Active not recruitingPHASE3INTERVENTIONAL

Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM

This research study is for adults who have a heart condition called non-obstructive hypertrophic cardiomyopathy, often shortened to nHCM. This condition makes the heart muscle thicker than it should be, but without a blockage. The study aims to find out if a new medicine, aficamten, can help people feel better, improve their ability to exercise, and lead to better health outcomes. Participants will either receive aficamten or a placebo (a dummy medicine with no active ingredients). Researchers will compare these two groups to understand the effects of aficamten on symptoms, daily life, and overall heart health. The goal is to see if this new treatment offers a safe and effective way to manage nHCM.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Cytokinetics

Enrolment target

500

Start

30 Aug 2023

Estimated completion

01 Sep 2026

Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

What is this study about?

This clinical trial is designed to investigate a new medication named aficamten for adults living with a heart condition called non-obstructive hypertrophic cardiomyopathy, or nHCM. In nHCM, the heart's pumping muscle wall becomes unusually thick, making it harder for the heart to work efficiently. Unlike some forms of hypertrophic cardiomyopathy, nHCM doesn't involve a blockage in the main blood flow out of the heart.

The main purpose of this study is to see if aficamten can improve how people live with nHCM. Specifically, researchers will be looking at whether it helps with symptoms, makes it easier to exercise, and generally improves patients' health and well-being. To do this, some participants will receive aficamten, while others will receive a placebo, which looks just like the real medicine but contains no active ingredients. This comparison helps researchers truly understand if any changes are due to the new medicine or other factors.

Finding new ways to treat nHCM is important because current treatments often focus on managing symptoms rather than directly addressing the underlying heart muscle issue. If aficamten proves to be safe and effective, it could offer a valuable new option for people living with this condition, potentially improving their quality of life and long-term health. The study uses standard medical assessments to measure these improvements throughout the trial.

Key takeaways

This study evaluates a potential new drug, aficamten, for a heart condition called nHCM.
It aims to see if aficamten improves quality of life, exercise capacity, and overall health.
Participants will receive either aficamten or a dummy medicine (placebo).
Regular health checks, heart scans, and exercise tests will monitor progress.
The study hopes to find a new, effective treatment for nHCM.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you generally need to be an adult between 18 and 85 years old. You must have been diagnosed with non-obstructive hypertrophic cardiomyopathy (nHCM), and your heart scans and other tests need to show certain details about your heart's thickness and how it's pumping, but without significant blockages. You also need to be experiencing symptoms that affect your daily life due to your heart condition.

There are also some specific health conditions that would prevent someone from joining. For example, if you have other serious heart valve problems, or certain inherited conditions that mimic nHCM, you would likely not be able to participate. If you've recently had a heart attack or have very poor heart function, or physical limitations that stop you from exercising, you might also be excluded. The study also screens for other significant lung problems or certain irregular heart rhythms.

Basically, the study is looking for a specific group of adults with nHCM to ensure the results are clear and reliable. A doctor or study team will carefully review all your medical information to see if you fit all the necessary criteria.

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.

Are you between 18 and 85 years old?
Have you been diagnosed with non-obstructive hypertrophic cardiomyopathy (nHCM)?
Are you experiencing symptoms from your nHCM that affect your daily life?
Do you have other significant heart or lung conditions that might prevent you from joining?
Are you able to do exercise tests like walking on a treadmill or cycling?
Do you have certain specific heart measurement results (your doctor can check these)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will likely have several visits to the clinic over a period of time. During these visits, you will undergo various medical checks, including heart scans (echocardiograms), blood tests, and exercise tests to see how your heart is working and how you're feeling. You will also be given either the study medicine (aficamten) or a placebo, which you will take as instructed. The study team will regularly monitor your health and any side effects you might experience. They will also ask you to complete questionnaires about your quality of life. The total duration of your participation, including all visits and follow-up, will be several months.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new investigational medication that could improve your nHCM symptoms and quality of life, or getting very close medical monitoring of your condition. However, there are also potential risks, as any medication can have side effects, and some tests might be uncomfortable or carry small risks. There's also no guarantee that the study medicine will help you, or that you'll receive the active drug rather than the placebo. It's important to remember that joining a study is always voluntary, and you have the right to withdraw at any time for any reason without it affecting your usual medical care.

Locations (180)

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
UC San Diego Health - Sulpizio Cardiovascular Center
Verified postcode
La Jolla, United States
Keck Medical Center of USC (Outpatient Clinic)
Verified postcode
Los Angeles, United States
Cedars-Sinai Medical Center (Smidt Heart Institute)
Verified postcode
Los Angeles, United States
UCLA Medical Center Cardiovascular Clinic
Verified postcode
Los Angeles, United States
University of California San Francisco
Verified postcode
San Francisco, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Verified postcode
Torrance, 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 Medical Group - Cardiology Associates
Verified postcode
Fort Lauderdale, United States
Investigational Drug Services, AdventHealth Orlando
Verified postcode
Orlando, United States
Emory Clinic
Verified postcode
Atlanta, United States

Common questions

What is nHCM?

nHCM stands for non-obstructive hypertrophic cardiomyopathy. It's a heart condition where the heart muscle is too thick, making it harder for the heart to pump blood effectively, but without a blockage in the blood flow out of the heart.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real drug but contains no active ingredients. It's used in studies to compare the effects of the new medicine against no treatment, to see if the real medicine is actually working.

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

No, this type of study is 'blinded,' meaning neither you nor your study doctor will know whether you are receiving aficamten or the placebo. This helps ensure the results are unbiased.

What will happen during the study visits?

During study visits, you'll have various health checks, including heart ultrasounds, blood tests, physical exams, and exercise tests. You'll also discuss how you're feeling and any symptoms you have.

Can I leave the study at any time?

Yes, your participation is completely voluntary, and you can choose to leave the study at any point and for any reason without it affecting your regular medical care.