MyoKardia reports initiating trial for invasive septal reduction therapy
MyoKardia Inc. (MYOK) announced that it has dosed the first person for the Phase 3 VALOR-HCM clinical trial. The study aims to provide direct clinical evidence of mavacamten’s capability to alleviate the requirement for invasive SRT procedures. The company plans to carry out several studies to evaluate the potential of mavacamten as a backbone therapy for HCM.
VALOR-HCM is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study. It involves patients with symptomatic, obstructive HCM, who meet guideline criteria for septal reduction therapy and have been referred for an invasive procedure. Jay Edelberg, MyoKardia’s Chief Medical Officer said, “VALOR has the potential to demonstrate improved outcomes in a more severe segment of the HCM population and reflects a broader range of anticipated real-world practices.” The study will use individualized dosing based on non-invasive echocardiographic measures.
The study expects to enroll nearly 100 participants who will be randomized on a 1:1 basis to receive mavacamten or placebo. The trial incorporates three treatment periods over 128 weeks. These three periods are a 16-week placebo-controlled period, a 16-week active treatment period and a 96-week long-term extension period. In the active treatment period, the patients will be administered Mavacamten while during the long-term extension period, the administration of the drug candidate will continue.
Initially, the patients will be given a 5mg dosage which will later be adjusted based on echocardiographic assessments. If the gradient in a patient is above 30mmHg and left ventricular ejection fraction is at or above 50 percent, then the dose will be up-titrated. The doses will be adjusted in a blinded manner and may be down-titrated at any point of time.
The primary endpoint of the trial is a composite of a) the number of subjects who decide to proceed with SRT prior
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