Cyclosporine A to Treat Hypertrophic Cardiomyopathy (HCM)

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Dec 9, 2002

Keywords

ClinConnect Summary

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 ...

Gender

ALL

Eligibility criteria

• • 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.