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Clinical Trial results in new Medication for Hypertrophic Cardiomyopathy


A clinical trial has found that Mavacamten is effective in treating symptomatic obstructive HCM

Recent medical research has provided promising results for those with obstructive hypertrophic cardiomyopathy (HCM). A new medication, mavacamten, has been found to have significant impacts on exercise physiology. This finding comes from a prespecified exploratory analysis of the EXPLORER-HCM study, a randomized, double-blind, placebo-controlled, phase 3 trial conducted in 68 cardiovascular centers in 13 countries.

The study included 251 patients with symptomatic obstructive HCM, who were split into two groups - one receiving mavacamten and the other a placebo. Cardiopulmonary exercise testing (CPET) was performed at baseline and week 30 to measure peak oxygen uptake and other exercise performance parameters.

Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is a genetic heart condition that affects around 1 in 500 people. It is characterized by the thickening of the heart muscle, which can lead to a variety of symptoms including chest pain, shortness of breath, dizziness, and fainting. It can affect the heart’s ability to pump blood and can cause arrhythmias and sudden cardiac death. HCM is most commonly diagnosed in adolescents and young adults but can affect individuals of any age.

Treatment includes lifestyle changes, medications, and, in some cases, surgery. Early diagnosis and ongoing management can help reduce the risk of complications and improve quality of life.


Mavacamten is a new drug that can be used to treat hypertrophic cardiomyopathy (HCM), a condition that causes an abnormal thickening of the heart muscle. It works by targeting the underlying cause of the condition and has been shown to improve symptoms, functional capacity, biomarkers, and key aspects of quality of life in patients with symptomatic obstructive HCM. It has also been associated with a significant improvement in oxygen levels in the blood at week 30 compared with placebo.

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The results of the trial showed that mavacamten improved peak exercise performance, as well as other peak-exercise CPET parameters such as carbon dioxide output, minute ventilation, peak ventilation/carbon dioxide ratio, ventilatory efficiency, peak respiratory exchange ratio, peak circulatory power, ventilatory power, ventilatory threshold, peak metabolic equivalents, and peak exercise time.

Beyond peak-exercise performance, mavacamten also improved submaximal exertional tolerance, with improvements in nonpeak-exercise CPET parameters such as ventilatory efficiency/carbon dioxide slope and ventilatory power.

The Trial

The results of this clinical trial are promising for those with obstructive HCM. Mavacamten showed significant improvements in both peak-exercise and submaximal exertional performance parameters, indicating consistent and broad benefits on maximal exercise capacity. These findings provide further insight into the beneficial impact of mavacamten for those with obstructive HCM.

The EXPLORER-HCM study was the first randomized, double-blind, placebo-controlled, phase 3 trial of mavacamten for symptomatic obstructive HCM and the results are encouraging. It is important to note that further research into the effects of mavacamten is needed to definitively demonstrate the long-term effects of this medication.

For those with obstructive HCM, this research provides hope for improved exercise performance. The results of this clinical trial show that mavacamten may be a viable and effective treatment option for those with symptomatic obstructive HCM, with favorable effects on peak and submaximal exertional performance.
Mayo Clinic, May-24-22

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This content is for informational and educational purposes only. It is not intended to provide medical advice or to take the place of such advice or treatment from a personal physician. All readers/viewers of this content are advised to consult their doctors or qualified health professionals regarding specific health questions. CenTrial Data Ltd. does not take responsibility for possible health consequences of any person or persons reading or following the information in this educational content. Treatments and clinical trials mentioned may not be appropriate or available for all trial participants. Outcomes from treatments and clinical trials may vary from person to person. Consult with your doctor as to whether a clinical trial is a suitable option for your condition. Assistance from generative AI tools may have been used in writing this article.