What side effects are associated with this type of intervention?

Common treatments for Hypertrophic Cardiomyopathy (HCM) include beta-blockers, calcium channel blockers, and cardiac myosin inhibitors like Aficamten. Beta-blockers can cause side effects such as fatigue, dizziness, and bradycardia. Calcium channel blockers may lead to peripheral edema, constipation, and hypotension. Cardiac myosin inhibitors, such as Aficamten, are being studied for their efficacy and safety, with potential side effects including heart failure and arrhythmias. It is important to discuss these options and their risks with a healthcare provider.

What prior FDA approvals exist?

Hypertrophic Cardiomyopathy (HCM) treatments have included various drug classes, but specific FDA approvals for Cardiac Myosin Inhibitors are limited. Mavacamten, another Cardiac Myosin Inhibitor, has been approved by the FDA for the treatment of symptomatic obstructive HCM. This drug works similarly to Aficamten by targeting the myosin protein to reduce excessive contractility of the heart muscle. Other treatments for HCM have traditionally included beta-blockers, calcium channel blockers, and antiarrhythmic drugs, which help manage symptoms but do not directly target the underlying pathophysiology like Cardiac Myosin Inhibitors.

What other types of research exist?

Promising novel alternatives to Aficamten for Hypertrophic Cardiomyopathy patients include Mavacamten, another cardiac myosin inhibitor that has shown efficacy in reducing left ventricular outflow tract obstruction and improving symptoms. Additionally, gene therapies targeting specific genetic mutations associated with HCM are under investigation and may offer future treatment options. It is essential to consult with a healthcare provider to determine the most appropriate treatment based on individual patient profiles and the latest clinical evidence.

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Aficamten for Hypertrophic Cardiomyopathy (ACACIA-HCM Trial)

Q: What is the mechanism of action of this treatment?

Hypertrophic Cardiomyopathy (HCM) treatments often target the excessive contractility and abnormal heart muscle thickening characteristic of the disease. Cardiac myosin inhibitors, such as aficamten, work by reducing the hypercontractility of the heart muscle, thereby decreasing the obstruction in the left ventricular outflow tract and improving symptoms. This is crucial for HCM patients as it helps alleviate symptoms like shortness of breath and chest pain, and can improve exercise capacity and overall quality of life. Other common treatments include beta-blockers and calcium channel blockers, which reduce heart rate and contractility, and septal myectomy or alcohol septal ablation, which physically reduce the thickened heart muscle. These treatments collectively aim to manage symptoms, prevent complications, and improve the functional status of HCM patients.

Phase 3

Recruiting

Research Sponsored by Cytokinetics

Eligibility Criteria Checklist

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

Must have

NYHA class II or III

Respiratory exchange ratio of ≥ 1.00 at screening by cardiopulmonary exercise testing (CPET) and predicted peak oxygen uptake (pVO2) ≤ 90% for age and sex

Must not have

Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM

History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to end of study, week 72

Awards & highlights

Pivotal Trial

Summary

This trial is testing aficamten, a medication that may help people with a thickened heart muscle condition. It aims to improve their quality of life and ability to exercise. The drug works by making the heart muscle less stiff, which could help the heart pump blood better.

Who is the study for?

Adults aged 18-85 with non-obstructive hypertrophic cardiomyopathy (nHCM), specifically those who are somewhat limited in physical activity but not bedridden. They must have a body mass index under 40, no recent heart surgeries or treatments with similar drugs, and be able to exercise on a treadmill or bicycle.

What is being tested?

The trial is testing Aficamten against a placebo to see if it improves life quality, exercise ability, and health outcomes for people with nHCM. Participants will either receive the actual drug or a placebo without knowing which one they're getting.

What are the potential side effects?

While specific side effects of Aficamten aren't listed here, common ones for heart medications may include dizziness, changes in blood pressure or heart rhythm, fatigue, headache, and potential gastrointestinal issues.

Eligibility Criteria

Inclusion Criteria

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

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My heart condition mildly or moderately affects my daily activities.

Select...

My exercise test shows I use a lot of oxygen and my peak oxygen level is low for my age and sex.

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I have been diagnosed with non-obstructive hypertrophic cardiomyopathy.

Exclusion Criteria

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

Select...

I have a condition that causes thickening of the heart muscle, similar to nHCM.

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My heart's pumping strength is below normal.

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I have low oxygen levels without extra oxygen or severe lung/blood pressure issues.

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I have had fainting spells or dangerous heart rhythms during exercise in the last 3 months.

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My high blood pressure has not improved with treatment.

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I have been treated with aficamten before.

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I have a type of irregular heartbeat known as atrial fibrillation.

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I have a severe blockage in my heart's arteries or had a heart attack before.

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I have been treated with mavacamten within the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to end of study, week 72

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to end of study, week 72

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to end of study, week 72 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)

Secondary study objectives

Change in LAVI

Change in NT-proBNP

Change in composite of two Z-scores of CPET parameters (pVO2 and VE/VCO2 slope)

+2 more

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: AficamtenExperimental Treatment1 Intervention

Participants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of aficamten with dose levels guided by echocardiography assessments, for up to 72 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants in this arm will receive placebo, for up to 72 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Aficamten

2023

Completed Phase 1

~50

0 / 12Eligibility 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 excessive contractility and abnormal heart muscle thickening characteristic of the disease. Cardiac myosin inhibitors, such as aficamten, work by reducing the hypercontractility of the heart muscle, thereby decreasing the obstruction in the left ventricular outflow tract and improving symptoms. This is crucial for HCM patients as it helps alleviate symptoms like shortness of breath and chest pain, and can improve exercise capacity and overall quality of life. Other common treatments include beta-blockers and calcium channel blockers, which reduce heart rate and contractility, and septal myectomy or alcohol septal ablation, which physically reduce the thickened heart muscle. These treatments collectively aim to manage symptoms, prevent complications, and improve the functional status of HCM patients.