In short
AC01 is an investigational oral medicine being studied for chronic advanced heart failure with reduced ejection fraction (HFrEF). One Phase 2 trial, NCT07584967, is currently authorised and plans to enrol 400 participants. The trial compares AC01 against placebo to see whether it improves heart structure and function on echocardiography.
Key points
- AC01 is an investigational oral substance studied in chronic advanced heart failure with reduced ejection fraction (HFrEF).
- The single registered trial is a Phase 2 study comparing AC01 with placebo.
- The trial is currently authorised and plans to enrol 400 participants.
- The main measure is the change in a composite echocardiography Z-score from baseline to Week 12.
- The composite Z-score includes left ventricular stroke volume, end systolic volume, ejection fraction, and left atrial minimal volume index.
What is AC01?
AC01 is an investigational oral medicine. Its full chemical name is 1-[(1S)-1-(2,3-dichloro-4-methoxyphenyl)ethyl]-3-methyl-3-[(4R)-1-methyl-3,3-dimethyl-4-piperidyl]urea. In the trial registry it is recorded as a drug given by oral use. AC01 is not yet an authorised medicine; it is being studied in a clinical trial to find out whether it is safe and effective.
How does AC01 work?
The registry record for this trial does not describe the mechanism of action of AC01. The study is designed to evaluate its effect on cardiac structure and function assessed by echocardiography.
Which conditions is AC01 studied for?
- Chronic advanced heart failure with reduced ejection fraction (HFrEF) — a condition in which the heart muscle does not pump blood as well as it should, and the fraction of blood ejected from the left ventricle with each beat is reduced.
How is AC01 given?
AC01 is given by oral use, in milligram amounts. The trial record lists two AC01 arms with oral use and a placebo arm with tablets of the same qualitative composition but no active drug substance. The exact dose in milligrams was not reported in the register.
What do the clinical trials show?
One Phase 2 trial, NCT07584967, is authorised to evaluate the safety and efficacy of AC01 compared with placebo in patients with chronic advanced heart failure with reduced ejection fraction (HFrEF). The trial plans to enrol 400 participants.
The primary outcome is the absolute change from baseline in a composite echocardiography Z-score at Week 12. The composite Z-score integrates left ventricular stroke volume (LVSV), left ventricular end systolic volume (LVESV), left ventricular ejection fraction (LVEF), and left atrial minimal volume index (LAVImin). These measures describe how much blood the left ventricle pumps out with each beat, how much blood remains in the ventricle at the end of a contraction, the percentage of blood ejected from the ventricle, and the smallest volume of the left atrium.
What side effects are known?
No information on side effects is available in the registry record for this trial.