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Drug Trial Enrolls Patients at UPMC for Treatment of Rare Heart Condition

November 13, 2018
The UPMC Heart and Vascular Institute recently enrolled its first patient in the phase 2 MAVERICK-HCM clinical trial of mavacamten, a new drug in development to treat non-obstructive hypertrophic cardiomyopathy (HCM). If clinical testing ultimately demonstrates safety and efficacy, mavacamten, manufactured by MyoKardia, will be the first approved drug for the condition that specifically targets an underlying disease pathway.

HCM refers to a rare heart condition characterized by thickening of the heart muscle, which makes it harder for the heart to pump blood. The most common cause of HCM is mutations of myosin proteins that result in excessive contraction, stiffening of the left ventricle and a reduced ability for the heart to properly fill. Mavacamten binds to a region of the myosin protein affected by commonly occurring mutations and is intended to inhibit the excess contraction underlying the disease. 

UPMC, western Pennsylvania’s only Center of Excellence in HCM as designated by the Hypertrophic Cardiomyopathy Association, will enroll several participants into the MAVERICK-HCM trial, all of whom must meet stringent inclusion criteria to allow investigators to accurately determine the safety and tolerability of the drug, as well as its impact on functional capacity and quality of life.

Affecting about 1 in 500 people, HCM may manifest in two forms. In HCM’s obstructive form, the enlarged muscle physically blocks blood flow from the left ventricle, but in its non-obstructive form, HCM leaves the heart unable to fill or pump blood effectively without an actual blockage. Drugs such as beta blockers may provide symptom relief, and some obstructive HCM patients can undergo surgery to cut away the obstructed muscle, but non-obstructive patients lack this option, and some eventually require a heart transplant.

“Current therapy was never designed to target the underlying cause of HCM, leaving a large symptom burden that goes untreated. An approved drug that targets a fundamental disease pathway at the molecular level would change the way we care for our patients,” said Timothy Wong, M.D., MAVERICK-HCM’s primary investigator at UPMC. “We are excited to offer non-obstructive HCM patients the opportunity to participate in this important clinical trial. If mavacamten is ultimately proved safe and effective, we will be able to provide patients with newfound hope for more effective management of this condition.”

Thirty centers from across the country will collectively enroll 60 participants, and each will be randomly assigned to receive a daily oral dose of mavacamten or a placebo for 16 weeks. Participants will be monitored for an additional eight weeks after their dosage is complete. 

Preliminary data from the MAVERICK-HCM trial are expected in late 2019.  If the results of the MAVERICK-HCM study are encouraging, a larger, more definitive phase 3 clinical trial of mavacamten in patients with non-obstructive HCM would likely need to be conducted.

UPMC also is an enrollment site for the phase 3 EXPLORER-HCM clinical trial, which is testing mavacamten’s efficacy in patients with obstructive HCM. For additional information about all of the clinical trials offered at the UPMC Heart and Vascular Institute, visit the clinical trials website.