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A study testing ninerafaxstat compared to placebo in patients with symptomatic non-obstructive hypertrophic cardiomyopathy

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What is this trial about?

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

This study involves people with non-obstructive hypertrophic cardiomyopathy, which is a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood effectively. In this type, the thickening does not block the path through which blood leaves the heart, but it still causes symptoms such as shortness of breath, chest pain, fatigue, and reduced ability to exercise. The study will test a medication called Ninerafaxstat, which is given as a modified-release tablet that slowly releases the medicine over time. Some people in the study will receive ninerafaxstat while others will receive a placebo.

The purpose of the study is to see if ninerafaxstat can improve health status in people with symptomatic non-obstructive hypertrophic cardiomyopathy. The study will look at how the medication affects symptoms and quality of life, as well as how well people can exercise. The treatment will be given over a period of 12 weeks, with the highest daily dose being 400 milligrams and a total maximum dose of 33,600 milligrams over the entire treatment period.

During the study, participants will undergo various assessments including heart ultrasound examinations, which use sound waves to create pictures of the heart, and exercise tests where breathing and heart function are measured while exercising. The study will track changes in symptoms and how they affect daily activities by using questionnaires that ask about physical limitations, symptoms, quality of life, and overall health. Neither the participants nor the doctors will know who is receiving the actual medication and who is receiving the placebo during the study.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Treatment assignment

    Upon joining the trial, you will be randomly assigned to one of two treatment groups. This process is called randomization and ensures fairness in the study.

    You will receive either ninerafaxstat, which is the medication being tested, or a placebo, which is an inactive tablet that looks identical to the actual medication.

    Neither you nor your doctor will know which treatment you are receiving. This is called a double-blinded design and helps ensure accurate results.

  2. Step 2

    Medication administration

    You will take tablets by mouth. The tablets are modified-release, which means they release the medication slowly over time.

    If you are assigned to the active treatment group, you will receive ninerafaxstat trihydrochloride monohydrate.

    If you are assigned to the placebo group, you will receive tablets that look exactly the same but contain no active medication.

    You will need to follow the prescribed schedule for taking your tablets throughout the study period.

  3. Step 3

    Heart imaging tests

    You will undergo echocardiography tests during the trial. This is an ultrasound examination of your heart that uses sound waves to create images.

    These tests will be performed at the beginning of the trial and at scheduled intervals to monitor your heart function.

    The echocardiography is painless and non-invasive.

  4. Step 4

    Exercise testing

    You will complete cardiopulmonary exercise testing, abbreviated as CPET. This test measures how well your heart and lungs work during physical activity.

    The test will be performed at the beginning of the trial to establish your baseline measurements.

    You will repeat the same exercise test at Week 12 to measure any changes in your exercise capacity and breathing efficiency.

  5. Step 5

    Health status questionnaires

    You will be asked to complete the Kansas City Cardiomyopathy Questionnaire, abbreviated as KCCQ. This questionnaire asks about your symptoms and how they affect your daily life.

    You will complete this questionnaire at the start of the trial to record your baseline health status.

    You will complete the same questionnaire again at Week 12 to assess any changes in your condition.

    The questionnaire covers several areas including your overall symptoms, physical limitations, and quality of life.

  6. Step 6

    Regular monitoring visits

    Throughout the 12-week treatment period, you will attend scheduled visits for monitoring.

    During these visits, your health status will be assessed and any side effects will be recorded.

    You will be monitored for safety throughout the entire duration of your participation in the trial.

  7. Step 7

    End of treatment assessment

    At Week 12, you will complete your final assessments, which include the exercise test, heart imaging, and health questionnaires.

    These final measurements will be compared to your initial baseline measurements to determine if the treatment had any effect.

    Your participation in the active treatment phase will conclude after these assessments are completed.

Who can join the trial?

9 criteria

  • You must be able to understand and willing to sign a consent form that explains the study
  • You must be willing to follow all study procedures and requirements for the entire duration of the trial
  • You must understand the risks involved in participating in this study
  • You must be 18 years of age or older at the time you sign the consent form
  • You must have suitable conditions for echocardiography, which is an ultrasound test that creates images of your heart
  • You must have a confirmed medical diagnosis of hypertrophic cardiomyopathy, which is a condition where the heart muscle becomes abnormally thick
  • Your diagnosis must meet current medical guidelines from heart disease organizations
  • You must have unexplained left ventricular hypertrophy, which means the main pumping chamber of your heart has become thickened without a clear reason
  • The thickening of your heart muscle must not be caused by other heart diseases or body-wide conditions that could explain this level of heart muscle thickness

Who cannot join the trial?

1 criterion

  • No exclusion criteria have been provided in the available study information for this clinical trial.
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Investigated drugs

  • Ninerafaxstat

    is an investigational medication being studied for the treatment of symptomatic non-obstructive hypertrophic cardiomyopathy. Hypertrophic cardiomyopathy is a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. This medication is being tested to see if it can improve health status and symptoms in patients with this heart condition.

  • Placebo

    is an inactive substance that looks like the study medication but contains no active medicine. It is used in this trial to compare the effects of ninerafaxstat against no treatment, helping researchers determine if the medication truly works.

What is already known about the treatment

Ninerafaxstat – Ninerafaxstat is an investigational medication currently being studied in clinical trials for the treatment of non-obstructive hypertrophic cardiomyopathy, a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood effectively. The drug is being tested in a Phase 2b trial to evaluate its ability to improve health status and symptoms in patients with this heart condition. While the specific form of administration and exact mechanism of action are not detailed in the available trial information, the medication is designed to address the underlying problems in heart muscle function that cause symptoms in patients with this type of heart disease.

Investigated diseases

Non-obstructive Hypertrophic Cardiomyopathy – This is a heart condition where the heart muscle becomes abnormally thick, particularly affecting the lower left chamber of the heart. Unlike the obstructive form, this type does not block the blood flow out of the heart. The thickened muscle can make it harder for the heart to relax and fill with blood properly between beats. Patients typically experience symptoms such as shortness of breath, chest discomfort, fatigue, and reduced ability to exercise. The condition can cause the heart to work less efficiently over time. The disease progresses gradually, with symptoms often worsening during physical activity.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515125-28-00Protocol codeIMB101-010Estimated enrolment210 patientsSponsorImbria Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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