Reduced-Dose Steroids for Nephrotic Syndrome

(RESPONSE Trial)

Overseen By Jovanka Vasilevska-Ristovska

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: The Hospital for Sick Children

Must be taking: Prednisone

Disqualifiers: Steroid-resistant nephrotic syndrome, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

Duration: 6 weeks

50 Participants Needed

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.

Research Team

Rulan Parekh, MD MS

Principal Investigator

The Hospital for Sick Children

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

Have not been previously included in the RESPONSE trial

Participant located in Ontario, Canada at the time of study enrolment

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

Exclusion Criteria

Active pregnancy or lactation

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

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either reduced-dose or standard-dose oral prednisone until remission, followed by a maintenance dose for four weeks

6 weeks

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

46 weeks

Periodic visits (in-person)

Treatment Details

Interventions

• Prednisone

Trial Overview 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.

Participant Groups

Group I: Reduced-dose steroids Experimental Treatment 1 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 steroids Active Control 1 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:

• Allergic reactions
• Asthma
• Blood disorders
• Cancer
• Eye problems
• Immune system disorders
• Inflammatory conditions
• Multiple sclerosis
• Organ transplantation
• Rheumatoid arthritis
• Skin conditions

Approved in European Union as Prednisone for:

• Allergic reactions
• Asthma
• Blood disorders
• Cancer
• Eye problems
• Immune system disorders
• Inflammatory conditions
• Multiple sclerosis
• Organ transplantation
• Rheumatoid arthritis
• Skin conditions

Approved in Canada as Prednisone for:

• Allergic reactions
• Asthma
• Blood disorders
• Cancer
• Eye problems
• Immune system disorders
• Inflammatory conditions
• Multiple sclerosis
• Organ transplantation
• Rheumatoid arthritis
• Skin conditions

Who Is Running the Clinical Trial?

The Hospital for Sick Children

Lead Sponsor

Trials

724

Recruited

6,969,000+