Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruiting

Long-term Safety Study of Mavacamten for Adults with Hypertrophic Cardiomyopathy from Previous Trials

Fast replyInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

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

This clinical trial is focused on studying the long-term safety of a medication called Mavacamten in adults with a heart condition known as Hypertrophic Cardiomyopathy (HCM). HCM is a disease where the heart muscle becomes abnormally thick, which can make it harder for the heart to pump blood. The study involves participants who have already completed previous trials named MAVERICK-HCM and EXPLORER-HCM. These trials were designed to explore the effects of Mavacamten on different types of HCM, specifically non-obstructive and obstructive forms.

The purpose of this study is to assess how well participants tolerate Mavacamten over a longer period. Participants will take Mavacamten in capsule form, with doses ranging from 2.5 mg to 15 mg. The study will monitor the participants for any major heart-related events, such as heart attacks or strokes, as well as hospitalizations and other heart conditions like heart failure or irregular heartbeats. The study will also look at changes in the heart's structure and function using imaging techniques like cardiac magnetic resonance imaging (MRI).

Throughout the study, participants will be regularly checked for any side effects or changes in their heart condition. The study aims to provide valuable information on the long-term use of Mavacamten for managing Hypertrophic Cardiomyopathy, helping to understand its safety and effects on heart health over time.

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 visit

    Upon joining the study, the initial visit involves a comprehensive health assessment. This includes a review of medical history and a physical examination to ensure eligibility.

    Participants must have completed a previous study related to hypertrophic cardiomyopathy within 90 days prior to this visit.

  2. Step 2

    Medication administration

    Participants will receive mavacamten in the form of a capsule. The medication is taken orally.

    The dosage and frequency of administration will be determined by the study protocol and adjusted as necessary based on individual response and tolerability.

  3. Step 3

    Regular follow-up visits

    Regular follow-up visits are scheduled to monitor health status and the effects of the medication. These visits include physical examinations and laboratory tests.

    Participants will undergo echocardiography to assess heart function and structure.

  4. Step 4

    Cardiac magnetic resonance imaging (CMR)

    Participants may undergo CMR to evaluate changes in cardiac mass and structure over time.

    This imaging is part of a sub-study for those who meet specific criteria.

  5. Step 5

    Safety and tolerability assessment

    Throughout the study, the safety and tolerability of mavacamten are assessed. This includes monitoring for any adverse events or changes in health status.

    Participants are observed for any major cardiac events or hospitalizations.

  6. Step 6

    End of study visit

    At the conclusion of the study, a final visit is conducted to evaluate overall health and the long-term effects of the medication.

    Participants will undergo a final set of assessments, including echocardiography and laboratory tests.

Who can join the trial?

8 criteria

  • The patient must have completed the previous study within 90 days of signing consent. If more than 90 days have passed, special approval may be needed. Patients who left the previous study early might still be considered.
  • The patient must understand and follow the study procedures, be aware of the risks, and give informed consent before starting the study.
  • The patient must weigh more than 45 kg (about 99 pounds) at the start of the study.
  • The patient must have good enough heart imaging results to allow accurate tests.
  • The patient must have a **left ventricular ejection fraction (LVEF)** of 50% or higher. LVEF is a measure of how well the heart pumps blood.
  • The patient's lab test results must be within normal limits. If not, they can still participate if the results are not considered important by the doctor, liver enzyme levels are less than 3 times the normal limit, and kidney function is adequate.
  • Female patients must not be pregnant or breastfeeding. If sexually active, they must use effective birth control methods during the study and for 4 months after the last dose. These methods include hormonal contraception, an intrauterine device (IUD), or surgical sterilization. Male partners must also use contraception.
  • For the sub-study, the patient must meet specific criteria and be enrolled in the MYK-461-007 Study. Patients from the MAVERICK-HCM Study can join the sub-study, while those from the EXPLORER-HCM Study must have already participated in it.

Who cannot join the trial?

5 criteria

  • Participants who have not been previously enrolled in one of the two specific placebo-controlled trials: MAVERICK-HCM for non-obstructive hypertrophic cardiomyopathy or EXPLORER-HCM for obstructive hypertrophic cardiomyopathy.
  • Participants who do not have a diagnosis of hypertrophic cardiomyopathy, which is a condition where the heart muscle becomes thickened.
  • Participants who are not within the specified age range for the study.
  • Participants who are not part of the specified clinical trial groups.
  • Participants who are not considered part of the vulnerable population selected for the study.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Mavacamten (MYK-461) is a medication being studied for its long-term safety and effects in adults with hypertrophic cardiomyopathy (HCM). This condition causes the heart muscle to thicken, which can make it harder for the heart to pump blood. Mavacamten is designed to help the heart muscle relax, potentially improving heart function and symptoms in people with HCM. The study aims to understand how safe and tolerable mavacamten is over a long period and how it affects the heart's size and structure.

What is already known about the treatment

Mavacamten – Mavacamten is administered orally in the form of capsules. It is currently being studied in clinical trials for its long-term safety and effectiveness in treating hypertrophic cardiomyopathy, a condition where the heart muscle becomes abnormally thick. 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 cardiac structure in patients with both obstructive and non-obstructive forms of hypertrophic cardiomyopathy.

Investigated diseases

Hypertrophic Cardiomyopathy – This is a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The thickening can occur in different parts of the heart, but it most commonly affects the septum, the wall that separates the left and right sides of the heart. As the heart muscle thickens, it can lead to problems with the heart's electrical system, potentially causing irregular heartbeats. Over time, the thickened heart muscle can also lead to stiffness, reducing the heart's ability to fill with blood between beats. This condition can be obstructive, where the thickened muscle blocks blood flow out of the heart, or non-obstructive, where there is no blockage. Symptoms can vary widely, with some individuals experiencing shortness of breath, chest pain, or fainting, while others may have no symptoms at all.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502858-14-00Protocol codeMYK 461-007Estimated enrolment250 patientsSponsorMyokardia Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).