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

NCT ID: NCT07023341

Last Updated: 2026-01-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-30

Study Completion Date

2028-05-30

Brief Summary

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Researchers are looking for a better way to treat Japanese people who have symptomatic obstructive hypertrophic cardiomyopathy (symptomatic oHCM).

Obstructive hypertrophic cardiomyopathy (oHCM) is a type of heart disease where the heart muscles become thicker than normal due to over contraction. This thickening makes it harder than normal due to over contraction. This thickening makes it harder for the heart to pump blood out to the rest of the body. In symptomatic oHCM people with the condition experience symptoms like shortness of breath, chest pain, fainting, high blood pressure and irregular heartbeats.

The study treatment aficamten, also called BAY3723113, is under development to treat symptomatic oHCM. It aims to reduce the activity of cardiac myosin, a protein that helps heart muscles to contract, and thereby preventing over contraction and muscle thickening.

Although treatment options are available for symptomatic oHCM, there is still need for other treatment options that help target the root cause of the condition. In this study, researchers want to understand about the effects and long-term safety of aficamten in Japanese people with symptomatic oHCM.

The main purpose of the study is to learn how well aficamten works in Japanese with symptomatic oCHM.

For this, the researchers will check how participant's heart blood flow changes after 6 months of treatment. They do this by measuring the pressure needed for blood to leave the heart using a test called the left ventricular outflow tract (LVOT) gradient and a special breathing technique called Valsava maneuver.

Researchers will also look for:

* the number of participants who will have at least 1 level improvement on a scale doctors use to assess the effect of heart problems on daily activities after 3 and 6 months of treatment
* the change in the impact of heart problems on participant's daily lives based on their feedback on a questionnaire called Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS) after 3 and 6 months of treatment.

This study will have 2 treatment periods: main treatment period and long-term treatment period. During the main treatment period, participants will take aficamten tablets once daily by mouth for up to 6 months. After completing this period, the participants who can join the long-term treatment period will continue taking aficamten until the drug becomes commercially available in Japan or the study ends.

Each participant will be in the study as long as they benefit from the treatment.

Participants will visit the study site:

* once before the treatment starts
* 9 times with a gap of 2 to 4 weeks between the visits during treatment under the main treatment period, and in the long-term treatment period, participants will visit almost every 3 months until the treatment ends.
* then 2 more times with a gap of 1 month between the visits after the treatment ends.

During the study, the study doctors and their team will:

* check participant's health by performing tests such as blood and urine tests, and checking heart health using an electrocardiogram (ECG) and echocardiogram (ECHO)
* ask the participants questions about how they are feeling and what adverse events are they having

An adverse event is any medical problem that a participant has during a study. Study doctors keep track of all adverse events, irrespective if they think it is related or not to the study treatment. In addition, the participants will be asked to complete a questionnaire on quality of life at certain time points during the study.

If the participant benefits from the treatment, treatment with aficamten after the end of the study might be possible.

Detailed Description

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Conditions

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Obstructive Hypertrophic Cardiomyopathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Aficamten (BAY3723113)

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 echocardiography assessments, for up to 24 weeks. Participants will receive a dose of Aficamten until the drug becomes commercially available in Japan or the study ends.

Group Type EXPERIMENTAL

BAY3723113

Intervention Type DRUG

Oral tablet

Interventions

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BAY3723113

Oral tablet

Intervention Type DRUG

Other Intervention Names

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CK-3773274

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Kurume University Hospital

Kurume, Fukuoka, Japan

Site Status RECRUITING

Hyogo Prefectural HarimaHimeji General Medical Center

Himeji, Hyōgo, Japan

Site Status RECRUITING

Kobe City Medical Center General Hospital

Kobe, Hyōgo, Japan

Site Status RECRUITING

University of Tsukuba Hospital

Tsukuba, Ibaraki, Japan

Site Status RECRUITING

Iwate Prefectural Central Hospital

Morioka, Iwate, Japan

Site Status RECRUITING

Kitasato University Hospital

Sagamihara, Kanagawa, Japan

Site Status RECRUITING

SHOWA Medical University Fujigaoka Hospital

Yokohama, Kanagawa, Japan

Site Status RECRUITING

Kochi Medical School Hospital

Nankoku, Kochi, Japan

Site Status RECRUITING

Mie University Hospital

Tsu, Mie-ken, Japan

Site Status RECRUITING

Tohoku University Hospital

Sendai, Miyagi, Japan

Site Status RECRUITING

Saiseikai Kumamoto Hospital

Kumamoto, Mumamoto, Japan

Site Status RECRUITING

Kurashiki Central Hospital

Kurashiki, Okayama-ken, Japan

Site Status NOT_YET_RECRUITING

University of the Ryukyus Hospital

Ginowan, Okinawa, Japan

Site Status RECRUITING

Matsushita Memorial Hospital

Moriguchi, Osaka, Japan

Site Status RECRUITING

National Cerebral and Cardiovascular Center

Suita, Osaka, Japan

Site Status RECRUITING

The University of Osaka Hospital

Suita, Osaka, Japan

Site Status RECRUITING

Hamamatsu University Hospital

Hamamatsu, Shizuoka, Japan

Site Status RECRUITING

Nippon Medical School Hospital

Bunkyo-ku, Tokyo, Japan

Site Status RECRUITING

The University of Tokyo Hospital

Bunkyo, Tokyo, Japan

Site Status RECRUITING

Sakakibara Heart Institute

Fuchū, Tokyo, Japan

Site Status RECRUITING

Showa Medical University Koto Toyosu Hospital

Koto, Tokyo, Japan

Site Status RECRUITING

Saiseikai Fukuoka General Hospital

Fukuoka, , Japan

Site Status RECRUITING

Gifu University Hospital

Gifu, , Japan

Site Status NOT_YET_RECRUITING

Kumamoto University Hospital

Kumamoto, , Japan

Site Status RECRUITING

University Hospital, Kyoto Prefectural University of Medicine

Kyoto, , Japan

Site Status RECRUITING

Countries

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Japan

Central Contacts

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Bayer Clinical Trials Contact

Role: CONTACT

(+)1-888-84 22937

Related Links

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https://clinicaltrials.bayer.com/study/22976

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Other Identifiers

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22976

Identifier Type: -

Identifier Source: org_study_id

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