A study to learn more about how safe finerenone is, when it is taken for a longer time with standard treatment, in children and young adults 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, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

27 Apr 2026 → ongoing

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Bayer AG

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety

To demonstrate that finerenone in addition to standard of care (SoC) is safe when given long-term in pediatric left ventricular systolic dysfunction (LVSD) patients

Secondary objectives 3

1. To assess the long-term treatment effect of finerenone in addition to SoC in pediatric LVSD patients
2. To further confirm the systemic exposure of finerenone in pediatric LSVD patients
3. To assess the acceptability and palatability of the age-appropriate pediatric formulation in newborns <6 months of age

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. For participants rolling over from randomized controlled trial (RCT): Prior participation in the finerenone Phase 3 study FIORE (21466) and not permanently discontinued from the study intervention prior to the end of treatment (EoT) visit in FIORE
2. For newly enrolled infants <6 months of age: LV systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography
3. For newly enrolled infants <6 months of age: Elevated NT-pro BNP levels (> 500 mg/L) at screening
4. For newly enrolled infants <6 months of age: Heart failure etiologies include 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 at least 3 months prior to treatment assignment); neuromuscular disorder; inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (e.g., Kawasaki disease and postoperative HF); LV noncompaction
5. For newly enrolled infants <6 months of age: Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion (on a stable regimen for 30 days before baseline).
6. Newly enrolled newborns and infants < 6 months of age must have a body weight of ≥3 kg at Visit 1.

Exclusion criteria 6

1. For participants rolling over from RCT: To roll-over to FIORELLO, all participants: Potassium (K+) >5.5 mmol/L After unblinding: • For participants who received finerenone in FIORE: K+ >5.5 mmol/ L • For participants who received placebo in FIORE: K+ >5.0 mmol/L for children ≥2 years of age, and >5.3 mmol/L for children <2 years of age (if eGFR is <60 mL/min/1.73m² for participants <2 years of age, the serum potassium threshold of >5.0 mmol/L will be used for exclusion).
2. For newly enrolled newborns and infants < 6 months of age: Potassium ≥ 5.3 mmol/l (if eGFR is <60 mL/min/1.73m², the serum potassium threshold of >5.0 mmol/L will be used for exclusion)
3. For participants rolling over from RCT: Severe renal dysfunction with estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2 at FIORE EoT or Visit 1.
4. For newly enrolled infants < 6 months of age: Severe renal dysfunction with eGFR < 30 ml/min/1.73m2 at screening or Visit 1.
5. Treatment with a mineralocorticoid receptor antagonist, other than the study intervention, (e.g., spironolactone, eplerenone) within 30 days of Visit 1.
6. Requirement of any intravenous (IV) vasoactive agents; mechanical ventilation; mechanical circulatory support; sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to study treatment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Number of participants with treatment emergent adverse events (TEAEs)
2. Change in serum potassium levels from baseline to Day 270± 7 (Visit 6, EoT)
3. Change in systolic blood pressure (SBP) from baseline to Day 270± 7 (Visit 6, EoT)
4. Change in estimated glomerular filtration rate (eGFR) from baseline to Day 270± 7 (Visit 6, EoT)

Secondary endpoints 5

1. Change in NT-proBNP from baseline to Day 270± 7 (Visit 6, EoT)
2. Change in key echocardiographic parameters of left ventricular systolic function from baseline to Day 270± 7 (Visit 6, EoT)
3. Maximum observed finerenone concentration in plasma after multiple doses (Cmax, md)
4. Area under the curve for finerenone concentration in plasma after multiple doses (AUCτ,md)
5. Taste and texture questionnaire of the pediatric formulation

Investigational products

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

Test 4

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 158 files

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

Application history

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