ACACIA-HCM
A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
Paciente / Población Intervención / Exposición Comparación Desenlace
In 516 patients with symptomatic non-obstructive hypertrophic cardiomyopathy, aficamten was compared with placebo on KCCQ clinical summary score and peak oxygen uptake at 36 weeks.
N
516
Diseño
Phase 3 double-blind, placebo-controlled RCT, 1:1, echocardiography-guided dose titration at weeks 2, 4 and 6 (escalation only if LVEF ≥60%), primary analysis at week 36, treatment continued to 72 weeks
Desenlace
Dual primary — change from baseline to week 36 in KCCQ Clinical Summary Score and in peak oxygen uptake (pVO2)