Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This research study is for teenagers aged 12 to 17 who have a heart condition called hypertrophic cardiomyopathy (HCM). In HCM, the heart muscle becomes thick, which can make it harder for blood to pump out, leading to symptoms like breathlessness or chest pain. The study is testing a new medicine called mavacamten. Researchers want to find out if this medicine is effective in reducing symptoms and improving heart function, how safe it is to use in young people, and how the body processes the medication. Some participants will receive mavacamten, while others will receive a placebo (a dummy pill). The goal is to see if mavacamten can help teenagers with HCM feel better and improve their quality of life.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Bristol-Myers Squibb

Enrolment target

Start

17 Apr 2024

Estimated completion

28 Mar 2031

Cardiomyopathy, Hypertrophic

What is this study about?

This study is examining a medicine called mavacamten for young people with a heart condition called hypertrophic cardiomyopathy, or HCM. In HCM, the heart's muscle walls become thicker than normal. This extra thickness, especially in the main pumping chamber (the left ventricle), can make it difficult for blood to leave the heart and get to the rest of the body. This is often described as an 'obstruction' and can cause symptoms like feeling breathless, dizzy, or having chest pain.

The main purpose of this study is to explore how well mavacamten works in teenagers (aged 12 to 17) who have these symptoms. Researchers are also very interested in understanding if the medicine is safe for this age group and how their bodies absorb and use it. This kind of research is important because it could lead to new ways to help young people manage their HCM symptoms and improve their heart health.

Participants will either receive the new medicine, mavacamten, or a placebo. A placebo is a dummy pill that looks exactly like the actual medicine but contains no active drug. This allows researchers to accurately compare the effects of the medicine against not taking it, to understand if any improvements are truly due to mavacamten. This is a common and important step in developing new treatments.

Key takeaways

This study is testing a new heart medicine called mavacamten for teenagers with HCM.
It aims to see if mavacamten is safe and effective in reducing HCM symptoms.
Participants will either receive mavacamten or a placebo.
The study includes regular check-ups and heart assessments.
It's for teenagers aged 12 to 17 who have symptomatic HCM.
Taking part is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, teenagers must have a confirmed diagnosis of hypertrophic cardiomyopathy (HCM). It's also important that their heart condition causes a blockage where blood leaves the heart, and that they are currently experiencing symptoms because of their HCM, such as shortness of breath or chest pain.

There are also some reasons why someone might not be able to join the study. For example, if their heart muscle thickness is due to a different medical problem that isn't HCM. Also, if their heart's pumping ability is already very weak (less than 50%) within the last six months, they wouldn't be able to participate. The study also cannot accept individuals who are planning significant changes to their heart treatment or surgery in the near future.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I am between 12 and 17 years old.
I have been diagnosed with hypertrophic cardiomyopathy (HCM).
My HCM causes a blockage to blood flow (LVOT obstruction).
I am currently experiencing symptoms from my HCM.
My heart's pumping ability (LVEF) has not been very weak (below 50%) in the last 6 months.
I am not planning any major heart surgery or big changes to my HCM medication soon.

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the clinic for check-ups and assessments. You would be given study medication, either mavacamten or a placebo, to take as directed. These visits would include standard procedures like blood tests, heart scans, and symptom questionnaires to monitor your health and the effects of the treatment. You would be followed closely by the study team throughout the duration of the study, and possibly for a period afterwards to ensure your well-being. The total length of your participation would be explained in detail by the study team.

Potential risks and benefits

There is a potential benefit that mavacamten could help improve your HCM symptoms and heart function. However, as with any new medicine, there are potential risks, including side effects that are not yet fully known or understood. The study team will explain all known risks and closely monitor your health to ensure your safety. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (47)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Local Institution - 0017
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Birmingham, United States
Local Institution - 0050
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Phoenix, United States
Local Institution - 0032
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Los Angeles, United States
Local Institution - 0008
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Los Angeles, United States
Local Institution - 0033
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Palo Alto, United States
Local Institution - 0044
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San Diego, United States
Local Institution - 0038
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Aurora, United States
Local Institution - 0031
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St. Petersburg, United States
Local Institution - 0053
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Atlanta, United States
Local Institution - 0013
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Chicago, United States
Local Institution - 0009
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Indianapolis, United States
Local Institution - 0043
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Boston, United States

Common questions

What is hypertrophic cardiomyopathy (HCM)?

It's a heart condition where the heart muscle becomes unusually thick, making it harder for the heart to pump blood effectively.

What is mavacamten?

It's a new medicine being tested to see if it can help improve symptoms in people with HCM by affecting how the heart muscle works.

What is a placebo?

A placebo is a 'dummy pill' that looks like the study medicine but doesn't contain any active drug. It helps researchers compare the real medicine's effects.

Will I know if I'm getting the real medicine or the placebo?

No, this is usually kept secret from you and the study doctors to make sure the results are fair and unbiased.

Who is running this study?

This study is sponsored by Bristol Myers Squibb, a pharmaceutical company, to research new treatments.