A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.
Inclusion Criteria:
Between 18-85 years of age
Body mass index < 40 kg/m2
Diagnosed with nHCM and has a screening echocardiogram with the following:
End-diastolic left ventricular (LV) wall thickness:
Participants with a history of intracavitary obstruction are eligible.
NYHA class II or III
Respiratory exchange ratio of ≥ 1.00 at screening by cardiopulmonary exercise testing (CPET) and predicted peak oxygen uptake (pVO2) ≤ 90% for age and sex
KCCQ-CSS score of ≤ 85
NT-proBNP of:
Exclusion Criteria:
Significant valvular heart disease (per Investigator judgment)
Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM (eg, Noonan syndrome, Fabry disease, amyloidosis)
Known current unrevascularized coronary artery stenosis of ≥ 70% or documented history of myocardial infarction.
History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy
Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
Documented room air oxygen saturation reading < 90% at screening or history of significant chronic obstructive pulmonary disease or severe/significant pulmonary hypertension
History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia with exercise within 3 months prior to screening
History of resistant hypertension (persistently elevated blood pressure despite maximal doses of 3 or more classes of medications for hypertension control)
Screening diastolic blood pressure ≥ 100 mmHg
Received prior treatment with aficamten
Received treatment with mavacamten within 3 months prior to screening (must be discussed with the medical monitor prior to screening)
Undergone septal reduction therapy < 6 months prior to screening
Is being considered for or is likely to be considered for heart transplant listing or left ventricular assist device placement during the study period
Paroxysmal or permanent atrial fibrillation is excluded only if:
Torrance, California 90502, United States
Minneapolis, Minnesota 55455, United States
New York, New York 10032, United States
Spokane, Washington 99204, United States
São Paulo, 12916-542, Brazil
São Paulo, 13060-080, Brazil
São Paulo, 14051-140, Brazil
Berlin, 13353, Germany
Heidelberg, 69120, Germany
Jena, 07747, Germany
San Donato Milanese, Milano 20097, Italy
Florence, 50134, Italy
Foggia, 71122, Italy
Milan, 20149, Italy
Milan, 20162, Italy
Naples, 80131, Italy
Pisa, 56124, Italy
Roma, 00163, Italy
Lisbon, 1150-199, Portugal
Madrid, 28007, Spain