A study to find out how EMPAgliflozin is tolerated and if it helps children and adolescents with chronic KIDNEY disease (EMPA-KIDNEY Kids)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

2 Dec 2025 → ongoing

Decision date (initial)

2025-11-07

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-512577-27-00

WHO UTN

U1111-1312-1389

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Safety

The main objective of this trial is to assess the pharmacokinetics, safety, and efficacy of empagliflozin (po) in paediatric patients aged 2 to 17 years with chronic kidney disease at risk of progression

Conditions and MedDRA coding

Chronic kidney disease in paediatric patients

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-000828-PIP06-18

Plan to share IPD

Yes

IPD plan description

"Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed “Document Sharing Agreement”. Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined on the website. Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program. Access Criteria: For study documents – upon signing of a ‚Document Sharing Agreement‘. For study data – 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement."

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 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).
2. Age 2 to 17 years at screening Visit 1.
3. CKD of any underlying aetiology defined by (as measured by central laboratory at screening Visit 1): eGFR (U25Crea) ≥20 to <90 mL/min/1.73 m2 with a UACR ≥300 mg/g
4. Participants must be on a stable dose of maximally tolerated SoC therapy for 30 days before screening Visit 1, with no plans to change the dose throughout the duration of the placebo-controlled portion of the trial. SoC is anticipated to include a single RAAS inhibitor, such as angiotensin receptor blockers (ARB) or angiotensin converting enzyme inhibitors (ACEi), as appropriate and as 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
5. 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 period of the trial.
6. Further inclusion criteria apply

Exclusion criteria 9

1. Confirmed type 1 or type 2 diabetes mellitus.
2. History of ketoacidosis within 8 weeks prior to Visit 1 and up to randomisation.
3. Chronic dialysis or functioning kidney transplant or scheduled for transplantation throughout the duration of the trial.
4. Diagnosis of uncontrolled metabolic bone disease (at the Investigator’s discretion).
5. "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."
6. Gastrointestinal disorders that might interfere with trial drug absorption according to investigator assessment.
7. Presence of acute or active UTI with signs or symptoms of an active UTI or therapeutic treatment for an active UTI within 14 days before screening Visit 1.
8. Severe, uncontrolled hypertension (based on investigator’s judgement).
9. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Change from Day 1 to the Week 24 visit in UACR [mg/g]
2. Change from Day 1 to the Week 24 visit in urine glucose [mmol/L]

Secondary endpoints 5

1. Change in eGFR (U25Crea) over time during treatment with empagliflozin
2. Annual rate of change in eGFR (U25Crea) from Week 8 to Week 24, including treatment effect extrapolation from (adult) EMPA-KIDNEY data
3. Change from Day 1 to the Week 24 visit in UPCR
4. The observed predose plasma concentrations of empagliflozin at Week 26
5. 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)

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Third parties 1

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50, Sant Cugat Del Valles Sant Cugat Del Valles

City

Barcelona

Postcode

08174

Country

Spain

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

10 EU/EEA countries · 42 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 232 files

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

Application history

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