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

Phase 3

Recruiting

Conditions: Symptomatic hardening of the left ventricle
Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
10010394
10007510

Registration Number: NL-OMON56426

Lead Sponsor: Myokardia, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 31

Inclusion Criteria

1. Participants must be at least 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 10. KCCQ-23 CSS Score <= 85 at screening

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
Note: Investigators should not screen participants who have comprehensive Echo
features suggestive of amyloidosis, including
abnormal global longitudinal strain in the setting of an appropriate clinical
picture which could include low voltage on ECG and severely elevated NT-proBNP
or BNP
- 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 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 - Coronary artery
disease requiring intervention, including 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 Note: Use should be discontinued for a minimum of 5
elimination half-lives prior to first dose of study intervention. Other
Exclusion Criteria - Any other serious condition that in the opinion of the
investigator could prevent participation in the study and follow-up, including
active infection with COVID-19 from 4 weeks prior to screening up to
randomization - Completed a study with an investigational device < 30 days
prior to screening or an investigational drug <5 half-lives prior to screening
- Participants who have completed a study with mavacamten or aficamten
--Enrolled in another study and receiving any investigational treatment (device
or drug) other than the study intervention given in this study