitis B or C; nor unexplained abnormal LFTs. No inability to estimate LV wall thickness. No positive urine pregnancy test. No pregnant or lactating female patients. No concurrent use of immunosuppressives or steroids. No diabetes mellitus. No history of malignancy other than skin tumors (squamous and basal cell) in the last 5 years. No condition that excludes the patient from undergoing an MRI test.
Centros del Estudio
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National Heart, Lung and Blood Institute (NHLBI)
Bethesda, United StatesAbrir National Heart, Lung and Blood Institute (NHLBI) en Google Maps