Brief Summary
Researchers are looking for a better way to treat children who have heart failure with left ventricular systolic dysfunction (LVSD). Heart failure is a serious condition where the heart is unable to pump enough blood to meet the body's needs. This can lead to symptoms like shortness of breath, fatigue, and poor growth in children.
The study treatment, finerenone (also called BAY94-8862), works by blocking a protein involved in inflammation, scarring, and thickening of the heart and blood vessels. This may help the heart to pump blood more effectively. This is the first study to explore its use specifically for children with heart failure and LVSD.
The main purpose of this study is to learn if finerenone works to help the heart compared to placebo in children with heart failure and LVSD. For this, the researchers will collect and analyze data on the levels of a protein called NT-proBNP in the blood, which indicates heart stress, and monitor the safety of the treatment.
The study will include children with heart failure and LVSD aged from 6 months to less than 18 years. The study participants will be randomly assigned to one of two treatment groups. Based on their group, they will receive either finerenone or a placebo for a duration of 3 months. A placebo looks like a treatment but does not have any medicine in it. Throughout the study, all participants will continue to receive their standard heart failure treatments.
At the start of this study, the doctors will check each participant's medical history and current medications. If participants qualify for the treatment phase, they will undergo treatment for about 90 days. During this time, they will visit the study site at least 3 times. During these visits, the participants will:
* have their blood pressure, heart rate, temperature, respiratory rate, height and weight measured
* have their heart examined by electrocardiogram (ECG) and echocardiogram
* have blood samples taken
* have physical examinations
* answer questions about their medication and whether they have any adverse events, or have their parents or guardians' answers
An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.
After the initial three-month study, eligible participants will have the option to join a nine-month open-label extension study where all will receive finerenone. Participants who choose not to enroll in the extension will have a follow-up visit 30 days after their last treatment.
The study treatment, finerenone (also called BAY94-8862), works by blocking a protein involved in inflammation, scarring, and thickening of the heart and blood vessels. This may help the heart to pump blood more effectively. This is the first study to explore its use specifically for children with heart failure and LVSD.
The main purpose of this study is to learn if finerenone works to help the heart compared to placebo in children with heart failure and LVSD. For this, the researchers will collect and analyze data on the levels of a protein called NT-proBNP in the blood, which indicates heart stress, and monitor the safety of the treatment.
The study will include children with heart failure and LVSD aged from 6 months to less than 18 years. The study participants will be randomly assigned to one of two treatment groups. Based on their group, they will receive either finerenone or a placebo for a duration of 3 months. A placebo looks like a treatment but does not have any medicine in it. Throughout the study, all participants will continue to receive their standard heart failure treatments.
At the start of this study, the doctors will check each participant's medical history and current medications. If participants qualify for the treatment phase, they will undergo treatment for about 90 days. During this time, they will visit the study site at least 3 times. During these visits, the participants will:
* have their blood pressure, heart rate, temperature, respiratory rate, height and weight measured
* have their heart examined by electrocardiogram (ECG) and echocardiogram
* have blood samples taken
* have physical examinations
* answer questions about their medication and whether they have any adverse events, or have their parents or guardians' answers
An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.
After the initial three-month study, eligible participants will have the option to join a nine-month open-label extension study where all will receive finerenone. Participants who choose not to enroll in the extension will have a follow-up visit 30 days after their last treatment.
Brief Title
A Study to Learn More About How Well Finerenone Works, How Safe it is, and How it Moves Into, Through, and Out of the Body Compared to Placebo When Taken With Standard Treatment in Children With Heart Failure and Left Ventricular Systolic Dysfunction
Categories
Central Contacts
Central Contact Role
Contact
Central Contact Phone
(+)1-888-84 22937
Central Contact Email
clinical-trials-contact@bayer.com
Completion Date
Completion Date Type
Estimated
Conditions
Left Ventricular Systolic Dysfunction
Heart Failure (Pediatric)
Eligibility Criteria
Inclusion Criteria:
* Participants must be 6 months to \<18 years old at the time when the informed consent/assent is signed.
* Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography.
* Elevated NT-pro BNP levels
* \>500 ng/l for children ≥ 6 months to \< 2 years of age
* \>300 ng/l, for children ≥ 2 years to \<18 years
* Heart failure etiologies including congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode was at least 3 months prior to randomization); neuromuscular disorder (eg, duchenne muscular dystrophy); inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (eg, Kawasaki disease and postoperative heart failure \[HF\]); LV noncompaction.
* Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion and being on a stable regimen for 30 days prior to randomization.
* Study participants must have a body weight ≥ 4.0 kg at Visit 1.
Exclusion Criteria:
* Serum potassium:
* \> 5.0 mmol/L for children ≥ 2 years of age at either screening or randomization visit
* \> 5.3 mmol/L for children ≥ 6 months to \< 2 years of age at either screening or randomization visit (if estimated glomerular filtration rate \[eGFR\] \< 60 mL/min/1.73m², threshold of \> 5.0 mmol/L will be used)
* Severe renal dysfunction with eGFR \< 30 ml/min/1.73m² at screening or randomization visit.
* Systolic blood pressure (SBP) \< 5th percentile for age, sex and height at screening or randomization.
* Sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to randomization.
* Treatment with a mineralocorticoid receptor antagonist (e.g., spironolactone, eplerenone) within 30 days of randomization.
* Requirement of any intravenous (IV) vasoactive agents, mechanical ventilation, mechanical circulatory support within 30 days prior to randomization.
* Recent surgical procedure or other intervention to correct or palliate CHD within 3 months prior to randomization or anticipated to undergo cardiac surgery during the 3 months after randomization.
* Participants must be 6 months to \<18 years old at the time when the informed consent/assent is signed.
* Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography.
* Elevated NT-pro BNP levels
* \>500 ng/l for children ≥ 6 months to \< 2 years of age
* \>300 ng/l, for children ≥ 2 years to \<18 years
* Heart failure etiologies including congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode was at least 3 months prior to randomization); neuromuscular disorder (eg, duchenne muscular dystrophy); inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (eg, Kawasaki disease and postoperative heart failure \[HF\]); LV noncompaction.
* Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion and being on a stable regimen for 30 days prior to randomization.
* Study participants must have a body weight ≥ 4.0 kg at Visit 1.
Exclusion Criteria:
* Serum potassium:
* \> 5.0 mmol/L for children ≥ 2 years of age at either screening or randomization visit
* \> 5.3 mmol/L for children ≥ 6 months to \< 2 years of age at either screening or randomization visit (if estimated glomerular filtration rate \[eGFR\] \< 60 mL/min/1.73m², threshold of \> 5.0 mmol/L will be used)
* Severe renal dysfunction with eGFR \< 30 ml/min/1.73m² at screening or randomization visit.
* Systolic blood pressure (SBP) \< 5th percentile for age, sex and height at screening or randomization.
* Sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to randomization.
* Treatment with a mineralocorticoid receptor antagonist (e.g., spironolactone, eplerenone) within 30 days of randomization.
* Requirement of any intravenous (IV) vasoactive agents, mechanical ventilation, mechanical circulatory support within 30 days prior to randomization.
* Recent surgical procedure or other intervention to correct or palliate CHD within 3 months prior to randomization or anticipated to undergo cardiac surgery during the 3 months after randomization.
Inclusion Criteria
Inclusion Criteria:
* Participants must be 6 months to \<18 years old at the time when the informed consent/assent is signed.
* Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography.
* Elevated NT-pro BNP levels
* \>500 ng/l for children ≥ 6 months to \< 2 years of age
* \>300 ng/l, for children ≥ 2 years to \<18 years
* Heart failure etiologies including congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode was at least 3 months prior to randomization); neuromuscular disorder (eg, duchenne muscular dystrophy); inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (eg, Kawasaki disease and postoperative heart failure \[HF\]); LV noncompaction.
* Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion and being on a stable regimen for 30 days prior to randomization.
* Study participants must have a body weight ≥ 4.0 kg at Visit 1.
* Participants must be 6 months to \<18 years old at the time when the informed consent/assent is signed.
* Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography.
* Elevated NT-pro BNP levels
* \>500 ng/l for children ≥ 6 months to \< 2 years of age
* \>300 ng/l, for children ≥ 2 years to \<18 years
* Heart failure etiologies including congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode was at least 3 months prior to randomization); neuromuscular disorder (eg, duchenne muscular dystrophy); inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (eg, Kawasaki disease and postoperative heart failure \[HF\]); LV noncompaction.
* Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion and being on a stable regimen for 30 days prior to randomization.
* Study participants must have a body weight ≥ 4.0 kg at Visit 1.
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
Maximum Age
17 Years
Minimum Age
6 Months
NCT Id
NCT07188805
Org Class
Industry
Org Full Name
Bayer
Org Study Id
21466
Overall Status
Recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Investigate the Efficacy, Safety, and PK/ PD of Finerenone, in Addition to Standard-of-care, in Pediatric Patients, 6 Months to < 18 Years of Age With Heart Failure (HF) and Left Ventricular Systolic Dysfunction (LVSD)
Primary Outcomes
Outcome Measure
Change in NT-proBNP levels
Outcome Time Frame
From baseline to Day 90±3
Secondary Ids
Secondary Id
2024-519829-38-00
Secondary Outcomes
Outcome Description
TEAEs will be mapped to Medical Dictionary for Regulatory Activities (MedDRA) terms
Outcome Time Frame
From the start of study intervention to last study intervention (up to 93 days) + 3 days
Outcome Measure
Number of participants with treatment-emergent adverse events (TEAEs)
Outcome Time Frame
From baseline to Day 90±3
Outcome Measure
Change in serum potassium levels
Outcome Time Frame
From baseline to Day 90±3
Outcome Measure
Change in estimated glomerular filtration rate (eGFR)
Outcome Time Frame
From baseline to Day 90±3
Outcome Measure
Change in systolic blood pressure (SBP)
Outcome Description
Change measured by echocardiogram (%) from baseline to Day 90±3
Outcome Time Frame
From baseline to Day 90±3
Outcome Measure
Change in left ventricular systolic function
Outcome Time Frame
From baseline to Day 90±3
Outcome Measure
Total number of cardiovascular outcome events, including cardiovascular death, heart transplantation, and clinical worsening of heart failure
Outcome Time Frame
Pre-dose, post-dose (1.5-5 hours after intake of intervention), up to day 90±3
Outcome Measure
Maximum observed finerenone concentration in plasma after multiple doses (Cmax, md)
Outcome Time Frame
Pre-dose, post-dose (1.5-5 hours after intake of intervention), up to day 90±3
Outcome Measure
Area under the curve for finerenone concentration in plasma after multiple doses (AUCτ,md)
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
17
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Maryanne Chrisant
Investigator Email
mchrisant@montefiore.org
Investigator Sponsor Organization
External
Study Department
Pediatrics
Study Division
Pediatrics Cardiology
Categories Mesh Debug
Heart/Cardiovascular --- HEART FAILURE
Brain, Spinal Cord & Nervous System --- HEART DISEASES
Heart/Cardiovascular --- HEART DISEASES
Blood Disorders --- CARDIOVASCULAR DISEASES
Blood & Bone Marrow Cancers --- CARDIOVASCULAR DISEASES
Heart/Cardiovascular --- CARDIOVASCULAR DISEASES
MeSH Terms
VENTRICULAR DYSFUNCTION, LEFT
HEART FAILURE
VENTRICULAR DYSFUNCTION
HEART DISEASES
CARDIOVASCULAR DISEASES
FINERENONE