E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of children with chronic kidney disease and proteinuria |
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E.1.1.1 | Medical condition in easily understood language |
Chronic kidney disease, which is long-term kidney disease, and proteinuria, a condition in which a person has extra protein in the urine |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064848 |
E.1.2 | Term | Chronic kidney disease |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037032 |
E.1.2 | Term | Proteinuria |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The main objective is to demonstrate that finerenone in addition to an ACEI or ARB is safe when given long-term. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective is to assess the long-term treatment effects on proteinuria and kidney function of finerenone in addition to standard of care. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participants must be ≥1 year to 18 years of age, at the time of signing the informed consent/assent. 2. Prior participation in the finerenone Phase 3 study FIONA (19920) and not permanently discontinued from treatment by the end of treatment (EoT) visit in FIONA. 3. Participants must have a clinical diagnosis of chronic kidney disease (CKD) at Visit 1 which is defined as - CKD stages 1-3 (estimated glomerular filtration rate [eGFR] ≥30 mL/min/1.73m^2) for children ≥1 year to <19 years of age at FIONA EoT and at Visit 1 4. Treated with an angiotensin-converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) at optimized doses defined as maximally tolerable doses within the recommended dose range according to guidelines on blood pressure (BP) management, unchanged for at least 30 days prior to Visit 1. 5. K+ ≤5.0 mmol/L for children ≥2 years of age at both FIONA EoT and Visit 1, and ≤5.3 mmol/L for children <2 years of age at both FIONA EoT and Visit 1 6. Participant is able to receive enteral feeding (solid food, bottle or cup fed, feeding through nasogastric or gastric feeding tubes) with or without breastfeeding. |
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E.4 | Principal exclusion criteria |
1. Planned urological surgery expected to influence renal function 2. Patients who are candidates for renal transplantation, i.e., a kidney transplantation scheduled within the study time frame 3. Systemic hypertension Stage 2 defined according to institutional guidelines on BP management at Visit 1. 4. Systemic hypotension defined as symptomatic hypotension or a mean systolic BP below the 5th percentile for age, sex and height but no lower than 80 mmHg for participants <18 years and symptomatic hypotension or a mean systolic blood pressure (SBP) <90 mmHg in participants ≥18 years at Visit 1. 5. Known hypersensitivity to the study treatment (active substance or excipients) 6. Severe hepatic insufficiency defined by e.g. Child-Pugh C or analogous scores. 7. Participants using rituximab, cyclophosphamide, abatacept, or intravenous glucocorticoids 8. Concomitant therapy with a mineralocorticoid receptor antagonist (MRA) (eplerenone, spironolactone, esaxerenone, canrenone), any renin inhibitor (aliskiren, enalkiren, remikiren), any sodium-glucose co-transporter-2 (SGLT2) inhibitor (SGLT2i), sacubitril/valsartan combination (ARNI), or potassium-sparing diuretic (amiloride, triamterene) 9. Concomitant therapy with both ACEI and ARBs together 10. Concomitant therapy with strong cytochrome P450 isoenzyme 3A4 (CYP3A4) inhibitors, moderate or strong CYP3A4 inducers 11. Previous assignment to treatment during this study 12. Simultaneous participation in another interventional clinical study (e.g., Phase 1 to 4 clinical studies). 13. Experienced a drug-related serious adverse event (SAE) or an adverse event (AE) which led to permanent discontinuation of study intervention during the FIONA study 14. Pregnant or breastfeeding or intention to become pregnant during the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Number of participants with treatment emergent adverse events (TEAEs) 2. Change in serum potassium levels from baseline to Day 540±7 3. Change in systolic blood pressure (SBP) from baseline to Day 540±7 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Change in urinary protein-to-creatinine ratio (UPCR) and urinary albumin-to-creatinine ratio (UACR) from baseline to Day 540±7 2. Change in estimated glomerular filtration rate (eGFR) from baseline to Day 540±7 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 64 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
Israel |
Korea, Republic of |
United States |
Austria |
Finland |
France |
Lithuania |
Poland |
Sweden |
Netherlands |
Spain |
Switzerland |
Czechia |
Germany |
Greece |
Italy |
Belgium |
Denmark |
Hungary |
Portugal |
Turkey |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the study is defined as the date of the last visit of the last participant in the study globally. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 26 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 5 |
E.8.9.2 | In all countries concerned by the trial days | 10 |