A Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

In brief

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.

Key facts

Study ID

NCT06081894

Run by

Cytokinetics

People needed

500

Starts

2023-08-31

Expected to finish

2026-10-01

Last updated by the study team

2026-08-05

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• ≥ 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 AND
• Resting LVOT-G < 30 mmHg AND Valsalva LVOT-G < 50 mmHg AND
• LVEF ≥ 60%
• 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:
• NT-pro BNP ≥ 300 pg/mL or NT-proBNP ≥ 900 pg/mL if in atrial fibrillation or atrial flutter OR
• For Black participants, an NT-pro BNP ≥ 225 pg/mL or NT-proBNP ≥ 675 pg/mL if in atrial fibrillation or atrial flutter

You may not qualify if…

• Significant valvular heart disease (per Investigator judgment)
• Moderate or severe valvular aortic stenosis or fixed subaortic obstruction
• Moderate or severe mitral regurgitation
• 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:
• rhythm restoring treatment (e.g., direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤ 3 months prior to screening
• rate control and anticoagulation have not been achieved for at least 3 months prior to screening.

Where it is running

• UC San Diego Health - Sulpizio Cardiovascular Center — La Jolla, California, United States
• Keck Medical Center of USC (Outpatient Clinic) — Los Angeles, California, United States
• Cedars-Sinai Medical Center (Smidt Heart Institute) — Los Angeles, California, United States
• UCLA Medical Center Cardiovascular Clinic — Los Angeles, California, United States
• University of California San Francisco — San Francisco, California, United States
• Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center — Torrance, California, United States
• Yale New Haven Hospital — New Haven, Connecticut, United States
• MedStar Washington Hospital Center — Washington D.C., District of Columbia, United States
• Holy Cross Medical Group - Cardiology Associates — Fort Lauderdale, Florida, United States
• Investigational Drug Services, AdventHealth Orlando — Orlando, Florida, United States
• Emory Clinic — Atlanta, Georgia, United States
• Piedmont Heart Institute (CPET) — Fayetteville, Georgia, United States
• The Queen's Medical Center-Punchbowl — Honolulu, Hawaii, United States
• Northwestern University — Evanston, Illinois, United States
• Ascension St. Vincent — Indianapolis, Indiana, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• The University of Kansas Medical Center — Kansas City, Kansas, United States
• MedStar Union Memorial Hospital — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Brigham and Women's Hospital — Boston, Massachusetts, United States
• Lahey Hospital & Medical Center — Burlington, Massachusetts, United States
• Michigan Medicine — Ann Arbor, Michigan, United States
• Henry Ford Hospital — Detroit, Michigan, United States
• M Health Fairview University of Minnesota Medical Center - East Bank — Minneapolis, Minnesota, United States
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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