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Study of aficamten and 68Ga‑FAPI‑46 versus placebo in asymptomatic obstructive hypertrophic cardiomyopathy patients

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What is this trial about?

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

Hypertrophic cardiomyopathy is a condition in which the heart muscle becomes unusually thick, which can make it harder for blood to flow out of the heart. In some people the condition causes no symptoms, but the thickened muscle can still create a blockage. The trial tests an oral tablet called aficamten taken at a dose of 20 mg each day and compares it with a placebo, which looks the same but does not contain the active drug. An additional substance, 68GA‑FAPI‑46, is used during special imaging scans to help visualize the heart.

The purpose of the study is to see whether aficamten can lower the pressure difference measured as the left ventricular outflow tract gradient after a breathing maneuver called the Valsalva maneuver. Participants will take the study medication for about one year, with regular visits that include a simple breathing test, ultrasound images of the heart (echocardiography), a magnetic resonance imaging scan, and a positron emission tomography/computed tomography scan that uses the 68GA‑FAPI‑46 agent. Blood samples will be taken to check biomarkers such as NT‑proBNP and hsTnT, which are substances that rise when the heart is under stress.

Throughout the study, the size and function of the heart, the thickness of the heart wall, and any signs of heart failure or irregular heart rhythm will be recorded. The results will show whether the study drug can safely reduce the blockage and improve heart measurements compared with the placebo.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and receipt of study medication

    After enrollment you are assigned to receive either the active drug aficamten or an identical looking placebo. the study medication is provided in film‑coated tablets.

    You will be given a supply of tablets to start taking at home.

  2. Step 2

    Daily oral tablet intake

    Take one tablet containing 20 mg of aficamten (or the matching placebo) by mouth once each day.

    Continue this daily dosing for the entire study period, which lasts until week 54.

  3. Step 3

    Baseline assessments and first injection

    At the first study visit you will have blood drawn for laboratory tests and an echocardiogram to evaluate heart function.

    A cardiac magnetic resonance imaging (cMRI) scan is performed to measure heart muscle thickness and other structures.

    You will receive a single intravenous injection of 200 megabecquerel (MBq) of the radiotracer 68ga‑fapi‑46. this injection is used for a PET/CT scan that assesses heart tissue activity.

  4. Step 4

    Mid‑study assessment at week 18

    You return for a visit at week 18 where blood tests are repeated to measure biomarkers such as nt‑probnp and hstnt.

    An echocardiogram is performed again to check any changes in heart function.

    The study medication continues to be taken daily throughout this period.

  5. Step 5

    Final assessment and end of study at week 54

    At week 54 you undergo the same set of evaluations as at baseline: blood tests, echocardiogram, cMRI scan, and a second PET/CT scan after another 200 MBq 68ga‑fapi‑46 injection.

    After the final assessments you stop taking the study tablets.

    The study concludes for you after this visit.

Who can join the trial?

11 criteria

  • Be a man or woman who is between 18 and 70 years old.
  • Be able to understand the study information and be willing to sign an informed consent form.
  • Have a diagnosis of hypertrophic cardiomyopathy (HCM), which means the heart muscle is unusually thick but the chamber is not enlarged, and no other heart disease is present.
  • Have a heart‑wall thickness measured by the screening doctor that is:
    • at least 15 mm in any wall segment, or
    • at least 13 mm in any wall segment and have a known disease‑causing genetic change or a family history of HCM.
    • Have a resting left ventricular outflow tract gradient (LVOT‑G) of 30 mmHg or higher, or a gradient of 50 mmHg or higher after performing a Valsalva maneuver (a breathing technique used to stress the heart).
    • Have a left ventricular ejection fraction (LVEF) of 60% or higher, which measures how well the heart pumps blood.
    • Be classified as New York Heart Association (NYHA) Functional Class I, meaning you have no symptoms during ordinary activities.
    • Have a body mass index (BMI) lower than 35 kg/m² (a measure of body weight relative to height).
    • Be willing to follow the study’s contraception guidelines during the trial.
    • Have a blood hemoglobin level of at least 10 g/dL, which indicates adequate red blood cell count.

Who cannot join the trial?

14 criteria

  • You cannot join the study if you have a known or suspected infiltrative, genetic or storage disorder that makes the heart muscle thick and looks like the condition being studied.
  • You cannot join if you cannot tolerate a PET‑CT scan, for example because you cannot lie still or you have severe claustrophobia (fear of closed spaces).
  • You cannot join if you are allergic to any therapeutic radiopharmaceuticals (special medicines used in imaging scans).
  • You cannot join if your kidney function is poor, measured as an eGFR (estimated glomerular filtration rate) below 25 ml/min/1.73 m², which means the kidneys do not filter waste well.
  • You cannot join if you have any other disease, metabolic problem, physical exam finding, or lab result that could affect the study results or make participation very difficult, such as severe, unmanageable claustrophobia.
  • You cannot join if you have a serious other illness, cancer, active infection, or an allergy/hypersensitivity to the study drug aficamten or any ingredient in the placebo, or any condition that the doctor believes would be unsafe or would interfere with the study.
  • You cannot join if you have significant valvular heart disease (problems with the heart’s valves) as judged by the investigator.
  • You cannot join if you have a history of left‑ventricular systolic dysfunction, meaning the heart’s pumping ability is reduced with an LVEF (left ventricular ejection fraction) less than 45%.
  • You cannot join if you have previously had, or plan to have during the study, septal reduction therapy (a procedure to shrink part of the heart muscle).
  • You cannot join if you are currently pregnant or breastfeeding.
  • You cannot join if you have an untreated narrowing of a coronary artery that is 70% or more (coronary artery stenosis) or if you have a documented history of a Type 1 myocardial infarction (heart attack).
  • You cannot join if you have previously taken the study drug aficamten or a similar drug called mavacamten.
  • You cannot join if you have paroxysmal or permanent atrial fibrillation (an irregular heart rhythm), unless the principal investigator decides otherwise on a case‑by‑case basis.
  • You cannot join if you have a history of syncope (fainting) or a dangerous fast heart rhythm called sustained ventricular tachyarrhythmia that occurred during exercise within the past six months.
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Investigated drugs

  • MYQORZO

    is an oral tablet that contains the active ingredient aficamten. Aficamten is designed to gently relax the heart muscle, which may lower the pressure that builds up when blood leaves the heart in people with hypertrophic cardiomyopathy. In this study the drug is given to see if it can reduce the left‑ventricular outflow tract gradient after a Valsalva maneuver, which is a simple breathing test.

  • 68GA-FAPI-46

    is a special imaging agent that is injected into a vein. It contains a tiny amount of a radioactive substance that allows doctors to take detailed pictures of the heart with a PET scan. The images help researchers see how the heart tissue is behaving and whether the treatment with aficamten is affecting the heart’s structure or blood flow.

What is already known about the treatment

  • MYQORZO 5 mg film-coated tablets

    This medication is taken by mouth as a film‑coated tablet, typically 20 mg per dose. It is an investigational drug that is still being studied in clinical trials and has not yet received market approval. The main use being tested is for treating hypertrophic cardiomyopathy, a condition where the heart muscle is abnormally thick. It works by gently blocking the activity of cardiac myosin, the protein that makes heart muscle contract, which helps lower the pressure that builds up when the heart pumps blood. It belongs to the class of cardiac myosin inhibitors (also called myosin modulators).

  • 68GA-FAPI-46

    This agent is given as an injection into a vein, delivering a liquid solution that contains a radioactive tracer. It is also investigational and is used only for research imaging studies, not as a regular medicine. The tracer is designed to attach to a protein called fibroblast activation protein, which is found on certain cells involved in tissue scarring, allowing doctors to see those areas on a PET scan. Because it carries a small amount of the radioactive isotope gallium‑68, it is classified as a radiopharmaceutical used for diagnostic imaging.

  • Placebo

    The placebo is a tablet that looks the same as the study drug but contains no active ingredient and is taken by mouth. It is used only as a control in the trial to compare the effects of the active medication. Since it has no therapeutic substance, it has no clinical use outside of research studies. It is considered an inert substance and does not belong to any pharmacological class.

Investigated diseases

Hypertrophic cardiomyopathy - Hypertrophic cardiomyopathy is a condition where the heart muscle, especially the walls of the left ventricle, becomes unusually thick. The thickened muscle can make the chamber smaller and may impede the flow of blood out of the heart. Over time the thickness often increases, which can lead to a narrowing of the outflow tract in some people. The disease may remain without symptoms for many years, but the structural changes can gradually affect how the heart fills and pumps. In some individuals the thickening spreads to other parts of the heart, and the rhythm of the heartbeat can become irregular as the condition progresses.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-523090-42-00Protocol codeLEAF-001Estimated enrolment50 patientsSponsorErasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

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