Heart failure & cardiomyopathies
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
Authors' conclusion
In patients with HFrEF, AC01 over 28 days appeared safe and well tolerated, and no major harms were identified in this early-phase study.
Study context
- Population
- Adults aged 18–80 years with HF ≥6 months, EF ≤40% and a transvenous ICD for primary prevention, Netherlands, UK, Sweden and Italy32 phase 1b, 26 phase 2a
- Participants
- 58 adults
- Comparison
- AC01 vs placebo
- Primary endpoint
- Safety and tolerability
Methodological detailsphase 1b/2a, double-blind, placebo-controlled, 14 sites
- Phase 1b/2a
- Double-blind
- Placebo-controlled
- 14 sites
Primary result
As reported in the abstract
Mild or moderate treatment-emergent adverse events
- AC01-related adverse events, phase 1b
- 12
- AC01-related adverse events, phase 2a
- 18
- AC01-related serious adverse events
- 0
- Deaths during the study
- 0
Safety
Serious adverse events and other safety findings
- Treatment-related serious adverse event (increased hs-troponin I) in a placebo patient, phase 1b
- 1
- Symptomatic hypotension
- 0
- Most common treatment-emergent AEs: hypotension, non-sustained VT, dyspnoea, hyperglycaemia, dizziness or vertigo, headache
- ECG: no apparent signs of tachycardia, new-onset tachyarrhythmias, myocardial ischaemia, or morphological or conduction abnormalities
- No apparent effects of AC01 on hs-troponin I or NT-proBNP
Original publication
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. Lancet. Published 24 June 2026. doi:10.
Summarised from the published abstract; every number is checked against it