Obstructive Hypertrophic Cardiomyopathy (oHCM)

The study will describe how mavacamten is used in routine care for Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), including use as monotherapy or with other background/concomitant oHCM medications.

This multicenter, randomized, open-label, parallel-group, non-inferiority trial will evaluate whether sequential tapering and discontinuation of beta-blockers is non-inferior to maintenance of the baseline stable beta-blocker dose in adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who have achieved predefined clinical and hemodynamic stability during mavacamten treatment. Eligible participants will be randomly assigned in a 1:1 ratio to a sequential tapering and discontinuation group or a baseline stable-dose maintenance group. In the tapering group, the beta-blocker dose will be reduced stepwise approximately every 4 weeks, with complete discontinuation planned at Week 12 if predefined safety and stability criteria are met. Participants will be followed through Week 24. The primary objective is to compare the proportion of participants who maintain clinical and hemodynamic stability without protocol-defined treatment failure through Week 24. The feasibility and safety of complete beta-blocker discontinuation will also be evaluated.

This study aims to evaluate whether, under a treatment strategy primarily guided by left ventricular ejection fraction (LVEF), dose up-titration in adult patients with symptomatic obstructive hypertrophic cardiomyopathy who have received Mavacamten treatment for at least 12 weeks with a stable maintenance dose (as baseline) and with LVEF ≥55% can further reduce left ventricular outflow tract (LVOT) gradient after 24 weeks of treatment. It will also assess changes in hemodynamics, clinical symptoms, cardiac structure, and function. Furthermore, the study will conduct stratified analyses based on patients' baseline resting or Valsalva-provoked LVOT gradient levels at enrollment \[\<30 mmHg vs. ≥30 mmHg\] to explore differences in the aforementioned outcome measures among patients with different gradient levels, with the goal of providing evidence for individualized clinical dose adjustment.

The purpose of this study is to understand the real-world clinical outcomes and treatment patterns of adults with obstructive hypertrophic cardiomyopathy (HCM) treated with mavacamten, and to understand patient and physician experiences with mavacamten treatment, in the US community-based practice

This study is a multicenter, double-blind, randomized controlled Phase 2 trial designed to evaluate the safety and efficacy of Ademetionine in patients with obstructive hypertrophic cardiomyopathy (oHCM). The study will recruit patients with oHCM who, under double-blind conditions, will be randomly assigned to either the Ademetionine group or the placebo group. Follow-up visits will be conducted every 4 weeks until 16 weeks from baseline. After 16 weeks, the study will evaluating the effect of Ademetionine on exercise capacity, heart failure symptoms, cardiac structure and function, and quality of life, as well as safety and tolerability of Ademetionine in this patient population.

The European Medicines Agency has required pre-treatment genotyping of CYP2C19 to determine the initial and maximum dosage, in order to avoid overexposure to mavacamten associated with a decrease in ventricular ejection fraction below 50% in slow metabolisers of CYP2C19 (AUC multiplied by 3.4). The study is a requalification for the search for DNA samples obtained during treatment in order to genotype the genes of interest.The primary objective of the study is to estimate, for each of the CYP2C19 phenotypes of interest determined by genotyping (ultra-rapid, rapid, normal/extensive and intermediate metabolisers), the proportion of patients who are non-responders to mavacamten at each time point (D0, Week 4, Week 8, Week 12 and Week 24 in the treatment of HCM). This is a multicentre (3 centres which are hospitals of APHP) study aiming to include 300 patients with obstructive hypertrophic cardiomyopathy treated with Mavacamten who underwent or are undergoing CYP2C19 genotyping at the start of treatment. The inclusion period is 36 months and the follow-up period is 6 months. The total duration of the study is 42 months.

The purpose of this study is to assess the real-world effectiveness and safety of mavacamten in patients with obstructive hypertrophic cardiomyopathy (HCM) receiving mavacamten in Japan