Status:
ACTIVE_NOT_RECRUITING
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM
Lead Sponsor:
Cytokinetics
Collaborating Sponsors:
Sanofi
Bayer
Conditions:
Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
Eligibility:
All Genders
18-85 years
Phase:
PHASE3
Brief Summary
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.
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
August 30 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 1 2026
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT06081894
Start Date
August 30 2023
End Date
September 1 2026
Last Update
October 29 2025
Active Locations (180)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
UC San Diego Health - Sulpizio Cardiovascular Center
La Jolla, California, United States, 92037
3
Keck Medical Center of USC (Outpatient Clinic)
Los Angeles, California, United States, 90033
4
Cedars-Sinai Medical Center (Smidt Heart Institute)
Los Angeles, California, United States, 90048