NCT05186818

Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM

A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 282 (actual)
• Sponsor: Cytokinetics · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) versus placebo in adults with symptomatic hypertrophic cardiomyopathy (HCM) and left ventricular outflow tract obstruction.

Detailed description

CY 6031 was a Phase 3, randomized, placebo-controlled, double-blind, multi-center trial in participants with symptomatic oHCM. Eligible participants were randomized in a 1:1 ratio to receive aficamten or placebo. Randomization was stratified by use of beta-blockers (yes or no) and cardiopulmonary exercise testing (CPET) exercise modality (treadmill or bicycle). Enrollment limits were applied as follows: participants taking beta-blockers were capped at approximately 70% of total enrollment; participants taking disopyramide were capped at approximately 10% of total enrollment; participants with persistent atrial fibrillation (AF) at screening were capped at approximately 15% of total enrollment; and participants using the bicycle CPET exercise modality were capped at approximately 50% of total enrollment. Investigational product (IP) was administered orally once daily (QD) with or without food for 24 weeks. During the initial 6 weeks of the treatment period, IP doses were individually titrated at Weeks 2, 4, and 6 based on echocardiography-guided criteria. Dose escalation at Weeks 2, 4, and 6 occurred only if a participant had a Valsalva left ventricular outflow tract gradient (LVOT-G) ≥ 30 mmHg and a biplane left ventricular ejection fraction (LVEF) ≥ 55%. Echocardiograms were performed at each subsequent visit during the trial, and the IP dose was down-titrated if the LVEF was \< 50%. The primary endpoint of peak oxygen uptake (pVO2) was measured by CPET at screening and at the end of treatment (Week 24). A participant's background HCM therapy was individually optimized according to local practice prior to enrollment in the study.

Conditions

• Obstructive Hypertrophic Cardiomyopathy (oHCM)

Interventions

Type	Name	Description
DRUG	Aficamten (5 mg, 10 mg, 15 mg, and 20 mg)	Aficamten tablets were administered orally once daily.
DRUG	Placebo to match aficamten	Placebo tablets were administered orally once daily.

Timeline

• Start date: 2022-02-01
• Primary completion: 2023-11-10
• Completion: 2023-12-18
• First posted: 2022-01-11
• Last updated: 2026-03-03
• Results posted: 2026-03-03

Locations

114 sites across 14 countries: United States, China, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Netherlands, Poland, Portugal, Spain, United Kingdom

Regulatory

• FDA-regulated drug study