Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM (SEQUOIA-HCM)

Trial ID or NCT#

NCT05186818

Status

not recruiting iconNOT RECRUITING

Purpose

The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) in adults with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction

Official Title

A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

Eligibility Criteria

Ages Eligible for Study: 18 Years to 85 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * Males and females between 18 and 85 years of age, inclusive, at screening.* Body mass index \<35 kg/m2.* Diagnosed with HCM per the following criteria:
  2. * 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:
  3. * ≥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 post-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 ≥10g/dL at screening.* Respiratory exchange ratio (RER) ≥1.05 and pVO2 ≤90% predicted on the screening CPET per the core laboratory.* Patients on beta-blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for \>6 weeks prior to randomization and anticipate remaining on the same medication regimen during the trial. Patients treated with disopyramide must also be concomitantly treated with a beta blocker and/or calcium channel blocker.
    1. Key
Exclusion Criteria:
  1. * Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics oHCM (eg, Noonan syndrome, Fabry disease, amyloidosis).* Significant valvular heart disease (per investigator judgment).
  2. * Moderate-severe valvular aortic stenosis. * Moderate-severe mitral regurgitation not due to systolic anterior motion of the mitral valve.* History of LV systolic dysfunction (LVEF \<45%) or stress cardiomyopathy at any time during their clinical course.* Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations).* Has been treated with septal reduction therapy (surgical myectomy or percutaneous alcohol septal ablation) or has plans for either treatment during the trial period.* Documented paroxysmal atrial fibrillation during the screening period.* Paroxysmal or permanent atrial fibrillation is only excluded IF:
  3. * rhythm restoring treatment (eg, 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.* History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to screening.* Has received prior treatment with CK-3773274 or mavacamten.
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Investigator(s)

Matthew Wheeler
Matthew Wheeler
Cardiologist, Heart failure cardiologist
Associate Professor of Medicine (Cardiovascular Medicine)
Masataka Kawana
Cardiologist, Heart failure cardiologist
Assistant Professor of Medicine (Cardiovascular Medicine)

Contact us to find out if this trial is right for you.

Contact

Jennifer Marino
650-725-6911

View on ClinicalTrials.gov