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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bayer AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

9 Jan 2026 → ongoing

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacodynamic, Therapy, Pharmacokinetic, Safety

To evaluate the effect of finerenone in addition to standard of care (SoC) on reducing NT-proBNP levels compared to placebo

Secondary objectives 3

1. To assess the safety profile of finerenone in addition to standard of care (SoC) in pediatric left ventricular systolic dysfunction (LVSD) patients as compared to placebo
2. To demonstrate the beneficial effect of finerenone in addition to SoC as compared to placebo
3. To confirm the dose and systemic exposure of finerenone in pediatric LVSD patients

Conditions and MedDRA coding

Heart failure due to systemic left ventricular systolic dysfunction

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001623-PIP03-20

Plan to share IPD

No

IPD plan description

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Participants must be 6 months to <18 years old at the time when the informed consent/assent is signed.
2. LV systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography
3. 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
4. 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.
5. 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
6. Study participants must have a body weight ≥ 4.0 kg at Visit 1.

Exclusion criteria 7

1. 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)
2. Severe renal dysfunction with eGFR < 30 ml/min/1.73m2 at screening or randomization visit
3. Systolic blood pressure (SBP) < 5th percentile for age, sex and height at screening or randomization
4. Sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to randomization
5. Treatment with a mineralocorticoid receptor antagonist (e.g., spironolactone, eplerenone) within 30 days of randomization.
6. Requirement of any intravenous (IV) vasoactive agents, mechanical ventilation, mechanical circulatory support within 30 days prior to randomization.
7. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in NT-proBNP from baseline to 3 months in participants receiving active study intervention compared to placebo

Secondary endpoints 8

1. Number of participants with treatment-emergent adverse events (TEAEs)
2. Change in serum potassium levels from baseline to end of treatment (EoT)
3. Change in systolic blood pressure (SBP) from baseline to EoT
4. Change in renal function from baseline to EoT
5. Change in echocardiographic parameters of left ventricular systolic function and size from baseline to EoT
6. Total number of cardiovascular outcome events, including cardiovascular death, heart transplantation, and clinical worsening of heart failure from baseline to EoT
7. Maximum observed finerenone concentration in plasma after multiple doses (Cmax, md)
8. Area under the curve for finerenone concentration in plasma after multiple doses (AUCτ,md)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bayer AG

Sponsor organisation

Bayer AG

Address

-

City

Leverkusen

Postcode

51368

Country

Germany

Scientific contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Public contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Third parties 6

Locations

13 EU/EEA countries · 47 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 253 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications