Reduced-Dose Steroids for Nephrotic Syndrome (RESPONSE Trial)

Palo Alto (17 mi)

Overseen byRulan Parekh, MD MS

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: The Hospital for Sick Children

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 3 jurisdictions

Trial Summary

What is the purpose of this trial?This is a pilot feasibility study for a proposed full-scale randomized controlled trial to evaluate the effectiveness and safety of a reduced-dose oral prednisone (steroids) regimen to treat childhood steroid-sensitive nephrotic syndrome relapses versus standard-dose prednisone (i.e., usual standard of care). This internal pilot study is a single-center, open-label, randomized controlled trial at The Hospital for Sick Children (Toronto, ON, Canada). The primary objective of this pilot study is to determine the feasibility, safety, and resources needed to conduct the future full-scale randomized controlled trial.

Eligibility Criteria

This trial is for children with steroid-sensitive nephrotic syndrome, which includes conditions like lipoid nephrosis and minimal change disease. Participants must be able to take oral medication.

Inclusion Criteria

I have been diagnosed with a specific kidney condition involving high protein in urine, low blood protein, or swelling.

I can take pills and will follow the study's prednisone plan.

I am currently experiencing a relapse of nephrotic syndrome.

I am between 1 and 18 years old.

Exclusion Criteria

I have had blood clots during a relapse of my condition.

My condition worsened again less than 6 weeks ago.

I am currently on a high-dose prednisone therapy.

My kidney condition was diagnosed before I turned 1 or is caused by a single gene.

I have experienced a recent worsening in my kidney function.

I have severe swelling in my limbs that leaves a deep indent.

I have been hospitalized for my current relapse.

Treatment Details

The study tests a reduced-dose of the oral medication prednisone against the standard dose typically used to treat relapses of childhood nephrotic syndrome. It's an initial test run at one medical center before a larger study.

2Treatment groups

Experimental Treatment

Active Control

Group I: Reduced-dose steroidsExperimental Treatment1 Intervention

Reduced-dose steroid protocol (intervention): oral prednisone or prednisolone 30mg/m2 (1mg/kg; max 40mg) daily until remission, then 20mg/m2 (0.66mg/kg; max 25mg) on alternate days for four weeks.

Group II: Standard-dose steroidsActive Control1 Intervention

Standard-dose steroid protocol (control): oral prednisone or prednisolone 60mg/m2 (2mg/kg; max 60mg) daily until remission, then 40mg/m2 (1.5mg/kg; max 50mg) on alternate days for four weeks.

Prednisone is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Prednisone for: