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Study of Mavacamten for Adolescents 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, which is a disease where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The study will evaluate a treatment called Mavacamten, which is taken as a capsule. The purpose of the study is to see how Mavacamten affects the heart's ability to pump blood in adolescents aged 12 to under 18 years who have this condition.

Participants in the study will be randomly assigned to receive either Mavacamten or a placebo, which looks like the medication but does not contain the active ingredient. The study will last for several months, during which participants will take the capsules and have regular check-ups to monitor their heart health. The main focus will be on changes in the heart's function, particularly in the area called the left ventricular outflow tract, which is often affected in this condition.

Throughout the study, doctors will assess various aspects of heart health, including how well the heart pumps blood and any changes in heart muscle thickness. The study will also look at how participants feel during physical activities and any side effects they might experience. The goal is to gather information on the effectiveness and safety of Mavacamten for treating Symptomatic Obstructive Hypertrophic Cardiomyopathy in young people.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age between 12 and 18 years, a diagnosis of hypertrophic cardiomyopathy (HCM), and the presence of symptoms and left ventricular outflow tract (LVOT) obstruction.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either the active medication, mavacamten, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  3. Step 3

    Medication administration

    The medication, mavacamten, is provided in capsule form and taken orally. The dosage and frequency are determined by the study protocol and are consistent throughout the trial period.

  4. Step 4

    Monitoring and assessments

    Regular monitoring and assessments are conducted to evaluate the effect of the treatment. This includes measuring changes in the LVOT gradient, heart wall thickness, and other cardiac functions at specified intervals, such as Week 28.

  5. Step 5

    Primary and secondary outcomes

    The primary outcome is the change in the Valsalva LVOT gradient from the start of the study to Week 28. Secondary outcomes include changes in resting and post-exercise LVOT gradients, heart wall thickness, and other cardiac measurements.

  6. Step 6

    Completion of study

    The study is estimated to conclude by April 2028. Upon completion, participants undergo a final assessment to evaluate the overall impact of the treatment.

Who can join the trial?

4 criteria

  • Must be an adolescent aged 12 to less than 18 years old at the time of agreeing to participate.
  • Must have a diagnosis of **HCM**. This stands for **Hypertrophic Cardiomyopathy**, which is a condition where the heart muscle becomes thickened.
  • Must have **LVOT obstruction**. This means there is a blockage in the path where blood leaves the heart.
  • Must have symptoms related to the condition.

Who cannot join the trial?

8 criteria

  • Patients who have other serious heart conditions besides **Symptomatic Obstructive Hypertrophic Cardiomyopathy** cannot participate. This condition involves the heart muscle becoming thickened, which can block blood flow.
  • Patients who have had recent heart surgery or procedures may not be eligible.
  • Patients with severe kidney or liver problems are excluded.
  • Patients who are pregnant or breastfeeding cannot take part in the study.
  • Patients who are currently participating in another clinical trial are not allowed to join.
  • Patients who have a history of drug or alcohol abuse may be excluded.
  • Patients who have certain allergies or reactions to the study medication or similar drugs cannot participate.
  • Patients who are unable to follow the study procedures or attend the required visits are not eligible.
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Investigated drugs

Mavacamten is a medication being studied for its potential to help adolescents with a heart condition called symptomatic obstructive hypertrophic cardiomyopathy. This condition causes the heart muscle to thicken, which can block blood flow. Mavacamten works by helping the heart muscle relax, which may reduce the blockage and improve blood flow. The study aims to see how effective mavacamten is in reducing the obstruction in the heart's left ventricular outflow tract.

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 symptomatic obstructive hypertrophic cardiomyopathy, particularly in adolescents. The medication works by inhibiting cardiac myosin, which reduces excessive contraction of the heart muscle, thereby improving heart function. Mavacamten is classified as a cardiac myosin inhibitor and is gaining attention in medical literature for its potential to address heart muscle disorders.

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 thickened heart muscle can obstruct blood flow out of the heart, leading to symptoms such as chest pain, shortness of breath, and fainting, especially during physical activity. Over time, the obstruction can worsen, causing increased difficulty in blood flow and more pronounced symptoms. The condition can also lead to irregular heartbeats, which may contribute to the symptoms experienced. The progression of the disease can vary, with some individuals experiencing a gradual worsening of symptoms, while others may remain stable for long periods.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2023-505650-17-00Protocol codeCV027-010Estimated enrolment40 patientsSponsorBristol-Myers Squibb Services Unlimited Company

sourced from the EU Clinical Trials Register and site verification

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