Nephrotic Syndrome in ChildrenPhase 3RESPONSENCT06635720
REduced-dose Steroid PrOtocol for Childhood Nephrotic SyndromE (RESPONSE)
A Phase 3 study testing Prednisone in people with Nephrotic Syndrome in Children and Nephrotic Syndrome, Minimal Change, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Nephrotic Syndrome in Children, Nephrotic Syndrome, Minimal Change, Nephrotic Syndrome
- What is being tested?
- Prednisone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteCanada
- Study dates
- 1 Nov 2024to 28 Feb 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Provide informed consent ± assent
- Participant age 1-18 years
- 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)
- 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])
- Ability to take oral medication and willingness to adhere to either study prednisone regimen
- Ability and willingness to adhere to home urine and symptom monitoring during the initial two-week period after assigned treatment initiation
- Have not been previously included in the RESPONSE trial
- Participant located in Ontario, Canada at the time of study enrolment
Who cannot join
- Prednisone treatment (at any dose) for the active relapse episode for \>2-days prior to study enrolment
- Relapse episode within the past 6-weeks (i.e., date of relapse onset within 6-weeks prior to date of enrolment)
- Current receipt of high-dose maintenance prednisone therapy (dose \>0.6mg/kg on alternate days or \>0.3mg/kg daily)
- 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)
- Congenital or monogenic cause of nephrotic syndrome (defined as age at diagnosis \<1-year or known/suspected monogenic cause of nephrotic syndrome)
- 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)
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- Presence of moderate-to-severe peripheral edema (grade 3+; indentation depth ≥5mm and rebound time \>15 seconds)
- Hospitalization since the onset of the active relapse episode
- Acute kidney injury (KDIGO stage ≥1) since the onset of the active relapse episode
- Active or prior known or suspected venous thromboembolism during a relapse episode
- Active pregnancy or lactation
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: REduced-dose Steroid PrOtocol for Childhood Nephrotic SyndromE (RESPONSE): a Pilot Open-label Randomized, Controlled 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.
Read the detailed description
Study design:
This is a pilot study for a planned multi-center, Bayesian adaptive, non-inferiority RCT (Figure 1). This planned multi-center RCT will require additional funding, which we will apply for if feasibility is shown by this pilot. This pilot is a single-center (SickKids) open-label RCT comparing reduced vs. standard-dose steroids to treat nephrotic syndrome relapses.
Study population:
We will include children (1-18 years) from Ontario, Canada that are diagnosed with idiopathic SSNS and present in relapse (≥3+ dipstick protein or protein:creatinine ratio ≥200mg /mmol for ≥3 consecutive days). We will exclude children that have received \>2 days of standard-dose prednisone; are on maintenance high-dose prednisone (\>0.3mg/kg per day or \>0.6mg/kg alternate days); have relapsed within the past 6 weeks; have grade 3+ peripheral edema (i.e., moderate-severe); are hospitalized; have stage 2+ acute kidney injury; or have thromboembolism. Children receiving other steroid-sparing immunosuppressives are eligible, but target drug levels will remain constant until the 2-week visit. No additional laboratory or imaging tests are needed, to maximize recruitment.
Interventions:
Reduced-dose steroids (intervention): oral prednisone 30mg/m2 (1mg/kg; max 40mg) daily until remission, then 20mg/m2 (0.66mg/kg; max 25mg) on alternate days for four weeks.
Standard-dose steroids (control): oral prednisone 60mg/m2 (2mg/kg; max 60mg) daily until remission, then 40mg/m2 (1.5mg/kg; max 50mg) on alternate days for four weeks.
Randomization 1:1 using permuted blocks. If a participant does not achieve remission by 2-weeks, develops symptomatic edema, stage 2+ acute kidney injury, or thromboembolism, they will be escalated to standard-dose steroids.
Outcomes:
The primary outcome is recruitment rate (number enrolled per month), since it is the greatest anticipated barrier to RCT feasibility. Other feasibility, tolerability, and safety outcomes are listed in Table 1. The pilot study will test case report forms, data management, quality control, and training systems. We will estimate treatment effect for the full-scale RCT's primary outcome (complete remission without steroid dose escalation by 2-weeks), to re-estimate sample size and evaluate full-scale RCT feasibility.
Sample size:
For this pilot study, we will recruit 50 children (25 per arm), which is 15% of the estimated sample size of the full-scale RCT. Sample size for this pilot must be adequate to assess RCT feasibility.
- Conditions
- Nephrotic Syndrome in Children, Nephrotic Syndrome, Minimal Change, Nephrotic Syndrome, Nephrotic Syndrome，Idiopathic
- Being tested
- Prednisone
- Keywords
- Nephrotic syndrome, Idiopathic nephrotic syndrome, Childhood nephrotic syndrome, Minimal change disease, Focal segmental glomerulosclerosis
- Medical subject headings
- Nephrosis, Lipoid, Nephrotic Syndrome, Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Also called Prednisolone
This study will compare two steroid dosing protocols for the treatment of childhood nephrotic syndrome relapses. The medications used for both intervention and control arms are oral prednisone 5mg tablets (DIN: 00312770) for children able to swallow or use crushed tablets, or oral prednisolone sodium phosphate 5mg base/5mL liquid (DIN: 02245532). Participants hospitalized after study enrollment will be permitted to switch to intravenous methylprednisolone sodium succinate (DIN: 02367955) at the same dose without being considered to have violated their treatment assignment.
| Study group | Role | What they receive |
|---|---|---|
| Reduced-dose steroids | Experimental | 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. Receives: Drug: Prednisone |
| Standard-dose steroids | Active comparator | 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. Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Study recruitment rate
Number of participants enrolled per study month
Measured: 1 year
Secondary outcomes (10)
Treatment effect - treatment failure
Failure to achieve complete remission or steroid dose escalation by 2-weeks
Measured: From enrolment to 2-week study visit
Number of eligible participants
Number (%) of screen eligible participants
Measured: 1 year
Number of participants that initiate assigned treatment
Number (%) of participants that initiate assigned treatment
Measured: 1 year
Participant drop-out rate
Number (%) of study participants that drop out
Measured: 1 year
Treatment preference
Treatment preference ratings
Measured: 1 year
Hospitalizations
Number (%) of participants that are hospitalized
Measured: From enrolment to 1 year
Cumulative steroid dose
Cumulative steroid dose (mg/kg) prescribed
Measured: From enrolment to 1 year
Adverse events
Number (%) of participants that experience adverse events and serious adverse events
Measured: From enrolment to 1 year
Treatment adherence
Number (%) of participants that are adherent to assigned treatment and urine testing
Measured: From enrolment to 1 year
Number of participants that complete 2-week study visit
Number (%) of participants that complete 2-week study visit
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2024
- Study start1 Nov 2024
- Primary completion16 Feb 2026
- Study completion28 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nephrotic Syndrome in ChildrenStudies of the same condition, open and under way.
- More trials run by The Hospital for Sick ChildrenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rulan Parekh, The Hospital for Sick Children
- Investigators
- Rulan Parekh, MD MS, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06635720
- Sponsor's protocol ID
- 1000081679
- Acronym
- RESPONSE
- Status last verified
- Apr 2026
- First submitted
- 30 Sep 2024
- Last updated
- 11 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- REduced-dose Steroid PrOtocol for Childhood Nephrotic SyndromE (RESPONSE): a Pilot Open-label Randomized, Controlled Trial
- ClinicalTrials.gov ID
- NCT06635720
- Acronym / sponsor ID
- RESPONSE / 1000081679
- Conditions
- Nephrotic Syndrome in Children, Nephrotic Syndrome, Minimal Change, Nephrotic Syndrome, Nephrotic Syndrome，Idiopathic
- Keywords
- Nephrotic syndrome, Idiopathic nephrotic syndrome, Childhood nephrotic syndrome, Minimal change disease, Focal segmental glomerulosclerosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- 1 Nov 2024 (actual)
- Primary completion
- 16 Feb 2026 (actual)
- Study completion
- 28 Feb 2027 (estimated)
- First posted
- 10 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study design:
This is a pilot study for a planned multi-center, Bayesian adaptive, non-inferiority RCT (Figure 1). This planned multi-center RCT will require additional funding, which we will apply for if feasibility is shown by this pilot. This pilot is a single-center (SickKids) open-label RCT comparing reduced vs. standard-dose steroids to treat nephrotic syndrome relapses.
Study population:
We will include children (1-18 years) from Ontario, Canada that are diagnosed with idiopathic SSNS and present in relapse (≥3+ dipstick protein or protein:creatinine ratio ≥200mg /mmol for ≥3 consecutive days). We will exclude children that have received \>2 days of standard-dose prednisone; are on maintenance high-dose prednisone (\>0.3mg/kg per day or \>0.6mg/kg alternate days); have relapsed within the past 6 weeks; have grade 3+ peripheral edema (i.e., moderate-severe); are hospitalized; have stage 2+ acute kidney injury; or have thromboembolism. Children receiving other steroid-sparing immunosuppressives are eligible, but target drug levels will remain constant until the 2-week visit. No additional laboratory or imaging tests are needed, to maximize recruitment.
Interventions:
Reduced-dose steroids (intervention): oral prednisone 30mg/m2 (1mg/kg; max 40mg) daily until remission, then 20mg/m2 (0.66mg/kg; max 25mg) on alternate days for four weeks.
Standard-dose steroids (control): oral prednisone 60mg/m2 (2mg/kg; max 60mg) daily until remission, then 40mg/m2 (1.5mg/kg; max 50mg) on alternate days for four weeks.
Randomization 1:1 using permuted blocks. If a participant does not achieve remission by 2-weeks, develops symptomatic edema, stage 2+ acute kidney injury, or thromboembolism, they will be escalated to standard-dose steroids.
Outcomes:
The primary outcome is recruitment rate (number enrolled per month), since it is the greatest anticipated barrier to RCT feasibility. Other feasibility, tolerability, and safety outcomes are listed in Table 1. The pilot study will test case report forms, data management, quality control, and training systems. We will estimate treatment effect for the full-scale RCT's primary outcome (complete remission without steroid dose escalation by 2-weeks), to re-estimate sample size and evaluate full-scale RCT feasibility.
Sample size:
For this pilot study, we will recruit 50 children (25 per arm), which is 15% of the estimated sample size of the full-scale RCT. Sample size for this pilot must be adequate to assess RCT feasibility.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Reduced-dose steroids | Experimental | Drug: PrednisoneReduced-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. |
| Standard-dose steroids | Active comparator | Drug: PrednisoneStandard-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. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Provide informed consent ± assent
- Participant age 1-18 years
- 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)
- 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])
- Ability to take oral medication and willingness to adhere to either study prednisone regimen
Key exclusion criteria (12 listed)
- Prednisone treatment (at any dose) for the active relapse episode for \>2-days prior to study enrolment
- Relapse episode within the past 6-weeks (i.e., date of relapse onset within 6-weeks prior to date of enrolment)
- Current receipt of high-dose maintenance prednisone therapy (dose \>0.6mg/kg on alternate days or \>0.3mg/kg daily)
- 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)
- Congenital or monogenic cause of nephrotic syndrome (defined as age at diagnosis \<1-year or known/suspected monogenic cause of nephrotic syndrome)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PrednisoneAlso: PrednisoloneThis study will compare two steroid dosing protocols for the treatment of childhood nephrotic syndrome relapses. The medications used for both intervention and control arms are oral prednisone 5mg tablets (DIN: 00312770) for children able to swallow or use crushed tablets, or oral prednisolone sodium phosphate 5mg base/5mL liquid (DIN: 02245532). Participants hospitalized after study enrollment will be permitted to switch to intravenous methylprednisolone sodium succinate (DIN: 02367955) at the same dose without being considered to have violated their treatment assignment. | Drug | Reduced-dose steroids, Standard-dose steroids |
Primary outcome
Study recruitment rate
Time frame: 1 year
Number of participants enrolled per study month
10 secondary outcomes
Treatment effect - treatment failure
Time frame: From enrolment to 2-week study visit
Number of eligible participants
Time frame: 1 year
Number of participants that initiate assigned treatment
Time frame: 1 year
Participant drop-out rate
Time frame: 1 year
Treatment preference
Time frame: 1 year
Hospitalizations
Time frame: From enrolment to 1 year
Cumulative steroid dose
Time frame: From enrolment to 1 year
Adverse events
Time frame: From enrolment to 1 year
Treatment adherence
Time frame: From enrolment to 1 year
Number of participants that complete 2-week study visit
Time frame: 2 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rulan Parekh, MD MS principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone in people with Nephrotic Syndrome in Children and Nephrotic Syndrome, Minimal Change, run by The Hospital for Sick Children at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Study recruitment rate (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 May 2026.