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A study of the safety and efficacy of AC01 in patients with heart failure with reduced ejection fraction

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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study involves patients with chronic advanced heart failure with reduced ejection fraction (HFrEF), a condition where the heart’s pumping ability is weakened and the amount of blood expelled with each beat is lower than normal. The investigational medicine is identified as AC01, an oral ghrelin receptor agonist that is being tested at two different dose levels. Participants will receive either the study drug or placebo tablets that look the same but contain no active ingredient.

The aim of the trial is to evaluate the safety and efficacy of the study drug compared with placebo on heart structure and function. Participants will be randomly assigned to one of the treatment groups, will take the assigned tablets once daily for 12 weeks, and will attend scheduled visits where a heart ultrasound called echocardiography will be performed to measure several aspects of heart performance, including the amount of blood pumped out of the main chamber each beat (left ventricular stroke volume), the volume remaining in that chamber after pumping (left ventricular end systolic volume), the percentage of blood expelled with each beat (left ventricular ejection fraction), and the size of the upper chamber when it is at its smallest (left atrial minimal volume index). Safety checks and routine lab tests will be done throughout the 12‑week period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline assessments

    After joining the study, you will undergo initial tests that include a physical exam, blood tests, and an echocardiography scan to measure how your heart is working. these results will be used as the starting point for later comparisons.

  2. Step 2

    Randomization and receipt of study medication

    Based on a random assignment, you will receive either ac01 tablets (the active drug) or placebo tablets (inactive). both types of tablets look the same and are taken by mouth.

    The prescribed dose is a tablet containing 000 mg of the study substance, taken once each day. the exact amount you receive will be written on the medication label.

  3. Step 3

    Daily medication intake

    You will swallow one tablet each morning with water. continue this routine every day for a total of 12 weeks.

    If you miss a dose, take it as soon as you remember unless it is almost time for the next scheduled dose; then skip the missed dose and continue with the regular schedule.

  4. Step 4

    Regular clinic visits for safety monitoring

    You will visit the clinic approximately every four weeks (at week 4 and week 8) for safety checks. during these visits, staff will review any side effects you may have experienced, repeat basic blood tests, and confirm that you are taking the medication as instructed.

  5. Step 5

    Final assessment at week 12

    At the end of the 12‑week period, you will return for a final set of tests. this includes another echocardiography scan to evaluate changes in heart function, as well as blood tests and a review of any adverse events.

    The results will be compared with the baseline measurements to determine the effect of the study medication.

  6. Step 6

    Study completion procedures

    After the final visit, you will stop taking the study tablets. the study team may arrange a short follow‑up call or visit to ensure you are feeling well after stopping the medication.

Who can join the trial?

10 criteria

  • Informed consent signed and dated before any study procedures, meaning you agree in writing after learning what the study involves.
  • Male participants must either avoid heterosexual intercourse or use two effective birth‑control methods (one must be a barrier method such as a condom) during the study and for at least 90 days after the last dose, unless their female partner cannot become pregnant.
  • Age must be between 18 and 85 years at the time you sign the consent.
  • You must have a history of chronic heart failure diagnosed at least 6 months before screening and be in NYHA Class II‑IV, a scale doctors use to describe how severe your heart‑failure symptoms are.
  • You must have chronic advanced heart failure with reduced ejection fraction, defined as a left ventricular ejection fraction (LVEF) of 35 % or less on a heart imaging test done at least 6 months before screening and again at the screening visit, with no record of LVEF higher than 35 % between those tests. LVEF measures how well the heart pumps blood.
  • Your heart rhythm must be either normal (sinus rhythm) or a type of irregular rhythm called atrial fibrillation (AF), and your resting heart rate must be 55‑90 beats per minute at screening and 50‑95 beats per minute at randomization. Heart rate is the number of heartbeats each minute.
  • Blood test for NT‑proBNP must be at least 400 pg/mL if you have sinus rhythm, or at least 800 pg/mL if you have atrial fibrillation. This test shows how much stress is on your heart.
  • You must have an implanted ICD (implantable cardioverter‑defibrillator) for primary prevention that is active and set to deliver a backup pacing signal at 40 beats per minute if needed. An ICD is a device that can correct dangerous heart rhythms.
  • You must be receiving stable, optimal heart‑failure medicines according to current guidelines, unless a specific drug cannot be used because of side effects or other reasons. This means your medication regimen should be steady and appropriate for heart failure.
  • Female participants who could become pregnant must use a highly effective method of contraception starting at least 14 days before randomization, continue throughout the study, and for at least 90 days after the last dose.

Who cannot join the trial?

33 criteria

  • Known hypersensitivity or intolerance to AC01, any of its ingredients, or the placebo.
  • History of receiving a solid organ transplant (such as kidney, liver, or heart) or planning to receive one during the study.
  • Any medical or surgical condition that could change how the body absorbs, distributes, metabolizes, or eliminates AC01.
  • Specific heart‑electrical test (ECG) results: QTcF longer than 450 ms, first‑degree AV block with PQ interval over 240 ms, or second/third‑degree AV block.
  • Past episodes of stopped cardiac arrest, sustained fast heart rhythm lasting more than 30 seconds, Torsades de Pointes (a dangerous rhythm) unless it was caused by a reversible factor, congenital long QT syndrome, or a family history of sudden unexplained death or long QT syndrome in a parent, sibling, or child.
  • Any heart rhythm other than atrial fibrillation that would make ECG or heart‑ultrasound interpretation difficult, such as extra heartbeats (premature ventricular contractions) making up more than 20 % of beats, or being paced by a device.
  • Ventricular tachycardia that required anti‑arrhythmic medication, an implanted cardioverter‑defibrillator (ICD) shock, or anti‑tachycardia pacing within the past 12 months.
  • Use of cardiac devices such as cardiac resynchronization therapy, cardiac contractility modulation, or a pacemaker (other than backup pacing of an ICD).
  • Use of mechanical heart‑support devices, kidney‑support machines, or a ventilator within 7 days before randomization.
  • Treatment with intravenous (IV) inotropes, vasodilators, or vasopressors within 3 days before randomization.
  • Treatment with IV diuretics or supplemental oxygen within 6 hours before randomization.
  • Any serious additional illness that could shorten life to less than 12 months (non‑heart cause), an unplanned hospital stay within 28 days for reasons other than heart failure, major infection, or severe liver, pancreas, kidney, blood, metabolic or endocrine disease that might cause early study stop or affect results.
  • Use of systemic steroids for more than 7 days within the 28 days before randomization or current steroid use at randomization.
  • Use of strong inducers of the enzyme CYP3A4 (e.g., rifampin, phenytoin) within 28 days before randomization or planned during the study.
  • Use of strong inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir) within 7 days before randomization or planned during the study.
  • Use of anti‑arrhythmic drugs within 28 days before randomization or planned, except for amiodarone, ivabradine, digoxin, and beta‑blockers.
  • Use of any medication known to lengthen the QT interval (e.g., sotalol, dofetilide, certain antibiotics, some antidepressants or antipsychotics) within 28 days before randomization or planned.
  • Changes in diabetes‑lowering therapy within 28 days before randomization.
  • Current or recent participation in another interventional clinical study where an investigational product was given within 4 weeks (or 5 half‑lives) before screening.
  • Kidney function test showing estimated glomerular filtration rate (eGFR) less than 20 mL/min/1.73 m², planned dialysis in the next 6 months, or already on dialysis.
  • Blood potassium level lower than 3.5 or higher than 5.2 mEq/L at screening.
  • Liver enzymes ALT or AST more than three times the normal upper limit, or total bilirubin more than twice the normal limit, or known severe liver or pancreatic disease at screening (a mild bilirubin rise due to Gilbert’s syndrome is allowed).
  • Hospital admission for heart failure within 24 hours before randomization, or being hospitalized for heart failure longer than 10 days.
  • Consumption of foods or drinks that affect CYP450 enzymes (such as grapefruit juice, cranberry juice, pomelo, star fruit, or Seville orange) within 7 days before randomization and unwilling to avoid them during the study.
  • Women who are pregnant, breastfeeding, or planning to become pregnant during the study.
  • Participant or a first‑degree relative (parent, sibling, or child) who works for or owns part of the sponsor organization.
  • Factors that would make it difficult to attend all study visits or follow procedures, including ongoing substance abuse.
  • Active cancer requiring chemotherapy, surgery, radiation, or palliative care at screening.
  • Recent serious heart events such as heart attack (ST‑elevation or non‑ST‑elevation myocardial infarction), unstable angina, stroke, transient ischemic attack, severe ventricular arrhythmia, or major cardiac procedures (device implantation, catheter ablation, angioplasty, valve repair/replacement, or heart surgery) within 60 days before randomization, or planned during the study, as well as planned electrical or drug cardioversion or infusion therapy for heart failure.
  • Severe untreated valve disease, hypertrophic or infiltrative cardiomyopathy, active myocarditis (inflammation of the heart muscle), constrictive pericarditis, or clinically significant congenital heart disease.
  • Systolic blood pressure higher than 130 mmHg or lower than 90 mmHg at screening, or higher than 140 mmHg or lower than 85 mmHg at randomization.
  • Uncontrolled diabetes, defined as HbA1c 9.0 % or higher, or serious diabetes complications such as advanced eye disease, retinal swelling, foot problems, or recent severe high blood sugar requiring hospitalization.
  • Body weight less than 50 kg or body‑mass index (BMI) below 18 kg/m² or above 45 kg/m² at screening.
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Investigated drugs

AC01 is an oral tablet that works as a ghrelin receptor agonist. In this study, it is being tested at two different dose levels over a period of 12 weeks. The medicine is given to patients with chronic advanced heart failure and a reduced ejection fraction to see if it can improve the heart’s structure and function, as measured by echocardiography.

What is already known about the treatment

  • Placebo

    The placebo is given as an oral tablet that looks the same as the study drug but contains no active ingredient. It is used in the trial as a control to show how patients do without the active medication. Since it has no drug, it has no therapeutic effect and is not listed in medical references. Its purpose is only to help researchers compare results between treated and untreated groups.

  • AC01

    AC01 is taken as an oral tablet and is being studied as an experimental ghrelin receptor agonist for people with chronic advanced heart failure with reduced ejection fraction. In medical literature it is still considered investigational and not yet approved for general use. It works by binding to the ghrelin (growth‑hormone secretagogue) receptor, which can raise growth‑hormone levels and may improve heart muscle function. AC01 is classified as a ghrelin receptor agonist and is being tested as a potential cardiovascular therapy.

Investigated diseases

Chronic advanced heart failure with reduced ejection fraction (HFrEF) - This condition is a long‑lasting inability of the heart to pump blood effectively, and the left ventricle does not contract strongly enough, leading to a low ejection fraction. Over time the heart muscle becomes stretched and thinner, causing the chamber to enlarge. The reduced pumping ability leads to a buildup of fluid in the lungs and other parts of the body. As the disease advances, symptoms such as shortness of breath, fatigue, and swelling become more noticeable. The heart’s structure continues to change, worsening its capacity to maintain adequate circulation.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-524469-25-00Protocol codeAC01-02Estimated enrolment400 patientsSponsorAnaCardio AB

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