The Efficacy and Safety of Dapagliflozin in the Treatment of Hereditary Kidney Disease With Proteinuria in Children

Launched by CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY · Mar 17, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called dapagliflozin to see how well it works and how safe it is for treating hereditary kidney diseases in children who have high levels of protein in their urine, a condition known as proteinuria. The trial aims to recruit children aged 2 to 17 who have been diagnosed with hereditary kidney disease, either through genetic testing or based on family history and clinical observations. To qualify, children must show specific signs of kidney issues, such as having a certain level of protein in their urine and a good kidney function measured by an estimated glomerular filtration rate (eGFR).

Participants in this study will be closely monitored and will need to be stable on their current medication for kidney issues for at least four weeks before joining. The trial will not be recruiting just yet, but when it does, families will need to give their consent for their child to participate. If eligible, children can expect to be part of a study that could help improve treatment options for hereditary kidney diseases in the future. It's important to note that there are some criteria that would exclude a child from participation, such as having diabetes or certain other medical conditions.

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Eligibility criteria

• Inclusion Criteria:
• • Confirmed diagnosis of hereditary kidney disease (identification of pathogenic genes through molecular genetic testing; for Alport syndrome, molecular diagnosis is not necessarily required if diagnosed based on clinical and pathological findings; for those with a clear family history and a high clinical suspicion of hereditary kidney disease).
• • 24 - hour urinary protein level \> 0.2 g or urinary protein to creatinine ratio (UPCR) \> 0.2 mg/mg.
• • Calculate the estimated glomerular filtration rate (eGFR) using the Schwartz formula (36.5 \* height in cm / serum creatinine in μmol/L), with eGFR ≥ 60 ml/min/1.73 m².
• • Stable use of the basic treatment drug RAASi (including ACEI/ARB) for more than 4 weeks, and no dosage adjustment during the treatment period.
• • Willingness to sign the informed consent form.
• Exclusion Criteria:Exclusion applies if any of the following criteria are met:
• • Treatment with hormones/immunosuppressive agents within the previous 4 weeks.
• • Treatment with SGLT2 inhibitors within the previous 4 weeks.
• • Comorbid diabetes.
• • Uncontrolled urinary tract infection.
• • Evidence of urinary tract obstruction such as dysuria.
• • Blood pressure below the 5th percentile for the same gender, age, and height.
• • Organ transplantation.
• • Tumor.
• • Presence of any of the following definite evidence of liver disease: ALT/AST reaching 2 times the normal value, hepatic encephalopathy, esophageal varices, or portal shunt surgery.
• • Comorbid medical conditions that may affect drug absorption, distribution, metabolism, and excretion, including but not limited to any of the following: active inflammatory bowel disease within the past 6 months, history of major gastrointestinal surgery (such as gastrectomy, gastroenterostomy, intestinal resection), gastrointestinal ulcer, gastrointestinal or rectal bleeding within the past 6 months, pancreatic injury or pancreatitis within the past 6 months.
• • Subjects at risk of dehydration or volume depletion, which may affect drug efficacy or safety.
• • Participation in other drug trials within the previous 4 weeks.
• • Blood loss exceeding 400 ml within the previous 8 weeks.
• • Poor past medication compliance or unwillingness to complete the trial.
• • Any other medical conditions that may place the patient at a higher risk due to participation in this study.