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Study on Mavacamten for Adults with Non-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 Non-obstructive Hypertrophic Cardiomyopathy. This condition involves the thickening of the heart muscle, which can make it harder for the heart to pump blood. The study will evaluate a treatment called Mavacamten, which is taken as a capsule. Mavacamten is being tested to see if it can help improve symptoms and physical limitations in people with this heart condition.

The purpose of the study is to compare the effects of Mavacamten with a placebo over a period of 48 weeks. Participants will be randomly assigned to receive either Mavacamten or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. The study will monitor changes in health status, including symptoms and exercise capacity, to determine the effectiveness of Mavacamten.

Throughout the study, participants will take the medication orally and will have regular check-ups to assess their condition. The study aims to provide valuable information on whether Mavacamten can be a beneficial treatment for individuals with Non-obstructive Hypertrophic Cardiomyopathy, potentially improving their quality of life by reducing symptoms and enhancing their ability to perform physical activities.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant will be randomly assigned to receive either the active medication, mavacamten, or a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

  2. Step 2

    Medication administration

    The participant will take the assigned medication orally in the form of a capsule. The dosage and frequency will be determined by the study protocol, and the treatment will last for a duration of 48 weeks.

  3. Step 3

    Monitoring and assessments

    Throughout the 48-week period, the participant's health status, symptoms, and physical limitations will be regularly assessed. This includes monitoring exercise capacity and other health indicators.

  4. Step 4

    Primary outcome evaluation

    The primary outcomes will be evaluated by measuring changes from the baseline in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS) and peak oxygen consumption (pVO2) at week 48.

  5. Step 5

    Secondary outcome evaluation

    Secondary outcomes will include changes in the VE/VCO2 slope, improvement in New York Heart Association (NYHA) class, and changes in biomarkers such as NT-pro BNP and cTn-T. Additionally, the time to first major adverse cardiovascular events (MACE-Plus) will be recorded.

  6. Step 6

    Completion of the study

    At the end of the 48-week period, the participant will complete the study. Final assessments will be conducted to evaluate the overall impact of the treatment.

Who can join the trial?

10 criteria

  • Participants must be at least 18 years old or the local age of majority.
  • Female participants must use highly effective birth control methods or have proof that they cannot have children.
  • Male participants do not need to use additional birth control measures.
  • Participants must have a diagnosis of **Hypertrophic Cardiomyopathy (HCM)**, which is a condition where the heart muscle becomes thickened, following specific medical guidelines.
  • The **peak LVOT pressure gradient** must be less than 30 mmHg at rest and less than 50 mmHg during certain tests. This measures the pressure difference in the heart.
  • Participants must have an oxygen level at rest greater than 90% during screening and be able to perform a special exercise test called **cardiopulmonary stress test (CPET)**. This test checks how well the heart and lungs work during physical activity.
  • Participants must be in **New York Heart Association (NYHA) Class II or III**, which describes the severity of heart failure symptoms.
  • Participants must have a **NT-proBNP** level of 200 pg/mL or higher, or a **BNP** level of 70 pg/mL or higher. These are blood tests that help measure heart function.
  • The **LVEF** (Left Ventricular Ejection Fraction) must be 60% or higher on an echocardiogram, which is an ultrasound of the heart.
  • The **KCCQ-23 CSS Score** must be 85 or lower at screening. This score measures the impact of heart failure on a person's life.

Who cannot join the trial?

8 criteria

  • Patients with other types of heart conditions that are not related to Non-obstructive Hypertrophic Cardiomyopathy cannot participate. This is a condition where the heart muscle becomes thickened without blocking blood flow.
  • Individuals who are not within the specified age range for the study are excluded. The study is designed for certain age groups only.
  • Participants who are unable to follow the study procedures or take the study medication as required will not be eligible.
  • People with certain medical conditions or who are taking medications that might interfere with the study treatment are not allowed to join.
  • Pregnant or breastfeeding women are not eligible to participate in the study.
  • Individuals who have participated in another clinical trial recently may be excluded to avoid interference with the study results.
  • Patients with a history of drug or alcohol abuse that could affect their ability to follow the study requirements are not eligible.
  • Anyone who has a known allergy or adverse reaction to the study medication or similar drugs cannot participate.
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Investigated drugs

Mavacamten is a medication being studied for its potential to help adults with symptomatic nonobstructive hypertrophic cardiomyopathy. This condition involves the thickening of the heart muscle, which can lead to symptoms like shortness of breath and reduced exercise capacity. Mavacamten works by targeting the heart muscle to improve its function, potentially reducing symptoms and improving the ability to exercise. The clinical trial aims to evaluate how effective a 48-week course of mavacamten is in improving patients' health status and physical limitations compared to not receiving the medication.

What is already known about the treatment

Mavacamten – Mavacamten is administered orally in the form of a capsule. It is currently being studied in clinical trials for its effectiveness in treating non-obstructive hypertrophic cardiomyopathy, a condition characterized by thickened heart muscle. The medication works by inhibiting cardiac myosin, a protein involved in heart muscle contraction, which helps to reduce excessive contraction and improve heart function. Mavacamten is classified as a cardiac myosin inhibitor and is being evaluated for its potential to improve symptoms and physical limitations in affected individuals.

Investigated diseases

Non-obstructive Hypertrophic Cardiomyopathy – This is a condition where the heart muscle becomes abnormally thick without blocking blood flow. The thickened heart muscle can make it harder for the heart to pump blood efficiently. Over time, this can lead to symptoms such as shortness of breath, chest pain, and palpitations. The disease may progress slowly, and symptoms can vary widely among individuals. Some people may experience significant physical limitations, while others may have mild or no symptoms. The condition is often inherited and can be detected through family history.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-506352-24-00Protocol codeCV027-031Estimated enrolment478 patientsSponsorMyokardia Inc.

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