Safety, pharmacokinetics, and exploratory efficacy of the oral ghrelin receptor agonist AC01 in heart failure with reduced ejection fraction (GOAL-HF1): a randomised, double-blind, placebo-controlled, phase 1b/2a study.

What Was Tested

This randomized controlled trial looks at Ghrelin.

In this exploratory study, the researchers aimed to assess the safety and tolerability of AC01, a novel oral calcium-sensitising inotrope and ghrelin receptor agonist, in patients with HFrEF.

Who Took Part

By PubMed's age tags, the people studied were teenagers, young adults, adults, middle-aged adults, older adults and people over 80, both men and women.

Other eligibility criteria included sinus rhythm or permanent, persistent, or paroxysmal atrial fibrillation or flutter (only allowed in phase 2a), with a mean resting heart rate of 55-90 beats per min.

Safety was monitored by physical examination, vital signs, safety laboratory assessments, and 12-lead electrocardiograms (ECGs) periodically during the treatment period and until the end-of-study visit (day 12 in phase 1b and day 42 in phase 2a), and cardiac rhythm was continuously monitored remotely using a patch device until day 9 in phase 1b and until day 4 in phase 2a.

How the Study Was Run

The design is described as randomized, double-blind, placebo-controlled and parallel-group.

The participants were allocated by chance, so the Ghrelin group and the comparison group should start out alike.

Neither the participants nor the staff measuring them knew who was on Ghrelin, so expectations could not easily colour the readings.

A placebo (an inactive look-alike) gave the Ghrelin group a baseline to be compared against.

The Ghrelin group and the comparison group ran side by side rather than switching.

Randomisation used permuted blocks, with block sizes of four for phase 1b and three for phase 2a.

The primary outcome was safety and tolerability.

Adverse or unexpected events, signs, or symptoms were recorded.

This study is registered with ClinicalTrials.gov, NCT05642507, and has been completed.

What Was Measured

Topics PubMed links to this paper include Heart Failure, Stroke Volume, Receptors, Ghrelin, Cardiotonic Agents, Indoles and Tryptophan.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Ghrelin.

Results are reported in around 12 sentences of the abstract; we leave them to the paper itself rather than summarise them.

The publication is from 2026 (Lancet (London, England)), a journal based in England.

More context: the Ghrelin compound page, all human trials.