A Phase 3, Open Label, Single Arm Study to Evaluate Efficacy, Safety and Tolerability of Aficamten in Adult Japanese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Status: Recruiting
Location: See all (25) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 85
Healthy Volunteers: f
View:

• 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:

‣ Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and

⁃ Has an end-diastolic LV wall thickness as measured by the echocardiography core laboratory of:

• ≥15 mm in one or more myocardial segments OR

∙ ≥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.

Locations
Other Locations
Japan
The University of Tokyo Hospital
NOT_YET_RECRUITING
Bunkyo
Nippon Medical School Hospital
RECRUITING
Bunkyo-ku
Sakakibara Heart Institute
RECRUITING
Fuchū
Saiseikai Fukuoka General Hospital
RECRUITING
Fukuoka
Gifu University Hospital
NOT_YET_RECRUITING
Gifu
University of the Ryukyus Hospital
NOT_YET_RECRUITING
Ginowan
Hamamatsu University Hospital
NOT_YET_RECRUITING
Hamamatsu
Hyogo Prefectural HarimaHimeji General Medical Center
RECRUITING
Himeji
Kobe City Medical Center General Hospital
RECRUITING
Kobe
Showa Medical University Koto Toyosu Hospital
RECRUITING
Koto
Kumamoto University Hospital
NOT_YET_RECRUITING
Kumamoto
Saiseikai Kumamoto Hospital
RECRUITING
Kumamoto
Kurashiki Central Hospital
NOT_YET_RECRUITING
Kurashiki
Kurume University Hospital
NOT_YET_RECRUITING
Kurume
University Hospital, Kyoto Prefectural University of Medicine
RECRUITING
Kyoto
Matsushita Memorial Hospital
NOT_YET_RECRUITING
Moriguchi
Iwate Prefectural Central Hospital
RECRUITING
Morioka
Kochi Medical School Hospital
NOT_YET_RECRUITING
Nankoku
Kitasato University Hospital
RECRUITING
Sagamihara
Tohoku University Hospital
NOT_YET_RECRUITING
Sendai
National Cerebral and Cardiovascular Center
NOT_YET_RECRUITING
Suita
The University of Osaka Hospital
RECRUITING
Suita
Mie University Hospital
NOT_YET_RECRUITING
Tsu
University of Tsukuba Hospital
RECRUITING
Tsukuba
SHOWA Medical University Fujigaoka Hospital
RECRUITING
Yokohama
Contact Information
Primary
Bayer Clinical Trials Contact
clinical-trials-contact@bayer.com
(+)1-888-84 22937
Time Frame
Start Date: 2025-06-30
Estimated Completion Date: 2028-05-30
Participants
Target number of participants: 34
Treatments
Experimental: 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.
Sponsors
Leads: Bayer

This content was sourced from clinicaltrials.gov