- ICH GCP
- Registr klinických studií v USA
- Klinická studie NCT07584967
A Study to Evaluate the Safety and Efficacy of AC01 Compared to Placebo in Participants With Chronic Heart Failure (GOAL-HF2)
11. září 2026 aktualizováno: 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.
Přehled studie
Postavení
Zatím nenabíráme
Intervence / Léčba
Detailní popis
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.
Typ studie
Intervenční
Zápis (Odhadovaný)
400
Fáze
- Fáze 2
Kontakty a umístění
Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.
Studijní kontakt
- Jméno: Robert Edfors, MD, PhD
- Telefonní číslo: +46 760 542 609
- E-mail: studyinfo@anacardio.com
Kritéria účasti
Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Ne
Popis
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.
Studijní plán
Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Paralelní přiřazení
- Maskování: Čtyřnásobek
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Experimentální: AC01 3 mg
Participants will receive AC01 orally twice daily (BID) for 12 weeks.
|
AC01 tablets for oral administration.
|
|
Experimentální: AC01 1 mg
Participants will receive AC01 orally BID for 12 weeks.
|
AC01 tablets for oral administration.
|
|
Komparátor placeba: Placebo
Participants will receive matching placebo orally BID for 12 weeks.
|
AC01 matching placebo tablets for oral administration.
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12
Časové okno: 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
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Absolute Change from Baseline in LVSV at Week 12
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in LVEF at Week 12
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in LVESV at Week 12
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change From Baseline in LAVImin at Week 12
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: At Week 12
|
At Week 12
|
|
|
Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Časové okno: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Number of Participants With Dialysis
Časové okno: At Week 12
|
At Week 12
|
|
|
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
Časové okno: 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
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
|
Spolupracovníci a vyšetřovatelé
Zde najdete lidi a organizace zapojené do této studie.
Sponzor
Termíny studijních záznamů
Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.
Hlavní termíny studia
Začátek studia (Odhadovaný)
15. listopadu 2026
Primární dokončení (Odhadovaný)
30. července 2028
Dokončení studie (Odhadovaný)
30. srpna 2028
Termíny zápisu do studia
První předloženo
4. května 2026
První předloženo, které splnilo kritéria kontroly kvality
8. května 2026
První zveřejněno (Aktuální)
13. května 2026
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
14. září 2026
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
11. září 2026
Naposledy ověřeno
1. září 2026
Více informací
Termíny související s touto studií
Klíčová slova
Další relevantní podmínky MeSH
Další identifikační čísla studie
- AC01-02
- 2025 (Grant/smlouva NIH USA: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-524469-25-00 (Ctis)
- PIP number (Jiný identifikátor: EMA/PE/0000267875)
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
ANO
Popis plánu IPD
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.
Časový rámec sdílení IPD
Links to the Study Protocol and Statistical Analysis Plan will be made available on ClinicalTrials.gov
Typ podpůrných informací pro sdílení IPD
- PROTOKOL STUDY
- MÍZA
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Ano
Studuje produkt zařízení regulovaný americkým úřadem FDA
Ne
produkt vyrobený a vyvážený z USA
Ne
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .