PHARMACOKINETICS, SAFETY AND EFFICACY OF EMPAGLIFLOZIN IN PAEDIATRIC PATIENTS WITH CHRONIC KIDNEY DISEASE: DESIGN OF THE EMPA-KIDNEY KIDS RANDOMISED CONTROLLED TRIAL

 

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https://storage.unitedwebnetwork.com/files/1099/afb85821518c2d06847857deba7a484b.pdf
PHARMACOKINETICS, SAFETY AND EFFICACY OF EMPAGLIFLOZIN IN PAEDIATRIC PATIENTS WITH CHRONIC KIDNEY DISEASE: DESIGN OF THE EMPA-KIDNEY KIDS RANDOMISED CONTROLLED TRIAL

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Salim
Hammad
Louise Oni lw1@liverpool.ac.uk University of Liverpool Department of Women's and Children's Health Liverpool United Kingdom - Great Ormond Street Hospital for Children Department of Paediatric Nephrology London United Kingdom University College London Department of Renal Medicine London United Kingdom
Howard Trachtman howardtrachtman21@gmail.com University of Michigan Department of Pediatrics, Division of Nephrology Michigan United States -
William E. Smoyer William.Smoyer@nationwidechildrens.org The Ohio State University The Research Institute at Nationwide Children's Hospital Columbus United States -
Svenja Seide svenja.seide@boehringer-ingelheim.com Boehringer Ingelheim Pharma GmbH & Co.KG Global Biostatistics & Data Sciences Ingelheim Germany -
Salim Hammad salim.hammad@boehringer-ingelheim.com Boehringer Ingelheim International GmbH Global Clinical Development Ingelheim Germany *
Jennifer McKenzie jennifer.mckenzie@boehringer-ingelheim.com Boehringer Ingelheim Pharmaceuticals, Inc Global Clinical Development Ridgefield, Connecticut United States -
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To describe the design of the EMPA-KIDNEY KIDS randomised controlled trial (RCT), which will evaluate the pharmacokinetics (PK), safety, and efficacy of oral empagliflozin in children with chronic kidney disease (CKD) at increased risk of progression. 

This randomised, double-blind, parallel-group, 2-arm, multicentre, international Phase III RCT will include a 4-week screening period, a 24-week double-blind placebo-controlled period, and a 48-week open-label period in which all participants will receive empagliflozin.

Eligibility criteria include age 2-17 years and CKD of any aetiology deemed to be at increased risk of progression, estimated glomerular filtration rate [eGFR] 20-90 mL/min/1.73 m2 determined using the U25 equation based on creatinine, and  urine albumin-creatinine ratio [UACR] ≥300 mg/g. Participants will be randomised 2:1 to once daily empagliflozin or placebo in addition to standard of care. Empagliflozin dosing will be weight-based:10mg (>50kg); 5mg (20kg-50 kg) and 2.5 mg (<20 kg).

The primary endpoint is a “primary endpoint family” with two members: absolute change in UACR and absolute change in urine glucose, each from Day 1 to Week 24. Secondary outcomes include changes in eGFR, urine protein-creatinine ratio, PK, and safety assessments.  Urinary biomarkers and plasma empagliflozin concentrations (pre- and post-dose) will also be assessed. Vital signs, including home weight measurements to monitor fluid status, will be monitored.

Primary and final analyses will occur after the randomised and open-label periods, respectively. Primary outcomes will be assessed using mixed-model repeated measures. Subgroup analyses of efficacy endpoints by disease aetiology (glomerular vs non-glomerular) are also planned.

The target sample is 120 participants (80 empagliflozin and 40 placebo). The statistical power for the primary endpoint family is >90% with an assumed geometric mean (gmean) ratio of 0.78 for change from Day 1 to Week 24 in UACR, and a mean difference of 57 mmol/L for change from Day 1 to Week 24 in urine glucose.

EMPA-KIDNEY Kids is the first study to provide high-quality PK, safety, and efficacy data for the SGLT2 inhibitor  empagliflozin in children aged 2-17 years with the full spectrum of CKD who are at increased risk of disease progression. Global enrolment is planned to begin in 19 countries in November 2025.

Previously presented at the 57th European Society for Paediatric Nephrology Annual Meeting, Athens, Greece, 15–18 October 2025.

Kewords