Brigham and Women’s Hospital researchers are enrolling participants in a potentially breakthrough study that may answer a longstanding medical question: Can we prevent or slow down the progress of hypertrophic cardiomyopathy (HCM) in family members of HCM patients who carry a gene mutation for this disease but do not yet exhibit any thickening of the heart muscle? HCM is the most common inherited cardiovascular disorder, affecting one in every 1,000 individuals. The condition is characterized by a thickening of the heart wall (left ventricular hypertrophy) and it can lead to heart rhythm irregularities, heart failure and, in some cases, sudden death.
Research at BWH led to the discovery of gene mutations responsible for HCM, and now, we want to use that information to develop better and more effective treatment to change the consequences of carrying a gene mutation. This new HCM study will investigate whether a medication called diltiazem, an FDA approved drug commonly used to treat hypertension, can be used to decrease and/or prevent the progression of HCM. You qualify for this study if you are known to carry a gene mutation that puts you at risk for developing HCM but do not currently have any symptoms or clinical signs of the disease. The study will last for five years and includes taking diltiazem or an inactive sugar pill once a day for the first four years. Participants will be asked to come to the hospital for a total of 11 study visits in five years (four years while taking the study drug and one year after for follow-up). Some of the early study visit evaluations can be performed by a local doctor and, therefore, do not require a trip to Boston.
To learn more about the study, please contact Allison L. Cirino, MS, CGC at 617-732-7921 or acirino@partners.org.
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