RecruitingPHASE2INTERVENTIONAL

A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy

This study, called FORTITUDE-HCM, is testing a new drug named ninerafaxstat. It's for people who have a heart condition called non-obstructive hypertrophic cardiomyopathy (nHCM), which can make the heart muscle thick and cause symptoms like shortness of breath and tiredness. Researchers want to find out if ninerafaxstat is safe and if it can help improve these symptoms when added to the usual care. Some participants will receive the new drug, while others will receive a placebo (a dummy pill) to compare the results. This is a scientific study where neither the patients nor their doctors know who is getting which treatment until the study ends. It's happening in multiple places around the world.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Imbria Pharmaceuticals, Inc.

Enrolment target

165

Start

06 Oct 2025

Estimated completion

01 Dec 2026

Non-obstructive Hypertrophic Cardiomyopathy

What is this study about?

This study is investigating a new medicine called ninerafaxstat. It's being tested for people who have a specific heart condition known as non-obstructive hypertrophic cardiomyopathy (nHCM). In nHCM, the heart muscle becomes thicker than it should, which can make it harder for the heart to pump blood effectively. This often leads to symptoms such as shortness of breath, tiredness, and chest pain, especially during exercise. The main goal of this study is to see if ninerafaxstat can help improve these symptoms and if it's safe for patients to use.

The study is designed to compare ninerafaxstat with a placebo, which is a tablet that looks exactly like the study drug but contains no active medicine. This comparison helps researchers understand if any improvements experienced by patients are truly due to the new drug or if they might be due to other factors. Some patients will receive ninerafaxstat alongside their usual treatment for nHCM, while others will receive the placebo with their usual treatment. Neither you nor your doctor will know which treatment you are receiving until the study is over. This is a common and important way to make sure the results are as fair and accurate as possible.

This is a Phase 2 study, which means it's still in the earlier stages of drug development. We're looking at various doses and how effective the drug is, as well as keeping a close eye on any side effects. The information gathered from studies like this is crucial for developing new and better treatments for heart conditions and improving the quality of life for patients. By taking part, you could be contributing to important medical research.

Key takeaways

This study is testing a new drug for specific heart condition called nHCM.
It aims to see if ninerafaxstat is safe and improves symptoms like breathlessness.
You might receive the new drug or a dummy pill (placebo).
Participation involves regular clinic visits, tests, and taking medication daily.
The study lasts about 24 weeks.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you would need to have been diagnosed with non-obstructive hypertrophic cardiomyopathy (nHCM) by your heart specialist, following specific guidelines from medical experts. This means your heart muscle is thickened, but the blood flow out of your heart isn't blocked. We would also need to confirm this diagnosis with special heart scans, both at rest and during some light exercise.

Your symptoms also play a role. You would need to be experiencing noticeable symptoms that limit your daily activities, such as becoming breathless or tired with everyday tasks. This is often described as being in 'NYHA Class II or III.' We would also assess how much your physical activity is limited through a special exercise test.

There are also some reasons why you might not be able to join. For example, if your heart thickening is thought to be caused by a different, specific condition, or if another medical problem is causing your breathlessness or tiredness. You also need to be able to do some light exercise, such as on a treadmill or exercise bike, and not have any problems that would stop you from doing a safe exercise test.

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.

Have I been diagnosed with non-obstructive hypertrophic cardiomyopathy (nHCM)?
Do I experience breathing problems or tiredness with everyday activities?
Am I able to do light exercise on a treadmill or stationary bike?
Are my symptoms not mainly caused by other medical conditions?
Am I at least 18 years old?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned by chance to either receive the new medicine, ninerafaxstat, or a placebo (a dummy pill), in addition to your usual heart medication. You won't know which one you are taking until the study ends. You'll take the study medication once a day.

Over the course of the study, you'll need to attend several clinic visits. These visits will involve various checks, such as physical exams, blood tests, and heart scans (echocardiograms) to see how your heart is doing. You'll also complete questionnaires about your symptoms and undergo exercise tests to measure your physical abilities. The total duration of your participation in this study, including all visits and follow-ups, would be around 24 weeks.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new investigational medication that could improve your symptoms. You will also receive very close medical attention and monitoring from a team of heart specialists throughout the study. However, there's no guarantee that you will personally benefit from taking part. The study medication may also have side effects, some of which we might not fully know yet. These will be carefully monitored, and you will be informed about any known risks. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (67)

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

Imbria Investigational Site
Verified postcode
La Jolla, United States· Recruiting
Imbria Investigational Site
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Los Angeles, United States· Recruiting
Imbria Investigational Site
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San Francisco, United States· Recruiting
Imbria Investigational Site
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Washington D.C., United States· Recruiting
Imbria Investigational Site
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Miami, United States· Recruiting
Imbria Investigational Site
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Chicago, United States· Recruiting
Imbria Investigational Site
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Oak Lawn, United States· Not yet recruiting
Imbria Investigational Site
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Baltimore, United States· Recruiting
Imbria Investigational Site
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Boston, United States· Recruiting
Imbria Investigational Site
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Burlington, United States· Recruiting
Imbria Investigational Site
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Grand Rapids, United States· Recruiting
Imbria Investigational Site
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Rochester, United States· Recruiting

Common questions

What is nHCM?

nHCM stands for non-obstructive hypertrophic cardiomyopathy. It's a condition where part of your heart muscle is thicker than normal, but it doesn't block blood flow out of your heart. It can cause symptoms like breathlessness and tiredness.

What is ninerafaxstat?

Ninerafaxstat is a new medicine being tested. It's hoped to help people with nHCM by improving their heart function and reducing symptoms.

What is a placebo?

A placebo is a 'dummy' pill that looks just like the real medicine but contains no active ingredients. It's used in studies to help researchers compare the new drug's effects accurately.

Will I know if I'm getting the active drug or the placebo?

No, neither you nor your doctor will know whether you are receiving ninerafaxstat or the placebo until the study is completed. This is called 'double-blind' and helps ensure fair results.

How long does the study last?

If you join, your participation in the study is expected to last for about 24 weeks, including all appointments and follow-ups.

How to find out more

Medical Monitor

info@imbria.com (617) 675-4060 Official trial record

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