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Study of Aficamten for Children with Symptomatic Obstructive Hypertrophic Cardiomyopathy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a heart condition called Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). This condition involves the thickening of the heart muscle, which can make it harder for the heart to pump blood effectively. The study will evaluate a treatment called Aficamten, also known by its code name CK-3773274. Aficamten is a medication taken as a film-coated tablet and is being tested to see how well it works and how safe it is for children and teenagers with this heart condition.

The purpose of the study is to compare the effects of Aficamten with a placebo in young participants who have oHCM. Participants will be randomly assigned to receive either Aficamten or a placebo. The study will be conducted in two parts. In the first part, participants will take the medication or placebo for a period of 12 weeks. During this time, doctors will monitor changes in the heart's function and any side effects. After completing the first part, participants may continue into an open-label extension, where everyone will receive Aficamten, to further assess its long-term safety and effectiveness.

Throughout the study, participants will have regular check-ups to monitor their heart health and overall well-being. The study aims to provide valuable information on how Aficamten can help manage symptoms of oHCM in a pediatric population, potentially leading to improved treatment options for this condition.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, you will undergo an initial assessment to confirm your eligibility. This includes checking your age, weight, and specific heart condition criteria.

    You will need to have an echocardiogram, which is an ultrasound of your heart, to ensure it meets the study's requirements.

  2. Step 2

    Randomization

    After the initial assessment, you will be randomly assigned to receive either the study medication, aficamten, or a placebo. A placebo is a tablet that looks like the medication but does not contain the active ingredient.

    This process is double-blind, meaning neither you nor the study team will know which one you are receiving.

  3. Step 3

    Medication administration

    If you are assigned to the aficamten group, you will take a 5 mg tablet once daily. The duration of this phase is 12 weeks.

    If you are in the placebo group, you will take a placebo tablet once daily for the same duration.

  4. Step 4

    Regular check-ups

    Throughout the 12-week period, you will have regular check-ups to monitor your heart condition and overall health.

    These check-ups will include echocardiograms and other tests to measure the effect of the medication or placebo.

  5. Step 5

    End of period 1

    At the end of the 12 weeks, your heart function will be evaluated to determine any changes from the start of the study.

    Your participation in the next phase will depend on the results and your overall health status.

  6. Step 6

    Open-label extension

    If eligible, you may enter an open-label extension phase where you will receive aficamten regardless of your initial group assignment.

    This phase involves taking the medication and attending regular check-ups every 12 weeks until the end of the study.

  7. Step 7

    Completion

    Upon completing the study, a final assessment will be conducted to evaluate your heart condition and overall health.

    The study team will provide you with information about your participation and any next steps.

Who can join the trial?

9 criteria

  • Participants must be males or females between the ages of 12 and 17 years old at the start of the study.
  • Participants must weigh at least 45 kg (about 99 pounds) initially, and then at least 35 kg (about 77 pounds) after the first group of 10 participants has completed a specific part of the study without certain heart issues.
  • Participants must have a specific heart condition called obstructive hypertrophic cardiomyopathy (oHCM), confirmed by an ultrasound of the heart. This includes having a thickened heart muscle and a normal-sized heart chamber, without other heart diseases. The heart's pumping ability must be at least 60%, and a specific heart pressure measurement must be at least 50 mmHg.
  • The oHCM must be of sarcomeric origin, which means it is related to the muscle fibers of the heart, confirmed by genetic testing. If genetic testing is not possible, it can be assumed if there are no other related health issues.
  • Participants must have a New York Heart Association (NYHA) Class II or higher heart condition, which means they have some limitations in physical activity due to their heart condition.
  • Participants must have adequate acoustic windows for echocardiography, meaning the ultrasound images of the heart must be clear enough for the study.
  • If participants are taking certain heart medications like beta blockers, verapamil, diltiazem, or disopyramide, they must have been on a stable dose for more than 4 weeks before starting the study.
  • Participants must have completed the first part of the study. If they couldn't complete it due to reasons not related to following the study rules or safety, a medical expert will review their eligibility.
  • After a break from medication, participants must have a heart pumping ability of at least 55%.

Who cannot join the trial?

9 criteria

  • Patients with **symptomatic obstructive hypertrophic cardiomyopathy** cannot participate. This is a condition where the heart muscle becomes thickened, making it harder for the heart to pump blood.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not willing or able to follow the study procedures cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are taking medications that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have a known allergy to the study medication cannot participate.
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Investigated drugs

Aficamten is a medication being studied for its potential to help people with a heart condition called obstructive hypertrophic cardiomyopathy (oHCM). This condition causes the heart muscle to become too thick, making it harder for the heart to pump blood. Aficamten is designed to help relax the heart muscle, which may improve blood flow and reduce symptoms like shortness of breath and chest pain. The trial aims to see how well aficamten works in children with this condition and to ensure it is safe for them to use.

What is already known about the treatment

Aficamten – Aficamten is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness and safety in treating children with symptomatic obstructive hypertrophic cardiomyopathy, a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The medication works by reducing the force of heart muscle contractions, which helps to improve blood flow and reduce symptoms. Aficamten belongs to a class of drugs known as cardiac myosin inhibitors, which target specific proteins in the heart muscle to achieve their effects.

Investigated diseases

Symptomatic Obstructive Hypertrophic Cardiomyopathy – This is a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The thickening often occurs in the septum, the wall dividing the left and right sides of the heart, which can obstruct blood flow out of the heart. As the disease progresses, individuals may experience symptoms such as shortness of breath, chest pain, and palpitations, especially during physical activity. Over time, the heart's ability to pump blood effectively can be further compromised, leading to increased symptoms. The obstruction in the heart can also cause the heart to work harder, potentially leading to further complications. The condition is often diagnosed in adolescence or early adulthood, but symptoms can appear at any age.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2024-511377-30-00Protocol codeCY 6023Estimated enrolment62 patientsSponsorCytokinetics Inc.

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