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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether a lower dose of steroids is as effective and safe as the standard dose for treating childhood nephrotic syndrome relapses. Nephrotic syndrome is a kidney condition that causes swelling and protein in the urine. The trial compares two groups: one taking a reduced dose of prednisone (a type of steroid) and the other taking the standard dose. Children in Ontario, Canada, who are experiencing a relapse of nephrotic syndrome and can take oral medication might be suitable candidates for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that lower doses of prednisone may be as safe as usual doses for treating relapses of childhood nephrotic syndrome. In earlier studies, most children on a reduced-dose plan did well after a year of treatment, suggesting that lower doses might be safe for children.

Prednisone is a common treatment for nephrotic syndrome, and the study aims to determine if using less is still effective and safe. Evidence so far suggests that lower doses could maintain safety while possibly reducing the side effects associated with higher doses. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about the reduced-dose steroid protocol for nephrotic syndrome because it aims to minimize side effects while maintaining effectiveness. Unlike the standard treatment, which uses higher doses of steroids like prednisone, this approach leverages a lower daily dose, reducing potential risks such as weight gain and bone density loss. By using less medication while still achieving remission, this protocol could offer a safer, more sustainable option for patients managing this condition.

What evidence suggests that reduced-dose steroids could be effective for nephrotic syndrome?

This trial will compare reduced-dose steroids with standard-dose steroids for treating nephrotic syndrome. Research has shown that a lower dose of prednisone, which participants in the reduced-dose arm of this trial may receive, can be as effective as a higher dose in helping children with steroid-sensitive nephrotic syndrome recover. Studies have found that even with a smaller amount of steroids, relapses can still be managed effectively. Additionally, taking prednisone every other day proves more effective than occasional dosing for children who frequently experience relapses. These findings suggest that a lower dose of steroids could be a viable option for treating this condition, potentially reducing side effects while maintaining effectiveness. ^{1 2 3 4 6}

Who Is on the Research Team?

Rulan Parekh, MD MS

Principal Investigator

The Hospital for Sick Children

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. ajkd.org ajkd.org/article/S0272-6386(22)00742-9/fulltext

Prednisone for Steroid-Sensitive Nephrotic Syndrome Alternate-day prednisone is more effective than intermittent prednisone in frequently relapsing nephrotic syndrome.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/31728673/

Lower prednisone dosing for steroid-sensitive nephrotic ... Treatment with a lower steroid dose may be as effective as the standard dose in achieving remission of steroid sensitive nephrotic syndrome ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06635720?term=AREA%5BConditionSearch%5D(%22Nephrosis%22)&rank=7

REduced-dose Steroid PrOtocol for Childhood Nephrotic ... 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 ...

4. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/584336

REduced-dose Steroid PrOtocol for Childhood Nephrotic ... This study will compare two steroid dosing protocols for the treatment of childhood nephrotic syndrome relapses. The medications used for both ...

5. frontiersin.org frontiersin.org/journals/nephrology/articles/10.3389/fneph.2024.1426156/pdf

Relapse treatment with low-dose steroids in ... Methods: The objective of this study was to analyze whether a low dose of steroids (LDS) is effective for the treatment of MCD relapses.

6. sciencedirect.com sciencedirect.com/science/article/pii/S2468024925005248

Randomized Controlled Trial on the Efficacy of Reduced ... Three-fourths of the patients treated with the low-dose regimen responded by the end of 1 year after initiating therapy.