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Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study
Juan Pablo Kaski1, Paul F Kantor2, Stephanie J Nakano3
1Centre for Paediatric Inherited and Rare Cardiovascular Disease, UCL Institute of Cardiovascular Science, University College London, United Kingdom (J.P.K.).
Insights
The CEDAR-HCM trial is evaluating aficamten, a cardiac myosin inhibitor, for treating obstructive hypertrophic cardiomyopathy (HCM) in children and adolescents. This study aims to determine the drug's safety and efficacy in younger populations.
Area of Science:
- Cardiology
- Pediatric Medicine
- Pharmacology
Background:
- Hypertrophic cardiomyopathy (HCM) is a significant cause of illness and death in children, with limited treatment options.
- Aficamten is a novel cardiac myosin inhibitor targeting the hypercontractility characteristic of HCM.
- Previous studies in adults showed aficamten improved exercise capacity and symptoms in obstructive HCM.
Purpose of the Study:
- To evaluate the efficacy, safety, and pharmacokinetics of aficamten in pediatric patients with symptomatic obstructive HCM.
- To assess aficamten's effect on left ventricular outflow tract (LVOT) gradient and other clinical parameters in children and adolescents.
- To provide crucial data on aficamten use in a pediatric population, including children as young as 6 years.
Main Methods:
- The CEDAR-HCM trial is an international, randomized, double-blind, placebo-controlled study with an open-label extension.
- Enrollment includes adolescents (12-18 years) and children (6-12 years) with symptomatic obstructive HCM and specific echocardiographic criteria.
- Participants receive aficamten or placebo, with echocardiogram-guided dose adjustments to achieve target LVOT gradient and ejection fraction.
Main Results:
- The trial is currently enrolling participants.
- Primary endpoint is the change in Valsalva LVOT gradient at 12 weeks.
- Secondary endpoints include resting LVOT gradient, cardiac biomarkers, and functional class.
Conclusions:
- Results from CEDAR-HCM will offer insights into the safety and efficacy of aficamten in pediatric patients.
- The study will help establish aficamten as a potential treatment for obstructive HCM in children and adolescents.
- This research addresses a critical unmet need for effective therapies in pediatric HCM.
Background:
Hypertrophic cardiomyopathy (HCM) is an important cause of morbidity and mortality in children, but treatment options are limited. Aficamten, a next-in-class cardiac myosin inhibitor, directly targets the hypercontractility underlying HCM. Aficamten improved exercise capacity, health status, and symptoms in adults with obstructive HCM in the pivotal, phase 3 SEQUOIA-HCM trial (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM; NCT05186818).
Methods:
CEDAR-HCM (Clinical Evaluation of dosing With Aficamten to Reduce Obstruction in Pediatric Population With HCM) is an international, multicenter, randomized, double-blind, placebo-controlled trial followed by an open-label extension to evaluate the efficacy, safety, and pharmacokinetics of aficamten in pediatric participants with symptomatic obstructive HCM. The trial will enroll ≈55 adolescents (12 to <18 years) and subsequently expand to include at least 10 children (6 to <12 years) with nonsyndromic obstructive HCM, left ventricular ejection fraction ≥60%, Valsalva left ventricular outflow tract gradient ≥50 mm Hg, and New York Heart Association functional class ≥II. Participants will be randomized 2:1 to aficamten or placebo in addition to standard of care therapy or as monotherapy, with echocardiogram-guided dose adjustments targeting a Valsalva left ventricular outflow tract gradient <30 mm Hg while maintaining left ventricular ejection fraction ≥50%. The primary end point is the change in Valsalva left ventricular outflow tract gradient from baseline to week 12. Secondary end points include change in resting left ventricular outflow tract gradient, cardiac biomarkers, New York Heart Association functional class, and assessment of pharmacokinetics. After completing the 12-week randomized period, eligible participants will continue into a long-term open-label extension.
Results:
The trial is currently enrolling.
Conclusions:
Results of CEDAR-HCM will provide insight into the safety and efficacy of aficamten in adolescents and in children as young as 6 years of age.
Registration:
URL: https://www.clinicaltrials.gov; Unique identifier: NCT06412666.
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