REduced-dose Steroid PrOtocol for Childhood Nephrotic SyndromE (RESPONSE)

Launched by THE HOSPITAL FOR SICK CHILDREN · Oct 8, 2024

Keywords

ClinConnect Summary

The RESPONSE trial is a study looking at how effective and safe a lower dose of prednisone (a type of steroid medication) is for treating children with nephrotic syndrome, which is a condition that causes swelling and protein leakage in urine. This trial is taking place at The Hospital for Sick Children in Toronto and aims to see if this reduced-dose treatment works as well as the usual higher dose. Right now, the study is in its early phase, focusing on making sure it can be done safely and effectively before starting a larger trial.

To be eligible for the study, children aged 1 to 18 must have been diagnosed with nephrotic syndrome and be experiencing a relapse at the time of enrollment. They should be able to take oral medication and follow some monitoring instructions at home. Participants will receive either the lower or standard dose of prednisone and will be closely monitored for two weeks to check their symptoms and urine. If you or someone you know is interested in participating, you can learn more about the study and see if you meet the criteria!

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Provide informed consent ± assent
• • 2. Participant age 1-18 years
• • 3. Diagnosis of idiopathic nephrotic syndrome (defined as nephrotic-range proteinuria \[first morning or 24-hour urine protein/creatinine ratio ≥200mg/mmol or ≥3+ protein on dipstick\] and either hypoalbuminemia \[serum albumin \<30g/L\] or edema)
• • 4. Active nephrotic syndrome relapse at time of enrolment (defined as recurrence of nephrotic-range proteinuria \[≥3+ protein on dipstick for ≥3 consecutive days18 OR first morning or 24-hour urine protein/creatinine ratio ≥200mg/mmol AND ≥1+ protein on dipstick for ≥3 consecutive days\])
• • 5. Ability to take oral medication and willingness to adhere to either study prednisone regimen
• • 6. Ability and willingness to adhere to home urine and symptom monitoring during the initial two-week period after assigned treatment initiation
• • 7. Have not been previously included in the RESPONSE trial
• • 8. Participant located in Ontario, Canada at the time of study enrolment
• Exclusion Criteria:
• • 1. Prednisone treatment (at any dose) for the active relapse episode for \>2-days prior to study enrolment
• • 2. Relapse episode within the past 6-weeks (i.e., date of relapse onset within 6-weeks prior to date of enrolment)
• • 3. Current receipt of high-dose maintenance prednisone therapy (dose \>0.6mg/kg on alternate days or \>0.3mg/kg daily)
• • 4. Steroid-resistant nephrotic syndrome classification (defined as lack of complete remission within 6-weeks after initiating daily steroid treatment at a standard dose for the initial episode of nephrotic syndrome)
• • 5. Congenital or monogenic cause of nephrotic syndrome (defined as age at diagnosis \<1-year or known/suspected monogenic cause of nephrotic syndrome)
• • 6. Secondary cause of nephrotic syndrome (includes membranous nephropathy, post-infectious glomerulonephritis \[GN\], complement-mediated GN \[e.g., C3 glomerulopathy and immune complex-GN\], IgA nephropathy, IgA vasculitis, lupus nephritis, medication-induced nephrotic syndrome, malignancy-induced nephrotic syndrome, active hepatitis B or C infection, or active HIV infection)
• • 7. Presence of moderate-to-severe peripheral edema (grade 3+; indentation depth ≥5mm and rebound time \>15 seconds)
• • 8. Hospitalization since the onset of the active relapse episode
• • 9. Acute kidney injury (KDIGO stage ≥1) since the onset of the active relapse episode
• • 10. Active or prior known or suspected venous thromboembolism during a relapse episode
• • 11. Active pregnancy or lactation
• • 12. Any condition or diagnosis, that could in the opinion of the Principal Investigator or delegate interfere with the participant's ability to comply with study instructions, might confound the interpretation of the study results, or put the participant at risk