NCT04219826

Dose-finding Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CK-3773274 in Adults With Hypertrophic Cardiomyopathy

A Multi-Center, Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 96 (actual)
• Sponsor: Cytokinetics · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

This study is being performed to understand the effect of different doses of CK-3773274 on patients with hypertrophic cardiomyopathy (HCM).

Detailed description

This was a Phase 2, multi-center, randomized, placebo-controlled, double-blind, dose-finding study in participants with symptomatic HCM. The study consisted of 4 cohorts. For Cohorts 1 and 2, participants with obstructive HCM (oHCM) and not receiving disopyramide were randomized 2:1 to active or placebo treatment and received up to 3 escalating doses of aficamten (5, 10, and 15 mg once daily in Cohort 1 and 10, 20, and 30 mg once daily in Cohort 2) or placebo based on site-read echocardiographic guidance. Cohort 3 consisted of participants with oHCM whose background HCM therapy included disopyramide. All participants in Cohort 3 received up to 3 escalating doses of aficamten (5, 10, and 15 mg once daily) based on echocardiographic guidance. Cohort 4 consisted of participants with non-obstructive HCM (nHCM) on standard of care background therapy. Cohort 4 participants received up to 3 doses of aficamten (5, 10, and 15 mg once daily), titrated based on site-read echocardiographic guidance. In all 4 cohorts, treatment duration was 10 weeks with a 4-week follow-up period after the last dose.

Conditions

• Hypertrophic Cardiomyopathy (HCM)

Interventions

Type	Name	Description
DRUG	CK-3773274 (5 - 15 mg)	CK-3773274 tablets administered orally once daily
DRUG	CK-3773274 (10 - 30 mg)	CK-3773274 tablets administered orally once daily
DRUG	Placebo for CK-3773274	Placebo administered orally once daily

Timeline

• Start date: 2020-01-10
• Primary completion: 2023-02-28
• Completion: 2023-02-28
• First posted: 2020-01-07
• Last updated: 2026-02-24
• Results posted: 2026-02-24

Locations

22 sites across 4 countries: United States, Italy, Netherlands, Spain

Regulatory

• FDA-regulated drug study