Heart Failure With Reduced Ejection Fraction (HFrEF)Phase 2GOAL-HF2NCT07584967
A Study to Evaluate the Safety and Efficacy of AC01 Compared to Placebo in Participants With Chronic Heart Failure
A Phase 2 study testing AC01 in people with Heart Failure With Reduced Ejection Fraction (HFrEF), run by AnaCardio AB.
Trial summary
The primary purpose of the study is to evaluate the safety and efficacy of 2 doses of AC01 compared to placebo over 12 weeks in participants with chronic advanced HFrEF. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Heart Failure With Reduced Ejection Fraction (HFrEF)
- What is being tested?
- AC01
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 400 participants
- Where
- No sites listed yet
- Study dates
- 15 Nov 2026to 30 Aug 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- History of Chronic Heart Failure (CHF) diagnosed greater than or equal to (>=6) months before screening, and in NYHA Class II to IV at screening.
- Chronic advanced HFrEF defined as:
- LVEF less than or equal to (<=) 35 percentage (%) by local reading >=6 months before screening or a record of LVEF qualitatively described as severely reduced or moderately-severely reduced >=6 months before screening, and
- LVEF <=35% at the screening echocardiography (confirmed by core lab), and
- no known LVEF greater than (>) 35% (or qualitatively described as less than moderately-severely reduced) between the 2 readings.
- Sinus rhythm or permanent, persistent, or paroxysmal atrial fibrillation flutter (AFF) (AFF at screening is capped at maximum 15% of participants enrolled) with mean resting heart rate of >=55 and <=90 beats per minute (bpm) at screening, and >=50 and <=95 bpm at randomization, regardless of rhythm.
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- NT-proBNP >=400 picograms per milliliter (pg/mL) in sinus rhythm and >=800 pg/mL in AFF at screening (confirmed by central laboratory).
- Transvenous implantable cardioverter-defibrillator (ICD) for primary prevention with back-up pacing set at 40 bpm.
- Treated with optimal, stable, medical therapy for HF consistent with prevailing local and international guidelines unless contraindicated or not tolerated, as judged and documented by the investigator.
Who cannot join
- Any acute or serious co-morbid condition that could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.
- Admitted to hospital with the primary reason of HF within 24 hours before randomization or currently hospitalized with the primary reason of HF for more than 10 days. Current hospitalization (>24 hours and <=10 days) is capped at a maximum of 15% of participants enrolled.
- Acute coronary syndrome, stroke or transient ischemic attack, severe ventricular arrhythmia, or major cardiac intervention, percutaneous coronary intervention, valvuloplasty/other cardiac valve repair or implantation, or cardiac surgery within 60 days before randomization.
- Systolic blood pressure >130 or ˂90 millimeters of mercury (mmHg) at screening, or >140 or ˂85 mmHg at randomization.
- Uncontrolled diabetes mellitus, defined as Hemoglobin A1c (HbA1c) >=9.0% (>=75 millimoles per mole [mmol/mol]) at screening, or severe complications of diabetes.
- Body weight <50 kilogram (kg) or body mass index (BMI) <18 kilograms per square meter (kg/m^2) or >45 kg/m^2 at screening.
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- Any of the following electrocardiogram (ECG) findings at screening: Corrected QT interval using Fridericia's formula (QTcF) >450 milliseconds (ms), 1st degree atrioventricular (AV) block with PQ >240 ms, or AV block 2nd or 3rd degree.
- History of aborted cardiac arrest, sustained ventricular tachycardia, or Torsades-de Pointes, or congenital long QT syndrome. Family history of sudden cardiac death, unexplained death, long QT syndrome, or death from a primary dysrhythmia.
- Cardiac resynchronization therapy, Cardiac Contractility Modulation, or pacemaker device other than the back-up pacing function of the ICD.
- Mechanical hemodynamic support, kidney support, or ventilation within 7 days before randomization.
- Treatment with i.v. inotropes, i.v. vasodilators, or i.v. vasopressors within 3 days before randomization.
- Treatment with any i.v. diuretics or supplemental oxygen within 6 hours before randomization.
- Use of any drugs or substances known to be strong inducers of Cytochrome P450 3A4 (CYP3A4) enzyme within 28 days before randomization or planned to be used during the study period or strong inhibitors of CYP3A4 within 7 days before randomization or planned to be used during the study period.
- Use of any drug that is known to prolong the QT interval (for example, sotalol, dofetilide, macrolides, some antidepressants, and some antipsychotics) within 28 days before randomization or planned to be used during the study period.
- Treatment changes in glucose lowering therapy within 28 days before randomization.
- Estimated glomerular filtration rate (eGFR) <20 milliliters per minute per 1.73 square meters (mL/min/1.73 m2) according to the Chronic Kidney Disease Epidemiology Collaboration formula, planned renal replacement therapy within the next 6 months, or receiving dialysis at screening.
- Serum potassium <3.5 or >5.2 milliequivalents per liter (mEq/L) at screening.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 *upper limit of normal (ULN) or total bilirubin >2 * ULN, or known cirrhosis, severe liver, or pancreatic disease at screening.
- Use of any anti-arrhythmic drugs within 28 days before randomization or planned to be used during the study period, except for amiodarone, ivabradine, digoxin, and beta blockers.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Evaluate the Safety and Efficacy of 2 Dose Levels of the Oral Ghrelin Receptor Agonist AC01 Over 12 Weeks in Patients With Chronic Advanced Heart Failure With Reduced Ejection Fraction (HFrEF)
The primary purpose of the study is to evaluate the safety and efficacy of 2 doses of AC01 compared to placebo over 12 weeks in participants with chronic advanced HFrEF.
Read the detailed description
This is a randomized, double-blind, placebo-controlled, parallel-group, multicenter study designed to evaluate the safety and efficacy of the ghrelin-receptor agonist AC01 compared to placebo in participants with chronic advanced HFrEF. Approximately 400 participants will be randomized to 1 of 3 treatment arms: 3 milligram (mg) AC01, 1 milligram (mg) AC01, or placebo twice daily for 12 weeks. The primary objective is to evaluate the effect of AC01 compared to placebo on cardiac structure and function assessed by echocardiography.
- Being tested
- AC01, Placebo
- Keywords
- Heart Diseases, Cardiovascular Diseases, Heart Failure
- Medical subject headings
- Heart Diseases, Cardiovascular Diseases, Heart Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AC01Drug
AC01 tablets for oral administration.
PlaceboOther
AC01 matching placebo tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| AC01 3 mg | Experimental | Participants will receive AC01 orally twice daily (BID) for 12 weeks. Receives: Drug: AC01 |
| AC01 1 mg | Experimental | Participants will receive AC01 orally BID for 12 weeks. Receives: Drug: AC01 |
| Placebo | Placebo comparator | Participants will receive matching placebo orally BID for 12 weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12
The composite echocardiography 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).
Measured: Baseline and Week 12
Secondary outcomes (21)
Absolute Change from Baseline in LVSV at Week 12
Measured: Baseline and Week 12
Absolute Change from Baseline in LVEF at Week 12
Measured: Baseline and Week 12
Absolute Change from Baseline in LVESV at Week 12
Measured: Baseline and Week 12
Absolute Change From Baseline in LAVImin at Week 12
Measured: Baseline and Week 12
Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Measured: Baseline and Week 12
Absolute Change From Baseline in Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS) at Week 12
The KCCQ is a validated, 23-item, self-administered questionnaire designed to assess health status in participants with congestive heart failure. It evaluates six domains: symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability. Each domain score is transformed to a scale of 0 to 100, with higher scores indicating better health status. The KCCQ-TSS is derived from the domain scores.
Measured: Baseline and Week 12
Absolute Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 12
PGIS is 1-item questionnaire used to rate the severity of a specific condition. Participants record their perceived severity of heart failure (HF) symptoms, specifically shortness of breath, fatigue, and swelling, and choose 1 response that best described the extent of their symptoms based on a 5-point scale. Response options include none, mild, moderate, severe, and very severe. Higher PGIS scores indicate more severe HF symptoms; lower scores indicate less severe symptoms.
Measured: Baseline and Week 12
Patient Global Impression of Change (PGIC) at Week 12
The PGIC questionnaire is administered to assess the participant's impression of change in HF symptoms since the initiation of study treatment, specifically changes in shortness of breath, fatigue, and swelling. Participants select one response to describe the overall change (if any) in HF symptoms on a 7-category scale: 7=very much improved, 6 =much improved, 5 =minimally improved, 4 =no change, 3 =minimally worse, 2 =much worse, and 1 =very much worse. Higher PGIC scores indicate greater improvement in HF symptoms, while lower scores indicate worsening or no change.
Measured: At Week 12
Number of Participants With Death, Cardiovascular Death, Hospitalization, Cardiovascular Hospitalization, Heart Failure Hospitalization, Heart Failure Events, Other Adjudicated Events, and Listed for Heart Transplantation
Measured: At Week 12
Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Measured: Baseline and Week 12
Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Measured: Baseline and Week 12
Number of Participants With Dialysis
Measured: At Week 12
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
The CNAQ is an 8-item, self-administered questionnaire used to assess appetite over time. Each item is scored on a scale of 1 to 5, and the total score is calculated as the sum of all item scores. The instrument has demonstrated acceptable psychometric properties, including internal consistency, construct validity, and predictive validity, for assessing appetite in participants with heart failure. Higher CNAQ scores indicate better appetite.
Measured: Baseline and Week 12
Absolute Change From Baseline in New York Heart Association (NYHA) Class
Measured: Baseline and Week 12
AUC,ss: Area Under the Concentration-Time Curve at Steady State of AC01 and its Metabolite M6
Population pharmacokinetic parameters
Measured: At Week 4 and Week 12
Cmax,ss: Maximum Observed Concentration at Steady State of AC01 and its Metabolite M6
Population pharmacokinetic parameters
Measured: At Week 4 and Week 12
Tmax,ss: Time to Reach Maximum Concentration at Steady State
Population pharmacokinetic parameters
Measured: At Week 4 and Week 12
Ctrough: Trough concentration at steady state of AC01 and its Metabolite M6
Population pharmacokinetic parameters
Measured: At Week 4 and Week 12
Rac (Cmax): Accumulation Ratio of Cmax for AC01 and its Metabolite M6
Population pharmacokinetic parameters. Accumulation ratio of Cmax calculated as Cmax at Week 12/Cmax at Week 4.
Measured: At Week 4 and Week 12
Rac (AUC): Accumulation Ratio of AUC for AC01 and its Metabolite M6
Population pharmacokinetic parameters. Accumulation ratio of AUC calculated as AUC at Week 12/AUC at Week 4.
Measured: At Week 4 and Week 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)
Measured: From first dose of study drug up to end of follow up (up to Week 16)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study start15 Nov 2026 (estimated)
- Primary completion30 Jul 2028 (estimated)
- Study completion30 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heart Failure With Reduced Ejection Fraction (HFrEF)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AnaCardio AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07584967
- Sponsor's protocol ID
- AC01-02
- Other IDs
- 2025-524469-25-00, PIP number, 2025
- Acronym
- GOAL-HF2
- Status last verified
- Sep 2026
- First submitted
- 4 May 2026
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual de-identified participant data will be shared with qualified scientific and medical researchers whose proposed use of data has been approved by the study executive committee, beginning 9 months and ending 36 months following article publication. Proposals should be directed to the Central Contact Person.
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Evaluate the Safety and Efficacy of 2 Dose Levels of the Oral Ghrelin Receptor Agonist AC01 Over 12 Weeks in Patients With Chronic Advanced Heart Failure With Reduced Ejection Fraction (HFrEF)
- ClinicalTrials.gov ID
- NCT07584967
- Acronym / sponsor ID
- GOAL-HF2 / AC01-02
- Keywords
- Heart Diseases, Cardiovascular Diseases, Heart Failure
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- AnaCardio ABIndustry (pharma, biotech, devices)
- Study start
- 15 Nov 2026 (estimated)
- Primary completion
- 30 Jul 2028 (estimated)
- Study completion
- 30 Aug 2028 (estimated)
- First posted
- 13 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary purpose of the study is to evaluate the safety and efficacy of 2 doses of AC01 compared to placebo over 12 weeks in participants with chronic advanced HFrEF.
Detailed description
This is a randomized, double-blind, placebo-controlled, parallel-group, multicenter study designed to evaluate the safety and efficacy of the ghrelin-receptor agonist AC01 compared to placebo in participants with chronic advanced HFrEF. Approximately 400 participants will be randomized to 1 of 3 treatment arms: 3 milligram (mg) AC01, 1 milligram (mg) AC01, or placebo twice daily for 12 weeks. The primary objective is to evaluate the effect of AC01 compared to placebo on cardiac structure and function assessed by echocardiography.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AC01 3 mg | Experimental | Drug: AC01Participants will receive AC01 orally twice daily (BID) for 12 weeks. |
| AC01 1 mg | Experimental | Drug: AC01Participants will receive AC01 orally BID for 12 weeks. |
| Placebo | Placebo comparator | Other: PlaceboParticipants will receive matching placebo orally BID for 12 weeks. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- History of Chronic Heart Failure (CHF) diagnosed greater than or equal to (>=6) months before screening, and in NYHA Class II to IV at screening.
- Chronic advanced HFrEF defined as:
- Sinus rhythm or permanent, persistent, or paroxysmal atrial fibrillation flutter (AFF) (AFF at screening is capped at maximum 15% of participants enrolled) with mean resting heart rate of >=55 and <=90 beats per minute (bpm) at screening, and >=50 and <=95 bpm at randomization, regardless of rhythm.
- NT-proBNP >=400 picograms per milliliter (pg/mL) in sinus rhythm and >=800 pg/mL in AFF at screening (confirmed by central laboratory).
- Transvenous implantable cardioverter-defibrillator (ICD) for primary prevention with back-up pacing set at 40 bpm.
Key exclusion criteria (19 listed)
- Any acute or serious co-morbid condition that could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.
- Admitted to hospital with the primary reason of HF within 24 hours before randomization or currently hospitalized with the primary reason of HF for more than 10 days. Current hospitalization (>24 hours and <=10 days) is capped at a maximum of 15% of participants enrolled.
- Acute coronary syndrome, stroke or transient ischemic attack, severe ventricular arrhythmia, or major cardiac intervention, percutaneous coronary intervention, valvuloplasty/other cardiac valve repair or implantation, or cardiac surgery within 60 days before randomization.
- Systolic blood pressure >130 or ˂90 millimeters of mercury (mmHg) at screening, or >140 or ˂85 mmHg at randomization.
- Uncontrolled diabetes mellitus, defined as Hemoglobin A1c (HbA1c) >=9.0% (>=75 millimoles per mole [mmol/mol]) at screening, or severe complications of diabetes.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AC01AC01 tablets for oral administration. | Drug | AC01 1 mg, AC01 3 mg |
| PlaceboAC01 matching placebo tablets for oral administration. | Other | Placebo |
Primary outcome
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12
Time frame: Baseline and Week 12
The composite echocardiography 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).
21 secondary outcomes
Absolute Change from Baseline in LVSV at Week 12
Time frame: Baseline and Week 12
Absolute Change from Baseline in LVEF at Week 12
Time frame: Baseline and Week 12
Absolute Change from Baseline in LVESV at Week 12
Time frame: Baseline and Week 12
Absolute Change From Baseline in LAVImin at Week 12
Time frame: Baseline and Week 12
Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Time frame: Baseline and Week 12
Absolute Change From Baseline in Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS) at Week 12
Time frame: Baseline and Week 12
Absolute Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 12
Time frame: Baseline and Week 12
Patient Global Impression of Change (PGIC) at Week 12
Time frame: At Week 12
Number of Participants With Death, Cardiovascular Death, Hospitalization, Cardiovascular Hospitalization, Heart Failure Hospitalization, Heart Failure Events, Other Adjudicated Events, and Listed for Heart Transplantation
Time frame: At Week 12
Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Time frame: Baseline and Week 12
Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline and Week 12
Number of Participants With Dialysis
Time frame: At Week 12
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
Time frame: Baseline and Week 12
Absolute Change From Baseline in New York Heart Association (NYHA) Class
Time frame: Baseline and Week 12
AUC,ss: Area Under the Concentration-Time Curve at Steady State of AC01 and its Metabolite M6
Time frame: At Week 4 and Week 12
Cmax,ss: Maximum Observed Concentration at Steady State of AC01 and its Metabolite M6
Time frame: At Week 4 and Week 12
Tmax,ss: Time to Reach Maximum Concentration at Steady State
Time frame: At Week 4 and Week 12
Ctrough: Trough concentration at steady state of AC01 and its Metabolite M6
Time frame: At Week 4 and Week 12
Rac (Cmax): Accumulation Ratio of Cmax for AC01 and its Metabolite M6
Time frame: At Week 4 and Week 12
Rac (AUC): Accumulation Ratio of AUC for AC01 and its Metabolite M6
Time frame: At Week 4 and Week 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)
Time frame: From first dose of study drug up to end of follow up (up to Week 16)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
- Individual participant dataSharing planned: Yes. Individual de-identified participant data will be shared with qualified scientific and medical researchers whose proposed use of data has been approved by the study executive committee, beginning 9 months and ending 36 months following article publication. Proposals should be directed to the Central Contact Person.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AC01 in people with Heart Failure With Reduced Ejection Fraction (HFrEF), run by AnaCardio AB.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Robert Edfors, MD, PhD (+46 760 542 609, studyinfo@anacardio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12 (time frame: Baseline and Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.