A Study to Evaluate the Safety and Efficacy of AC01 Compared to Placebo in Participants With Chronic Heart Failure (GOAL-HF2)

September 11, 2026 updated by: AnaCardio AB

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
400

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • 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.
  • 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.

Key Exclusion Criteria:

  • 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.
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: AC01 3 mg
Participants will receive AC01 orally twice daily (BID) for 12 weeks.
Drug: AC01
AC01 tablets for oral administration.
Experimental: AC01 1 mg
Participants will receive AC01 orally BID for 12 weeks.
Drug: AC01
AC01 tablets for oral administration.
Placebo Comparator: Placebo
Participants will receive matching placebo orally BID for 12 weeks.
Other: Placebo
AC01 matching placebo tablets for oral administration.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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).
Baseline and Week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Absolute Change from Baseline in LVSV at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change from Baseline in LVEF at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change from Baseline in LVESV at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change From Baseline in LAVImin at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Time Frame: Baseline and Week 12
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
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.
Baseline and Week 12
Absolute Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 12
Time Frame: Baseline and 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.
Baseline and Week 12
Patient Global Impression of Change (PGIC) at Week 12
Time Frame: 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.
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
At Week 12
Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline and Week 12
Baseline and Week 12
Number of Participants With Dialysis
Time Frame: At Week 12
At Week 12
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
Time Frame: Baseline and Week 12
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.
Baseline and Week 12
Absolute Change From Baseline in New York Heart Association (NYHA) Class
Time Frame: Baseline and Week 12
Baseline 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)
From first dose of study drug up to end of follow up (up to Week 16)
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
Population pharmacokinetic parameters
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
Population pharmacokinetic parameters
At Week 4 and Week 12
Tmax,ss: Time to Reach Maximum Concentration at Steady State
Time Frame: At Week 4 and Week 12
Population pharmacokinetic parameters
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
Population pharmacokinetic parameters
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
Population pharmacokinetic parameters. Accumulation ratio of Cmax calculated as Cmax at Week 12/Cmax at Week 4.
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
Population pharmacokinetic parameters. Accumulation ratio of AUC calculated as AUC at Week 12/AUC at Week 4.
At Week 4 and Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
AnaCardio AB

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 15, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 30, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 30, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 4, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 8, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 13, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 14, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 11, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AC01-02
  • 2025 (U.S. NIH Grant/Contract: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
  • 2025-524469-25-00 (Ctis)
  • PIP number (Other Identifier: EMA/PE/0000267875)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Time Frame

Links to the Study Protocol and Statistical Analysis Plan will be made available on ClinicalTrials.gov

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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