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- Sperimentazione clinica NCT07584967
A Study to Evaluate the Safety and Efficacy of AC01 Compared to Placebo in Participants With Chronic Heart Failure (GOAL-HF2)
11 settembre 2026 aggiornato da: 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.
Panoramica dello studio
Stato
Non ancora reclutamento
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Interventistico
Iscrizione (Stimato)
400
Fase
- Fase 2
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Robert Edfors, MD, PhD
- Numero di telefono: +46 760 542 609
- Email: studyinfo@anacardio.com
Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Descrizione
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.
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Quadruplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: AC01 3 mg
Participants will receive AC01 orally twice daily (BID) for 12 weeks.
|
AC01 tablets for oral administration.
|
|
Sperimentale: AC01 1 mg
Participants will receive AC01 orally BID for 12 weeks.
|
AC01 tablets for oral administration.
|
|
Comparatore placebo: Placebo
Participants will receive matching placebo orally BID for 12 weeks.
|
AC01 matching placebo tablets for oral administration.
|
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Absolute Change from Baseline in Composite Echocardiography Z-Score at Week 12
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Absolute Change from Baseline in LVSV at Week 12
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in LVEF at Week 12
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in LVESV at Week 12
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change From Baseline in LAVImin at Week 12
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: At Week 12
|
At Week 12
|
|
|
Change From Baseline in Guideline-directed Medical Therapy (GDMT) Use for HFrEF at Week 12
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Absolute Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Lasso di tempo: Baseline and Week 12
|
Baseline and Week 12
|
|
|
Number of Participants With Dialysis
Lasso di tempo: At Week 12
|
At Week 12
|
|
|
Absolute Change from Baseline in Council on Nutrition Appetite Questionnaire (CNAQ) Total Score
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
|
Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Stimato)
15 novembre 2026
Completamento primario (Stimato)
30 luglio 2028
Completamento dello studio (Stimato)
30 agosto 2028
Date di iscrizione allo studio
Primo inviato
4 maggio 2026
Primo inviato che soddisfa i criteri di controllo qualità
8 maggio 2026
Primo Inserito (Effettivo)
13 maggio 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
14 settembre 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
11 settembre 2026
Ultimo verificato
1 settembre 2026
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- AC01-02
- 2025 (Sovvenzione/contratto NIH degli Stati Uniti: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-524469-25-00 (Ctis)
- PIP number (Altro identificatore: EMA/PE/0000267875)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
SÌ
Descrizione del piano 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.
Periodo di condivisione IPD
Links to the Study Protocol and Statistical Analysis Plan will be made available on ClinicalTrials.gov
Tipo di informazioni di supporto alla condivisione IPD
- STUDIO_PROTOCOLLO
- LINFA
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
Sì
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
prodotto fabbricato ed esportato dagli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .