Mavacamten in non-obstructive HCM

Phase 3

Recruiting

• Conditions: symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)

• Registration Number: JPRN-jRCT2051220175
• Lead Sponsor: Aronson Ron

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-02-23
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 34

Inclusion 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

Exclusion 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
-Change from baseline in Kansas City Cardiomyopathy Questionnaire (23-item) Clinical Summary Score (KCCQ-23 CSS) at Week 52 [ Time Frame: Up to Week 52 ]<br>-Change from baseline in peak oxygen consumption (pVO2) at Week 52 [ Time Frame: Up to Week 52 ]