Study of Aficamten to Treat Symptomatic Obstructive Hypertrophic Cardiomyopathy in Children and Adolescents Aged 12 to Under 18 Years

What this Trial Is About

Researchers are studying aficamten in children and adolescents aged 6 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy oHCM, a condition causing thickened heart muscle and outflow obstruction. The study aims to evaluate the drugs effectiveness, safety, and how the body processes it in this pediatric group. Adolescents and children will be studied sequentially, starting with adolescents to establish safe and effective dosing before proceeding to younger children. The trial is a Phase 23 randomized, double-blind, placebo-controlled study with additional open-label extension periods for long-term assessment. Participants will be assigned randomly to receive either oral aficamten or a placebo once daily. The initial treatment period lasts 12 weeks and is double-blinded. After this, all participants receive aficamten for 52 weeks during an open-label extension, followed by a long-term extension of 144 weeks to further assess safety, tolerability, and efficacy. Doses range from 5 mg to 20 mg and are adjusted based on echocardiography findings. This staged approach ensures careful monitoring and dosing adjustments throughout the study. During the study, participants will undergo regular evaluations including heart ultrasounds to measure heart function and obstruction, blood tests for heart biomarkers, and assessments of heart failure symptoms using the New York Heart Association functional class. Researchers will monitor how aficamten affects the hearts outflow tract gradient over 12 weeks and track long-term safety over several years. The study includes multiple visits for clinical assessments, laboratory tests, and drug level measurements to understand treatment effects and ensure participant safety.

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).