A randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of ninerafaxstat in patients with symptomatic non-obstructive hypertrophic cardiomyopathy

Prior treatment

• Cardiovascular Conditions: • History of obstructive hypertrophic cardiomyopathy (HCM) with left ventricular outflow tract (LVOT) gradient ≥30 mmHg at rest or with provocation. • History of major adverse cardiovascular events (MACE) within 6 months prior to screening, including myocardial infarction, stroke, or cardiac surgery.
• Medications: • Use of disallowed medications that may interfere with the trial, such as other investigational drugs within 30 days prior to screening. • Requirement for treatment with drugs that may affect the study outcomes, such as certain antiarrhythmic or antihypertensive medications.
• Other Exclusions: • Participation in another clinical trial within 30 days prior to screening. • Any condition that, in the opinion of the investigator, would make participation in the trial unsafe or otherwise not in the best interest of the participant.

Other illness

• Medical Conditions: • Significant comorbidities that may impact the ability to complete the trial or the interpretation of results, such as uncontrolled diabetes, severe renal impairment, or active infection. • Known history of hepatitis B or C infection, or HIV infection.
• Substance Abuse: History of alcohol or substance abuse within the past year.

Other

• All exclusion criteria can be found in the protocol (Section 5.2). 1. Informed Consent: Inability or unwillingness to provide informed consent or comply with trial procedures.
• Pregnancy and Lactation: • Pregnant or breastfeeding women. • Women of childbearing potential not willing to use effective contraception during the study and for a specified period after the last dose.

• All inclusion criteria can be found in the protocol (Section 5.1). 1. Informed Consent: Able to comprehend and willing to sign an informed consent form (ICF), and willing to comply with all trial procedures and restrictions for the duration specified in the Schedule of Events. Understands the risks involved.
• Age: Individual ≥18 years of age at the time of signing informed consent.
• Echocardiography: Has adequate acoustic windows for echocardiography.
• Clinical Diagnosis: Has a clinical diagnosis of hypertrophic cardiomyopathy (HCM) consistent with current American College of Cardiology/American Heart Association and European Society of Cardiology Guideline definition, with unexplained left ventricular hypertrophy (LVH) in the absence of other cardiac or systemic diseases that can produce the magnitude of hypertrophy observed.