A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Cardiomyopathy

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 420

Inclusion Criteria

1. Participants must be 18 years old or local age of majority at the time of signing the informed consent
2. Female participants must adhere to highly effective contraceptive methods or have documented proof that they are not of childbearing potential
3. No additional contraceptive measures are required to be used for male participants
4. Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines.
5. Peak LVOT pressure gradient < 30 mmHg at rest and < 50 mmHg with provocation (Valsalva maneuver and stress echocardiography)
6. CPET: Documented oxygen saturation at rest >90% at Screening. Able to perform an upright cardiopulmonary stress test (CPET) and has a respiratory exchange ratio (RER) = 1.0 at Screening per central reading. If the RER is between 0.91 and 1.0, the participant may be enrolled if the central CPET laboratory determines that peak exercise has been achieved. Participants with subpeak performance may not be enrolled as described in the CPET Laboratory Manual.
7. New York Heart Association (NYHA) Class II or III
8. NT-proBNP=200 pg/mL or BNP=70 pg/mL
9. LVEF =60 % as determined by the echocardiography central laboratory

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 400
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

Medical Conditions
- Known infiltrative or storage disorder causing cardiac hypertrophy that mimics nHCM such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
- History of unexplained syncope within 6 months prior to screening
- History of sustained ventricular tachyarrhythmia (> 30 seconds) within 6 months prior to Screening
- Paroxysmal or persistent (non-permanent) AF detected at the time of screening. Permanent AF or history of paroxysmal or persistent AF is allowed if the participant is anticoagulated and the investigator considers the heart rate adequately controlled
- CV diseases or treatments that in the opinion of the investigator increase the unpredictability of or change the participants’ clinical course.
- Acute heart failure from 4 weeks prior to screening up to randomization
- Myocardial infarction (increase in cardiac enzymes in combination with symptoms of ischemia or new ischemic ECG changes), coronary artery bypass graft surgery, or other major CV surgery, stroke, or transient ischemic attack in the past 90 days
- Women who are breastfeeding or pregnant.

Prior/Concomitant Therapy
- Any adjustments of beta-blockers, verapamil, or diltiazem within 2 weeks prior to Screening and up to the day of randomization
- Concomitant use of strong inhibitors of cytochrome P450 (CYP) 2C19

Other Exclusion Criteria
- Any other serious condition that in the opinion of the investigator could prevent participation in the study and follow-up
- Completed a study with an investigational device or drug < 30 days or 5 half-lives prior to screening
- Participants who have completed a study with mavacamten or aficamten

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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