Heart failure & cardiomyopathies
Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM
Authors' conclusion
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.
Study context
- Population
- Adolescents (12 to <18 years) and children (6 to <12 years) with nonsyndromic symptomatic obstructive hypertrophic cardiomyopathy (HCM), LVEF ≥60%, Valsalva LV outflow tract gradient ≥50 mm Hg, NYHA class ≥IIplanned ≈55 adolescents + ≥10 children
- Comparison
- Aficamten vs placebo
- Primary endpoint
- Change in Valsalva LV outflow tract gradient from baseline
- Follow-up
- Week 12
Methodological detailsdesign and rationale, international, multicentre, double-blind, placebo-controlled, 2:1 randomisation, open-label extension
- Design and rationale
- International
- Multicentre
- Double-blind
- Placebo-controlled
- 2:1 randomisation
- Open-label extension
Primary result
As reported in the abstract
- Trial currently enrolling; no results
Original publication
Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM. Circ Heart Fail. Published 5 December 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →