NIPH Clinical Trials Search

Open label, single arm study of aficamten in Japanese oHCM

Basic Information

Recruitment status	Not Recruiting
Health condition(s) or Problem(s) studied	symptomatic obstructive hypertrophic cardiomyopathy
Date of first enrollment	30/06/2025
Target sample size	34
Countries of recruitment
Study type	Interventional
Intervention(s)	Aficamten will be administered orally once daily with or without food. Participants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of Aficamten with dose levels guided by echocardiographt assessments, for up to 24 weeks. Participants will receive a dose of Aficamten until the drug becomes commercially available in Japan or study ends.

Outcome(s)

Primary Outcome	Change in Valsalva LVOT-G from baseline to Week 24
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	<= 85age old
Gender	Both
Include 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: a) Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and b) 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.
Exclude criteria	-Significant valvular heart disease (per investigator judgement) a) Moderate-severe valvular aortic stenosis and/or regurgitation b) 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