Global Phase 2b Study of Ninerafaxstat for Treating Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

What this Trial Is About

Researchers are evaluating the effects of ninerafaxstat in adults with symptomatic non-obstructive hypertrophic cardiomyopathy nHCM, a heart condition characterized by thickened heart muscle without obstruction. This global Phase 2b study aims to assess the safety and effectiveness of ninerafaxstat compared to a placebo, both given alongside standard care, to improve symptoms and heart function. The trial is double-blinded and placebo-controlled to ensure reliable comparisons between groups. Participants will be randomly assigned to receive either ninerafaxstat 200mg modified release tablets or matching placebo tablets, both taken twice daily. The study treatment will be given over a 12-week period. This design helps researchers observe how ninerafaxstat performs against placebo while maintaining standard therapies for nHCM. During the study, participants will undergo assessments including cardiopulmonary exercise testing and completion of the Kansas City Cardiomyopathy Questionnaire to track symptoms, physical limitations, and overall health status. The main focus is on changes in the questionnaires clinical summary score from baseline to week 12. Safety and tolerability will also be monitored throughout the trial, which is expected to run until December 2026.

A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy