- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07584967
A Study to Evaluate the Safety and Efficacy of AC01 Compared to Placebo in Participants With Chronic Heart Failure (GOAL-HF2)
11. september 2026 opdateret af: 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.
Studieoversigt
Status
Ikke rekrutterer endnu
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
400
Fase
- Fase 2
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: Robert Edfors, MD, PhD
- Telefonnummer: +46 760 542 609
- E-mail: studyinfo@anacardio.com
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
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.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: AC01 3 mg
Participants will receive AC01 orally twice daily (BID) for 12 weeks.
|
AC01 tablets for oral administration.
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Eksperimentel: AC01 1 mg
Participants will receive AC01 orally BID for 12 weeks.
|
AC01 tablets for oral administration.
|
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Placebo komparator: Placebo
Participants will receive matching placebo orally BID for 12 weeks.
|
AC01 matching placebo tablets for oral administration.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12
Tidsramme: 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
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Absolute Change from Baseline in LVSV at Week 12
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in LVEF at Week 12
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Absolute Change from Baseline in LVESV at Week 12
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Absolute Change From Baseline in LAVImin at Week 12
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Absolute Change From Baseline in Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS) at Week 12
Tidsramme: 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.
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Baseline and Week 12
|
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Absolute Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 12
Tidsramme: 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.
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Baseline and Week 12
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Patient Global Impression of Change (PGIC) at Week 12
Tidsramme: 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.
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At Week 12
|
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Number of Participants With Death, Cardiovascular Death, Hospitalization, Cardiovascular Hospitalization, Heart Failure Hospitalization, Heart Failure Events, Other Adjudicated Events, and Listed for Heart Transplantation
Tidsramme: At Week 12
|
At Week 12
|
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Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
|
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Number of Participants With Dialysis
Tidsramme: At Week 12
|
At Week 12
|
|
|
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
Tidsramme: 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.
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Baseline and Week 12
|
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Absolute Change From Baseline in New York Heart Association (NYHA) Class
Tidsramme: Baseline and Week 12
|
Baseline and Week 12
|
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Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)
Tidsramme: From first dose of study drug up to end of follow up (up to Week 16)
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From first dose of study drug up to end of follow up (up to Week 16)
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AUC,ss: Area Under the Concentration-Time Curve at Steady State of AC01 and its Metabolite M6
Tidsramme: At Week 4 and Week 12
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Population pharmacokinetic parameters
|
At Week 4 and Week 12
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Cmax,ss: Maximum Observed Concentration at Steady State of AC01 and its Metabolite M6
Tidsramme: At Week 4 and Week 12
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Population pharmacokinetic parameters
|
At Week 4 and Week 12
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Tmax,ss: Time to Reach Maximum Concentration at Steady State
Tidsramme: At Week 4 and Week 12
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Population pharmacokinetic parameters
|
At Week 4 and Week 12
|
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Ctrough: Trough concentration at steady state of AC01 and its Metabolite M6
Tidsramme: At Week 4 and Week 12
|
Population pharmacokinetic parameters
|
At Week 4 and Week 12
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Rac (Cmax): Accumulation Ratio of Cmax for AC01 and its Metabolite M6
Tidsramme: At Week 4 and Week 12
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Population pharmacokinetic parameters.
Accumulation ratio of Cmax calculated as Cmax at Week 12/Cmax at Week 4.
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At Week 4 and Week 12
|
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Rac (AUC): Accumulation Ratio of AUC for AC01 and its Metabolite M6
Tidsramme: 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
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Anslået)
15. november 2026
Primær færdiggørelse (Anslået)
30. juli 2028
Studieafslutning (Anslået)
30. august 2028
Datoer for studieregistrering
Først indsendt
4. maj 2026
Først indsendt, der opfyldte QC-kriterier
8. maj 2026
Først opslået (Faktiske)
13. maj 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
14. september 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
11. september 2026
Sidst verificeret
1. september 2026
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- AC01-02
- 2025 (U.S. NIH-bevilling/kontrakt: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-524469-25-00 (Ctis)
- PIP number (Anden identifikator: EMA/PE/0000267875)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
JA
IPD-planbeskrivelse
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-delingstidsramme
Links to the Study Protocol and Statistical Analysis Plan will be made available on ClinicalTrials.gov
IPD-deling Understøttende informationstype
- STUDY_PROTOCOL
- SAP
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
produkt fremstillet i og eksporteret fra U.S.A.
Ingen
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