A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)

Официальное название

A Randomised, Double-blind, Placebo-controlled Trial With an Open-label Extension to Assess the Pharmacokinetics, Safety, and Efficacy of Empagliflozin Tablets in Paediatric Patients With Chronic Kidney Disease (EMPA-KIDNEY® Kids)

Обзор

This study is open to children aged 2 to 17 with chronic kidney disease (CKD). The purpose of this study is to find out if a medicine called empagliflozin helps children and adolescents with CKD. Other goals of the study are to find out how empagliflozin is tolerated and handled by the body in children and adolescents with CKD. Participants are put into 2 groups randomly, which means by chance. One group takes empagliflozin and the other group takes placebo. Placebo looks like empagliflozin but does not contain any medicine. Participants are twice as likely to be in the empagliflozin group. Participants take empagliflozin or placebo as tablets once a day for 6 months. After 6 months, participants in both groups take empagliflozin as tablets once a day for 1 year. Participants are in the study for a little over a year and a half. During this time, they visit the study site about 15 times and get at least 5 phone or video calls from the site staff. At the visits, the doctors take blood and urine samples from the participants. The doctors also regularly check participants' health and take note of any unwanted effects.

Вмешательства

• Препарат Empagliflozin
  Empagliflozin
• Препарат Placebo
  Placebo

Первичные конечные точки

• Change from Day 1 to the Week 24 visit in urine albumin-creatinine (UACR) [mg/g] [Срок оценки: Up to week 24]
• Change from Day 1 to the Week 24 visit in urine glucose [mmol/L] [Срок оценки: Up to week 24]

Вторичные конечные точки (5)

• Change in estimated glomerular filtration rate (eGFR) (U25Crea) over time during treatment with empagliflozin [Срок оценки: Up to week 73]
• Annual rate of change in eGFR (U25Crea) from the Week 8 to the Week 24 visit, including treatment effect extrapolation from (adult) EMPA-KIDNEY data [Срок оценки: Up to week 24]
• Change from Day 1 to the Week 24 visit in urine protein-creatinine ratio (UPCR) [Срок оценки: Up to week 24]
• The observed predose plasma concentrations of empagliflozin at the Week 26 visit [Срок оценки: Up to week 26]
• Occurrence of at least one SAE or AE of special interest (AESI) per participant between Day 1 and the Week 24 visit, and between the Week 24 visit and end of treatment (EoT) +7 days residual effect period (REP) [Срок оценки: Up to week 73]

Критерии участия

Критерии включения

• Signed and dated written informed consent provided by the patient's parent(s) (or legal guardian) and patient's assent in accordance with international council for harmonisation good clinical practice (ICH-GCP) and local legislation prior to admission to the trial (informed assent will be sought according to the patient's age, level of maturity, competence, and capacity).
• Age 2 to 17 years at screening Visit 1.
• Chronic kidney disease (CKD) of any underlying aetiology defined by (as measured by central laboratory at screening Visit 1): estimated glomerular filtration rate (eGFR) (U25Crea) ≥20 to <90 mL/min/1.73 m2 with a urine-albumine-creatinine (UACR) ≥300 mg/g
• Participants must be on a stable dose of maximally tolerated standard of care (SoC) therapy for 30 days before screening visit 1 with no plans to change the dose throughout the duration of the placebo-controlled duration of the trial. SoC is anticipated to include a single Renin-angiotensin-aldosterone system (RAAS) inhibitor, such as angiotensin receptor blockers (ARB) or angiotensin converting enzyme inhibitors (ACEi) as appropriate and tolerated. Additional use of a mineralocorticoid receptor antagonist (MRA, including finerenone if available) is permitted if needed and the dose is stable for 30 days before screening Visit 1 and no planned dose changes for the placebo-controlled portion of the trial.
• Participants receiving daily immunosuppressive therapy for an underlying immunological cause of CKD must be on a stable dose for the duration specified for each drug prior to screening and must remain on a stable regimen throughout the placebo-controlled portion of the trial.
• Further inclusion criteria apply.

Критерии исключения

• Confirmed type 1 or type 2 diabetes mellitus.
• History of ketoacidosis within 8 weeks prior to Visit 1 and up to randomisation.
• Chronic dialysis or functioning kidney transplant or scheduled for transplantation throughout the duration of the trial.
• Diagnosis of uncontrolled metabolic bone disease (at the Investigator's discretion).
• Body mass index (BMI) ≤10th percentile for children ≥4 years of age and ≤25th percentile for children <4 years of age according to Centers for Disease Control and Prevention (CDC) growth chart at screening Visit 1.
• Gastrointestinal disorders that might interfere with trial drug absorption according to investigator assessment.
• Presence of acute or active urinary tract infection (UTI) with signs or symptoms of an active UTI or therapeutic treatment for an active UTI within 14 days before screening Visit 1.
• Severe, uncontrolled hypertension (based on investigator's judgement).
• Further exclusion criteria apply.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 30 центров

• University of Alabama at Birmingham — Birmingham
• Phoenix Children's Hospital — Phoenix
• University of California Los Angeles — Los Angeles
• Stanford University School of Medicine — Palo Alto
• University of California Davis — Sacramento
• University of California San Francisco — San Francisco
• University of Miami — Miami
• Emory University — Atlanta
• … и ещё 22 центра

Великобритания · 8 центров

Список центров уточняется — проверьте первичный протокол.

Франция · 6 центров

• Hôpital Louis Pradel — Bron
• HOP Timone — Marseille
• HOP Enfants et Adolescents — Nantes
• HOP Armand-Trousseau — Paris
• Hopital Necker — Paris
• HOP des Enfants — Toulouse

Бельгия · 5 центров

• HUB CHU Brugmann — Brussels
• Cliniques Universitaires Saint-Luc — Brussels
• Universitair Ziekenhuis Gent — Ghent
• UZ Leuven — Leuven
• CHC Mont Légia — Liège

Канада · 5 центров

• University of Alberta Hospital (University of Alberta) — Edmonton
• BC Children's Hospital — Vancouver
• The Hospital for Sick Children — Toronto
• McGill University Health Centre (MUHC) — Montreal
• Jim Pattison Children's Hospital — Saskatoon

Нидерланды · 5 центров

• Amsterdam University Medical Center — Amsterdam
• Universitair Medisch Centrum Groningen — Groningen
• Radboud Universitair Medisch Centrum — Nijmegen
• Erasmus Medisch Centrum — Rotterdam
• UMC Utrecht — Utrecht

Польша · 5 центров

• Uniwersyteckie Centrum Kliniczne — Gdansk
• University Children's Hospital in Lublin — Lublin
• Wojewodzki Szpital Zespolony Im.L.Rydygiera W Toruniu — Torun
• The Children's Memorial Health Institute — Warsaw
• University Clinical Hospital in Wrocław — Wroclaw

Австралия · 4 центра

• The Children's Hospital at Westmead — Westmead
• Queensland Children's Hospital — South Brisbane
• Monash Children's Hospital — Clayton
• The Royal Children's Hospital — Parkville

Германия · 4 центра

• Universitätsklinikum Köln (AöR) — Cologne
• Universitätsklinikum Hamburg, Eppendorf — Hamburg
• Universitätsklinikum Heidelberg — Heidelberg
• Universitätsklinikum Tübingen — Tübingen

Италия · 4 центра

• Istituto G. Gaslini — Genova
• Fondazione IRCCS Ca'Granda-Ospedale Maggiore Policlinico — Milan
• Azienda Ospedaliera Universitaria di Padova — Padova
• Ospedale Pediatrico Bambino Gesù — Roma

Португалия · 4 центра

• CHUC - Centro Hospitalar e Universitario de Coimbra, EPE — Coimbra
• ULS de São José, E.P.E. - Hospital Dona Estefânia — Lisbon
• ULS de Santa Maria, E.P.E — Lisbon
• CHUP, EPE - Centro Materno Infantil do Norte — Porto

South Korea · 4 центра

Список центров уточняется — проверьте первичный протокол.

Испания · 4 центра

Список центров уточняется — проверьте первичный протокол.

Turkey (Türkiye) · 4 центра

Список центров уточняется — проверьте первичный протокол.

Аргентина · 3 центра

• Hospital Italiano de Buenos Aires — CABA
• Hospital de Niños Dr. Ricardo Gutierrez — CABA
• Equipo Ciencia — CABA

Венгрия · 3 центра

• Semmelweis University, Faculty of Medicine — Budapest
• University of Debrecen Clinical Centre — Debrecen
• University of Pecs — Pécs

Сингапур · 2 центра

• National University Hospital — Singapore
• KK Women's and Children's Hospital — Singapore

Швеция · 2 центра

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07107945 · 1245-0256 · 2024-512577-27-00 · U1111-1312-1389

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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