Mavacamten in non-obstructive HCM

JRCT ID: jRCT2051220175

Registered date:23/02/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)
• Date of first enrollment: 30/03/2023
• Target sample size: 34
• Countries of recruitment: Australia,Japan,Austria,Japan,Belgium,Japan,Brazil,Japan,Canada,Japan,Czech Republic,Japan,Denmark,Japan,France,Japan,Germany,Japan,Hungary,Japan,India,Japan,Israel,Japan,Italy,Japan,Korea,Japan,Netherlands,Japan,Norway,Japan,Poland,Japan,Portugal,Japan,Spain,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Mavacamten Arm: Mavacamten Participants will receive treatment with 1, 2.5, 5, 10, 15 mg dose once daily.
Placebo Arm: Placebo Participants will receive treatment once daily.

Outcome(s)

Primary Outcome

-Change from baseline in Kansas City Cardiomyopathy Questionnaire (23-item) Clinical Summary Score (KCCQ-23 CSS) at Week 48 [ Time Frame: Up to Week 48 ]
-Change from baseline in peak oxygen consumption (pVO2) at Week 48 [ Time Frame: Up to Week 48 ]

Secondary Outcome

-Change from baseline in ventilatory efficiency (VE/VCO2) slope to Week 48 [ Time Frame: Up to Week 48 ]
-Proportion of participants with at least 1 class of New York Heart Association (NYHA) improvement from baseline to Week 48 [ Time Frame: Up to Week 48 ]
-Change from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) to Week 52 [ Time Frame: Up to Week 48 ]
-Change from baseline in cardiac troponin-T (cTn-T) to Week 48 [ Time Frame: Up to Week 48 ]
-Change from baseline in hypertrophic cardiomyopathy symptom questionnaire-shortness of breath (HCMSQ-SoB) domain to Week 48 [ Time Frame: Up to Week 48 ]
-Time to first major adverse cardiovascular events (MACE)-plus events defined as any cardiovascular (CV) death, nonfatal myocardial infarction, non-fatal stroke, hospitalization for heart failure, or hospitalization for arrhythmias [ Time Frame: Up to 120 Weeks ]

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

-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 left ventricular (LV) wall thickness >= 15 millimeters (mm) (or >= 13 mm with positive family history of hypertrophic cardiomyopathy [HCM]) as determined by core laboratory interpretation
-Peak left ventricular outflow tract (LVOT) pressure gradient < 30 millimeters mercury (mm Hg) at rest and < 50 mm Hg with provocation (Valsalva maneuver and stress echocardiography)
-New York Heart Association (NYHA) Class II or III

Exclude criteria

-Known infiltrative or storage disorder causing cardiac hypertrophy that mimics non-obstructive hypertrophic cardiomyopathy (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

Related Information

• Primary Sponsor: Aronson Ron