account_circleRecruiting

obstructive hypertrophic cardiomyopathy

Bayer Identifier:
22976
ClinicalTrials.gov Identifier:
NCT07023341
EudraCT Number:
Not Available
EU CT Number:
Not Available
A study to learn more about how well Aficamten works in Japanese participants with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Trial purpose

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.

Key Participants Requirements

Sex

All

Age

18 - 85 Years
  • - 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.
  • - 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

Trial summary

Enrollment Goal
34
Trial Dates
June 2025 - April 2028
Phase
Phase 3
Could I Receive a placebo
No
Products
Aficamten (BAY3723113)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Not yet recruiting
Kochi Medical School HospitalNankoku, 783-8505, Japan
Not yet recruiting
Hamamatsu University HospitalHamamatsu, 431-3192, Japan
Not yet recruiting
National Cerebral and Cardiovascular CenterSuita, 564-8565, Japan
Recruiting
Saiseikai Fukuoka General HospitalFukuoka, 810-0001, Japan
Not yet recruiting
Kurashiki Central HospitalKurashiki, 710-8602, Japan
Not yet recruiting
Kumamoto University HospitalKumamoto, 860-8556, Japan
Not yet recruiting
SHOWA Medical University Fujigaoka HospitalYokohama, 227-8501, Japan
Not yet recruiting
University of Tsukuba HospitalTsukuba, 305-8576, Japan
Not yet recruiting
Showa Medical University Koto Toyosu HospitalKoto, 135-8577, Japan
Not yet recruiting
Sakakibara Heart InstituteFuchu, 183-0003, Japan
Not yet recruiting
The University of Tokyo HospitalBunkyo, 113-8655, Japan
Not yet recruiting
Hyogo Prefectural HarimaHimeji General Medical CenterHimeji, 670-8560, Japan
Not yet recruiting
Kobe City Medical Center General HospitalKobe, 650-0047, Japan
Not yet recruiting
Nippon Medical School HospitalBunkyo-ku, 113-8602, Japan
Not yet recruiting
Saiseikai Kumamoto HospitalKumamoto, 861-4193, Japan
Not yet recruiting
University of the Ryukyus HospitalGinowan City, 901-2725, Japan
Not yet recruiting
Gifu University HospitalGifu, 501-1194, Japan
Not yet recruiting
Matsushita Memorial HospitalMoriguchi, 570-8540, Japan
Not yet recruiting
Kitasato University HospitalSagamihara, 252-0375, Japan
Not yet recruiting
Iwate Prefectural Central HospitalMorioka-shi, 020-0066, Japan
Not yet recruiting
University Hospital, Kyoto Prefectural University of MedicineKyoto, 602-8566, Japan
Not yet recruiting
Mie University HospitalTsu, 514-8507, Japan
Not yet recruiting
Tohoku University HospitalSendai, 980-8574, Japan
Not yet recruiting
The University of Osaka HospitalSuita, 565-0871, Japan
Not yet recruiting
Kurume University HospitalKurume City, 830-0011, Japan

Primary Outcome

  • Change in Valsava LVOT-G
    date_rangeTime Frame:
    From baseline to Week 24

Secondary Outcome

  • Change in resting LVOT-G
    date_rangeTime Frame:
    From baseline to Weeks 12 and 24
  • Change in Valsava LVOT-G
    date_rangeTime Frame:
    From baseline to Week 12
  • Change in N-terminal prohormone brain natriuretic peptide (NT-proBNP) value
    date_rangeTime Frame:
    From baseline to Week 24
  • Proportion of patricipants with ≥1 class improvement in New York Heart Association (NYHA) Functional Class
    date_rangeTime Frame:
    From baseline to Weeks 12 and 24
  • Change in KCCQ-CSS score
    date_rangeTime Frame:
    From baseline to weeks 12 and 24
  • Incidence of left ventricular ejection fraction (LVEF) <40%
    date_rangeTime Frame:
    From baseline to last follow up, up to 4 weeks
  • Incidence of LVEF <50%
    date_rangeTime Frame:
    From baseline to last follow up, up to 4 weeks
  • Incidence of treatment emergent adverse events (TEAE)
    date_rangeTime Frame:
    From baseline to last follow up, up to 4 weeks
  • Incidence of treatment emergent serious adverse events (TESAE)
    date_rangeTime Frame:
    From baseline to last follow up, up to 4 weeks

Trial design

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
Trial Type
Interventional
Intervention Type
Drug
Trial Purpose
Treatment
Allocation
N/A
Blinding
N/A
Assignment
Single Group Assignment
Trial Arms
1

Additional Information

Click here to find further information and, after study completion, the study results according to Bayer's transparency standards.