Two recent cardiology trials from Cleveland Clinic have focused on a "gene silencing" therapy and an experimental drug used to treat heart disease that thickens a heart muscle.
A phase 3 clinical trial from Cleveland Clinic researchers found that mavacamten, an experimental drug, significantly reduced the need for invasive procedures in severely symptomatic, hypertrophic cardiomyopathy patients, the system said April 2.
Researchers enrolled 112 symptomatic patients at 19 sites across the nation and either prescribed them mavacamten or a placebo. After 16 weeks, 76.8 percent of the placebo patients continued to meet guideline criteria for surgery or elected to undergo a procedure compared to 17.9 percent of patients who received mavacamten.
A phase 1 trial showed an experimental "gene silencing" therapy reduced blood levels of lipoprotein, a key indicator of heart disease risk, by up to 98 percent, the system said April 3.
Participants in the trial who received higher doses of a small interfering RNA therapeutic that silences the gene responsible for lipoprotein production saw levels fall by up to 98 percent. Five months later, these participants' levels remained 71 percent to 81 percent lower than baseline.