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Assessment of Efficacy of Mirabegron, a New beta3-adrenergic Receptor in the Prevention of Heart Failure

Phase 2
Completed
Conditions
Hypertrophy, Left Ventricular
Interventions
Procedure: Echocardiography
Procedure: Cardiac MRI
Procedure: Maximal exercise capacity
Procedure: Blood sampling
Procedure: Endothelial function measurement
Radiation: 18FDG-PET
Registration Number
NCT02599480
Lead Sponsor
Jean-Luc Balligand
Brief Summary

This study will assess the efficacy of mirabegron, a new beta3-adrenergic receptor in the prevention of heart failure. This is a two armed, prospective, randomized, placebo-controlled, multi-centric european phase IIb trial with placebo and mirabegron distributed in a 1:1 fashion. The patients enrolled will have cardiac structural remodeling with or without symptoms of heart failure (maximum NYHA II).

Patients will be monitored for change in left ventricular mass (assessed by cardiac MRI) and/or changes in diastolic function (assessed by echocardiography) after 12 months of treatment.

Detailed Description

Background Heart failure (HF) represents a major and growing public health burden. Patients with HF are classically divided into two groups: those with HF with preserved ejection fraction (HFpEF), and those with HF and reduced ejection fraction (HFrEF). As HF is a progressive disorder increasing with age, the proportion of these patients is rising due to the aging of the population. Beside costs, HFpEF also puts a heavy burden on the quality of life of (mostly elderly) patients, with a loss of autonomy and the dyscomfort of repeated hospitalisations. Therefore, HFpEF is a chronic, costly, debilitating disease.

A major contributor to HFpEF is myocardial remodelling, e.g. hypertrophy and fibrosis, as well as cellular functional/structural modifications leading to alteration in contractile properties and ventricular distensibility. Unfortunately, there are currently no evidence-based treatment strategies.

Study claim The proposed clinical trial will provide a proof of concept in humans for the clinical efficacy of a novel therapeutic concept: β3AR activation to attenuate/prevent cardiac remodeling. Recently, a new, specific agonist at human β3-AR (mirabegron) with higher benefit/risk balance was developed and marketed for clinical use in a non-cardiovascular disease (overactive bladder disease). The trial will test the drug repurposing of mirabegron for the prevention of cardiac remodeling leading to HFpEF.

Using pre-clinical models, the investigators demonstrated that activation of β3AR attenuates myocardial hypertrophy and fibrosis in response to neurohormonal or hemodynamic stresses, without compromising LV function. Therefore, the recent availability of this new drug offers the possibility to test the potential benefit of mirabegron (vs placebo) as add-on therapy (on top of standard care) to prevent/delay myocardial remodelling in patients at high risk of developing HFpEF.

Who can participate? Patients with structural cardiac disease with or without HF symptoms (max. NYHA 2).

What does the study involve? Patients will be requested to go 5 times to the hospital to perform cardiac MRI (3X), echocardiography (3X), exercise tolerance test (2X), Pet scanning (2X) and blood sampling (4X).

Who is the sponsor? The Université catholique de Louvain (UCL) is the academic sponsor and Prof. Jean-Luc Balligand is the principal coordinator of the study.

Who is funding the study? Beta3_LVH is an investigator-initiated project funded by a Horizon 2020 grant from the European Commission.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
296
Inclusion Criteria
  • Age between 18 and 90 years
  • Arterial hypertension on stable therapy according to current guideline algorithms (including stable medication for at least four weeks before inclusion),
  • Morphological signs of structural cardiac remodelling by echocardiography, i.e. increased LV mass index (110 g/m2 or higher for female; 134 g/m2 or higher for male subjects (Devereux, Reichek 1977)) or end-diastolic wall thickness > or equal to 13 mm in at least one wall segment
  • Patients may have atrial fibrillation (AF), but with well-regulated ventricular response, i.e. heart rate<100/min (RACE II - (Groenveld et al. 2013, 2013)),
  • Written informed consent
  • For subjects unable to read and/or write, oral informed consent observed by an independent witness is acceptable if the subject has fully understood oral information given by the Investigator. The witness should sign the consent form on behalf of the subject.
Exclusion Criteria
  • Unstable hypertension with systolic BP≥160 mm Hg and/or diastolic BP≥100 mm Hg (based on office measurement, not ambulatory measurement)
  • Documented ischemic cardiac disease
  • History of hospitalization for overt heart failure within last 12 months
  • Patients after heart transplantation
  • Genetic hypertrophic or dilated cardiomyopathy
  • Dysthyroidism.
  • Severe valvulopathy
  • NYHA-class > II
  • BMI >40 kg/m2
  • EF < 50%, regardless of symptoms
  • Known other cause (i.e. COPD) of respiratory dysfunction; patients under positive pressure (CPAP) treatment for sleep apnea syndrome may be included, provided they have been under regular treatment for at least one year before inclusion in the study
  • eGFR < 30 ml/min (by MDRD formula)
  • Abnormal liver function tests
  • Type I diabetes, complicated type II diabetes
  • Patients with anemia
  • Patients with bladder outlet obstruction
  • Patients using antimuscarinic cholinergic drugs for treatment of OAB
  • Current use of digitalis, bupranolol, propranolol, nebivolol
  • Patients continuously treated with Sildenafil or other PDE5 inhibitors.
  • Current use of antifungal azole derivatives (fluconazole, itraconazole, miconazole, posaconazole, voriconazole)
  • Current treatment with mirabegron or indication for future treatment with mirabegron due to other indications
  • Contraindication for MRI
  • Pregnant or nursing women
  • Participation in any other interventional trial
  • Fertile women (within two years of their last menstruation) without appropriate contraceptive measures (hormonal implant, injections, oral contraceptives, intrauterine devices, partner with vasectomy) while participating in the trial (participants using a hormone-based method have to be informed of possible effects from the trial medication on contraception)
  • Contraindication to mirabegron (e.g. hypersensitivity) or any other components of the trial medication

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
mirabegronEchocardiographyPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
mirabegronCardiac MRIPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
mirabegronMaximal exercise capacityPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
mirabegronBlood samplingPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
mirabegronEndothelial function measurementPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
mirabegron18FDG-PETPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
PlaceboEchocardiographyPatients will be orally administererd with a placebo once a day during 12 months.
PlaceboCardiac MRIPatients will be orally administererd with a placebo once a day during 12 months.
PlaceboMaximal exercise capacityPatients will be orally administererd with a placebo once a day during 12 months.
PlaceboBlood samplingPatients will be orally administererd with a placebo once a day during 12 months.
PlaceboEndothelial function measurementPatients will be orally administererd with a placebo once a day during 12 months.
Placebo18FDG-PETPatients will be orally administererd with a placebo once a day during 12 months.
mirabegronmirabegronPatients will be orally administererd with 50 mg of mirabegron once a day during 12 months.
Primary Outcome Measures
NameTimeMethod
Change in left ventricular mass index (LVMI)12 months

Change in left ventricular mass index (LVMI in g/m2, defined as left ventricular mass divided by body surface) measured at baseline and 12 months after randomisation.

Change in diastolic function12 months

Change in diastolic function, assessed as the ratio of peak early transmitral ventricular filling velocity to early diastolic tissue Doppler velocity (E/e') measured at baseline and 12 months after randomisation.

Secondary Outcome Measures
NameTimeMethod
Left atrial volume index12 months

Left atrial volume index at baseline and at 12 months. This parameter determines diastolic filling (and was shown to predict treatment efficacy in HFpEF in the J-DHF trial (Yamamoto et al. 2013))

LV mass index (by cardiac MRI)6 months

LV mass index (by cardiac MRI) at 6 months,

Diastolic function (E/e')6 months

Diastolic function (E/e') at 6 months

metabolic parameters3, 6, 12 months

metabolic parameters (fasting glucose, modified HOMA test, HbA1c, serum lipids)

Maximal exercise capacity12 months

Maximal exercise capacity (peak VO2) at baseline and 12 months.

Cardiac fibrosis12 months

Cardiac fibrosis at baseline and at 12 months. Fibrosis is a key pathogenic mechanism of diastolic dysfunction, which is at the origin of HFpEF

serum biomarkers3, 6, 12 months

serum biomarkers (Galectin3, GDF15, NT-proBNP, hsTnT)

Emergence of treatment-related adverse events12 months

Incidence of Treatment-Emergent Adverse Events

Trial Locations

Locations (10)

Athens University Medical School (NKUA)

🇬🇷

Athens, Greece

Department of Heart Diseases at Wroclaw Medical University (UMW)

🇵🇱

Wroclaw, Poland

Nantes university hospital (CHU Nantes)

🇫🇷

Nantes, France

University Medical Center Göttingen (UMG-GOE)

🇩🇪

Göttingen, Germany

University of Leipzig

🇩🇪

Leipzig, Germany

Hospital "Papa Giovanni XXIII" (HPG23)

🇮🇹

Bergamo, Italy

Cliniques universitaires Saint-Luc

🇧🇪

Brussels, Belgium

Center for Cardiovascular Research Berlin (CCR/Charité)

🇩🇪

Berlin, Germany

Association for Research and Development of the Faculty of Medicine (AIDFM)

🇵🇹

Lisbon, Portugal

University of Oxford - Division of Cardiovascular Medicine (UOXF)

🇬🇧

Oxford, United Kingdom

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