A Study to Learn More About How Well Aficamten Works in Japanese Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Launched by BAYER · Jun 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medicine called aficamten to see how well it works for Japanese adults who have a heart condition called symptomatic obstructive hypertrophic cardiomyopathy (oHCM). In this condition, the heart muscle becomes thicker than normal, which makes it harder for the heart to pump blood properly. People with oHCM often experience symptoms like shortness of breath, chest pain, fainting, and irregular heartbeats. Aficamten aims to help by reducing the heart muscle’s over-contraction, potentially improving blood flow and easing symptoms.

Adults between 18 and 85 years old who are Japanese and have a confirmed diagnosis of oHCM may be eligible for this study. To join, participants must have specific heart measurements showing thickened heart muscle and some blockage to blood flow, along with symptoms that affect their daily activities. During the study, participants will take aficamten pills once a day for 6 months, with the option to continue longer if the treatment is helping. They will visit the study center regularly for health check-ups, heart tests, and to answer questions about how they’re feeling and any side effects they may experience. The researchers will measure changes in heart function and ask participants about their quality of life to better understand how aficamten may improve symptoms and daily living. If the medicine works well and is safe, participants might be able to keep taking it even after the study ends.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participant must be 18 to 85 years of age inclusive, at the time of signing the informed consent.
• * Diagnosed with HCM per the following criteria:
• • 1. Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and
• 2. Has an end-diastolic LV wall thickness as measured by the echocardiography core laboratory of:
• • 1. ≥15 mm in one or more myocardial segments OR
• • 2. ≥13 mm in one or more wall segments and a known-disease-causing gene mutation or positive family history of HCM
• • Has resting LVOT-G ≥ 30 mmHg and Valsalva LVOT-G =50 mmHg during screening as determined by the echocardiography core laboratory
• • LVEF ≥ 60% at screening as determined by the echocardiography core laboratory
• • NYHA Functional Class II or III at screening
• • Hemoglobin ≥10 g/dL at screening
• • Body mass index \<35 kg/m²
• • Japanese
• • Patients on beta-blockers, verapamil, diltiazem, or disopyramide/cibenzoline should have been on a stable regimen for \>6 weeks prior to the first dose of aficamten and anticipate remaining on the same medication regimen at least during the main study treatment period. Patients treated with disopyramide or cibenzoline must also be concomitantly treated with a beta blocker and/or calcium channel blocker.
• Exclusion Criteria:
• • Significant valvular heart disease (per investigator judgement)
• • 1. Moderate-severe valvular aortic stenosis and/or regurgitation
• • 2. Moderate-severe mitral regurgitation not due to systolic anterior motion of the mitral valve
• • Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics oHCM (e.g., Noonan syndrome, Fabry disease, amyloidosis)
• • History of LV systolic dysfunction (LVEF \<45%) or stress cardiomyopathy at any time during their clinical course
• • Documented paroxysmal atrial fibrillation during the screening period
• * Paroxysmal or persistent/permanent atrial fibrillation is only excluded IF:
• • Rhythm restoring treatment (e.g., direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤ 6 months prior to screening
• • Rate control and anticoagulation have not been achieved for at least 6 months prior to screening
• • Has been treated with SRT (surgical myectomy or percutaneous alcohol septal ablation) or cannot postpone plans for SRT until after the study period
• • History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to screening
• • Has received prior treatment with aficamten or mavacamten