What side effects are associated with this type of intervention?

Common treatments for Hypertrophic Cardiomyopathy (HCM) include beta-blockers, calcium channel blockers, and myosin inhibitors like Mavacamten. Beta-blockers can cause fatigue, dizziness, and bradycardia. Calcium channel blockers may lead to side effects such as peripheral edema, constipation, and hypotension. Mavacamten, a myosin inhibitor, is being studied for its potential to reduce the need for septal reduction therapy. Known side effects of myosin inhibitors can include heart failure, atrial fibrillation, and potential interactions with other cardiovascular medications. Patients should discuss these risks with their healthcare provider to determine the best treatment plan.

What prior FDA approvals exist?

Mavacamten is a myosin inhibitor currently being studied for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). As of now, there are no other myosin inhibitors approved by the FDA specifically for HCM. Traditional treatments for HCM include beta-blockers, calcium channel blockers, and disopyramide, which help manage symptoms but do not target the underlying myosin hypercontractility. Mavacamten represents a novel approach by directly modulating the cardiac myosin, potentially reducing the need for invasive procedures like septal reduction therapy.

What other types of research exist?

Promising novel alternatives to Mavacamten for Hypertrophic Cardiomyopathy patients include: 1) Aficamten, another myosin inhibitor currently under investigation, 2) Gene therapies targeting specific genetic mutations associated with HCM, and 3) Emerging pharmacological agents such as small molecule inhibitors and modulators of cardiac muscle contraction. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on their specific condition and the latest clinical evidence.

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Cardiac Myosin Inhibitor

Mavacamten for Hypertrophic Cardiomyopathy (VALOR-HCM Trial)

Phase 3

Waitlist Available

Research Sponsored by Bristol-Myers Squibb

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosed with oHCM consistent with current ACCF/AHA 2011 and meet their recommendations for invasive therapies

At least 18 years old at screening and body weight > 45 kg at screening

Must not have

Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at screening

Prior treatment with cardiotoxic agents, such as doxorubicin or similar

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 16

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called mavacamten to see if it can help people with a heart condition called obstructive hypertrophic cardiomyopathy (oHCM). The goal is to reduce the need for a surgical procedure by helping the heart muscle relax better. The study involves patients in the U.S. who have symptoms from this condition.

Who is the study for?

Adults over 18 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) who weigh more than 45 kg, have a left ventricular ejection fraction (LVEF) of at least 60%, and oxygen saturation above 90%. They should be eligible for septal reduction therapy and not have had prior invasive septal reductions or recent medication changes.

What is being tested?

The study is testing Mavacamten against a placebo to see if it can reduce the need for septal reduction procedures in patients with oHCM. Participants will be randomly assigned to receive either the drug or a placebo without knowing which one they are getting.

What are the potential side effects?

While specific side effects of Mavacamten are not listed here, common side effects may include symptoms related to heart function changes, such as dizziness or shortness of breath. Patients will be monitored for any adverse reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with obstructive hypertrophic cardiomyopathy according to the latest guidelines.

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I am over 18 years old and weigh more than 45 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have episodes of irregular heartbeat that come and go, and it was present during my screening.

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I have been treated with heart-damaging drugs like doxorubicin.

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I've had surgery or a procedure to reduce heart muscle thickness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 16 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16

Secondary study objectives

Change From Baseline to Week 16 in Cardiac Troponin

Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS)

Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP)

+2 more

Side effects data

From 2024 Phase 3 trial • 112 Patients • NCT04349072

Fatigue

Dizziness

Headache

Dyspnoea

Palpitations

Dyspnoea exertional

Hypertension

Arthralgia

Atrial fibrillation

Peripheral venous disease

Cardiac failure congestive

Acute respiratory failure

Gastrooesophageal reflux disease

Sudden cardiac death

Fall

Large intestine perforation

Pneumatosis intestinalis

Nephrolithiasis

Pulmonary embolism

COVID-19

Clostridium difficile infection

100%

80%

60%

40%

20%

Study treatment Arm

Mavacamten

Placebo

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Drug: MavacamtenExperimental Treatment1 Intervention

Mavacamten Capsules Other names: MYK-461

Group II: Drug: PlaceboPlacebo Group1 Intervention

Matching Placebo Capsules

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mavacamten

2022

Completed Phase 3

~530

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hypertrophic Cardiomyopathy (HCM) treatments often target the reduction of myocardial contractility and improvement of diastolic function. Common treatments include beta-blockers and calcium channel blockers, which reduce heart rate and myocardial oxygen demand, thereby alleviating symptoms. Mavacamten, a myosin inhibitor, works by directly reducing the excessive contractility of the heart muscle, which is a hallmark of HCM. This reduction in contractility helps to decrease the left ventricular outflow tract obstruction, improving symptoms and potentially reducing the need for invasive procedures like septal reduction therapy. Understanding these mechanisms is crucial for HCM patients as it helps tailor treatment plans that can effectively manage symptoms and improve quality of life.