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Enrolling by invitationPHASE2, PHASE3INTERVENTIONAL

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM

This research study is for adults aged 18 to 85 who have a heart condition called hypertrophic cardiomyopathy (HCM). HCM causes the heart muscle to become thick, making it harder for the heart to pump blood effectively. The study is particularly for those who have already taken part in a previous clinical trial for a medicine called aficamten. Its main goal is to find out more about the long-term safety and how well people tolerate aficamten over time. This helps doctors understand if the medicine is safe to use for longer periods. Participants will continue to receive aficamten at doses between 5 and 20 mg. Gathering this information is important for potentially making this treatment available to more people in the future.

At a glance

Status
Enrolling by invitation
Phase
PHASE2, PHASE3
Sponsor
Cytokinetics
Enrolment target
900
Start
06 May 2021
Estimated completion
01 Mar 2028

What is this study about?

This study is looking into a medication called aficamten for adults who have a heart condition known as hypertrophic cardiomyopathy, or HCM. In HCM, the heart's walls become unusually thick, which can make it harder for the heart to pump blood properly. This can lead to symptoms like shortness of breath or chest pain.

Specifically, this study wants to understand the long-term effects of aficamten. This means checking its safety and if people can comfortably take it over an extended period. Researchers are also interested in how well people tolerate the medicine, meaning if they experience any side effects or discomfort. This is important information to collect because medicines often behave differently when taken for a long time compared to just for a short period.

This study is open to both men and women. By continuing to gather information about aficamten, researchers hope to learn more about its overall benefits and potential risks, which could help people with HCM in the future.

Key takeaways

  • This study evaluates the long-term safety of aficamten for HCM.
  • It's for adults who have already participated in a previous aficamten trial.
  • The study monitors how well participants tolerate the medicine over time.
  • Participants will continue to receive aficamten (5-20mg).
  • Understanding long-term effects is crucial for future treatment options.
  • You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must be an adult between 18 and 85 years old. A key requirement is that you must have already participated in an earlier study that investigated aficamten, the same medicine being looked at here. Also, your heart's main pumping chamber (left ventricle) must be working well enough, specifically with a measurement called LVEF at or above 55% at the start of this study.

There are also some reasons why you might not be able to join. For example, if you have recently taken another similar medicine called mavacamten, or if you've been part of another medical study involving a new drug or device in the last month (or longer for certain medications). We also can't include you if you've had certain heart procedures or developed specific heart rhythm problems since your last aficamten study, unless enough time has passed and your condition is stable.

Also, if during your previous aficamten study your heart's pumping ability dropped significantly below 40% and caused your medicine dose to be stopped, you wouldn't be able to join this study. If you've had a special heart device (ICD) fitted very recently, within 30 days before the start of this study, you also wouldn't be eligible.

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.

  1. Are you between 18 and 85 years old?
  2. Have you participated in a previous study involving aficamten?
  3. Is your heart's main pumping chamber (LVEF) working well, at 55% or more?
  4. Have you NOT taken a similar medicine called mavacamten recently?
  5. Have you NOT had a new heart rhythm problem or heart surgery/procedure very recently?
Answer every question to see your result.

What does participation involve?

If you decide to take part, you will continue to receive the study medicine, aficamten, at a dose between 5 and 20 mg. Throughout your participation, you will have regular check-ups with the study team. These visits will involve various tests to monitor your health and how you are responding to the medicine, including checks of your heart function. The study is designed to follow you for a long time, so you'll continue taking the medication and attending appointments for an extended period, which will be discussed fully with you before you join. The total duration of your participation will depend on the overall study plan and your individual progress.

Potential risks and benefits

Taking part in this study could potentially offer continued access to a treatment that may help manage your hypertrophic cardiomyopathy. However, as with any medicine, there are potential risks, including side effects from aficamten – though the aim of this study is to better understand these long-term effects. The research team will carefully monitor you for any side effects throughout the study. You will receive detailed information about all known potential risks before you decide to join, and it's important to remember that you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (129)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • University of Alabama at Birmingham
    Verified postcode
    Birmingham, United States
  • Alaska Heart and Vascular Institute
    Verified postcode
    Anchorage, United States
  • Mayo Clinic Building - Phoenix
    Verified postcode
    Phoenix, United States
  • UC San Diego Health - Sulpizio Cardiovascular Center
    Verified postcode
    La Jolla, United States
  • Cedar-Sinai Medical Center
    Verified postcode
    Los Angeles, United States
  • Cedars-Sinai Medical Center (Smidt Heart Institute)
    Verified postcode
    Los Angeles, United States
  • UCSF Medical Center
    Verified postcode
    San Francisco, United States
  • Yale New Haven Hospital
    Verified postcode
    New Haven, United States
  • MedStar Washington Hospital Center
    Verified postcode
    Washington D.C., United States
  • Holy Cross Hospital / Cardiology Associates
    Verified postcode
    Fort Lauderdale, United States
  • Emory Clinic
    Verified postcode
    Atlanta, United States
  • Piedmont Fayette Hospital
    Verified postcode
    Fayetteville, United States

Common questions

What is hypertrophic cardiomyopathy (HCM)?

HCM is a heart condition where the heart muscle becomes unusually thick, making it harder for your heart to pump blood around your body effectively.

What is aficamten?

Aficamten is a medicine being studied for people with HCM. This study aims to understand its long-term safety and how well people tolerate it.

Why is this study only for people who have taken aficamten before?

This study is a continuation of earlier research. It's designed to gather more information about how aficamten works over a longer time in people who are already familiar with the treatment.

How long will I be in the study?

This is a long-term study, so your participation will continue for an extended period. The study team will explain the expected duration in detail before you join.

Can I stop participating in the study if I change my mind?

Yes, you have the right to withdraw from the study at any time, for any reason, without affecting your medical care.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "Open-label Extension Study to Evaluate the Long-term Safety …" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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