A study in subjects with Symptomatic genetic heart disease comparing sponsors drug Mavacamten with dummy drug.

Phase 3

Conditions: Health Condition 1: I422- Other hypertrophic cardiomyopathy

Registration Number: CTRI/2023/10/058870

Lead Sponsor: MyoKardia, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Open to Recruitment

Sex: Not specified

Target Recruitment: 0

Inclusion Criteria

1) Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines: unexplained left-ventricular hypertrophy with non-dilated ventricular chambers in the absence of other cardiac or systemic disease which can produce the required magnitude of hypertrophy of a maximal LV wall thickness greater than or equal to 15 mm. 2) Peak LVOT pressure gradient < 30 mm Hg at rest and < 50 mm Hg with provocation. 3) New York Heart Association (NYHA) Class II or III. 4) KCCQ-23 CSS Score less than or equal to 80 at screening. 5) LVEF greater than or equal to 60%. 6) CPET: Documented oxygen saturation at rest > 90% at screening. 7) NT-proBNP greater than or equal to 200 pg/mL or BNP greater than or equal to 70 pg/mL. 8) Evidence of myocardial damage or Evidence of LV diastolic dysfunction.

Exclusion Criteria

1) Known infiltrative or storage disorder causing cardiac hypertrophy that mimics nHCM. 2) History of unexplained syncope within 6 months prior to screening. 3) History of sustained ventricular tachyarrhythmia ( > 30 seconds) within 6 months prior to screening. 4) Paroxysmal or persistent (non-permanent) AF detected at the time of screening. 5) ICD placement or pulse generator change within 2 months prior to screening or planned new ICD placement during the study. 6) Acute heart failure from 4 weeks prior to screening up to randomization. 7) Coronary artery disease requiring intervention. 8) Heart transplant recipient or listed for heart transplant. 9) Currently implanted LV assist device. 10) Clinically significant pulmonary disease associated with exertional dyspnea. 11) Any documented active or suspected malignancy or history of malignancy within 2 years prior to screening. 12) Clinically documented LV aneurysm greater than or equal to 2 cm

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1) To assess the efficacy of a 48-week course of <br/ ><br>mavacamten compared to placebo on patient reported health status <br/ ><br>2) To assess the efficacy of a 48-week course of <br/ ><br>mavacamten compared to placebo on exercise <br/ ><br>capacity <br/ ><br>Timepoint: 1) Change from baseline in KCCQ-23 CSS at Week 4 <br/ ><br> <br/ ><br>2) Change from baseline in pVO2 at Week 48

Secondary Outcome Measures