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Nephrotic Syndrome in ChildrenPhase 4NCT03878914

Steroid Sensitive Nephrotic Syndrome in Children

A Phase 4 study testing Corticosteroids in people with Nephrotic Syndrome in Children, run by Wayne State University at 2 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in children and corticosteroid therapy is its most effective treatment. The total duration of treatment ranges anywhere from two to six months, generally about 3 months. Read more about this study

Who is it for?
Ages 1 year to 18 years
Conditions the study is for
Nephrotic Syndrome in Children
What is being tested?
Corticosteroids
Could I receive a placebo?
No, everyone receives an active treatment
How many people
34 participants
Where
2 sitesin 2 countries
Study dates
6 Aug 2019to 31 Oct 2021
Phase
Phase 4
Are there results?
None posted in the registry

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

  • Age 1 to <19 years
  • Newly diagnosed INS
  • Patient in remission with steroids
  • Written informed consent/Assent for the study OR as required by the local IRB

Who cannot join

  • Age < 1 year or ≥ 19 years
  • Uncertainty about patient/parent adherence.
  • Abnormal serum creatinine for patient age
  • Steroid resistant nephrotic syndrome
  • Any co-morbid condition that might require modification in treatment with steroids.

Trial locations and contacts

2 sites in 2 countries.

2 sites in 2 countries

United States1 site
  • Wayne Pediatrics

    Detroit, Michigan, 48201

Other trials in United States

China1 site
  • Children's Hospital of Fudan University

    Shanghai, 201102

Other trials in China

About this study

Official title: A Multinational Prospective Study on the Duration of Steroid Therapy in Steroid Sensitive Nephrotic Syndrome

Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in children and corticosteroid therapy is its most effective treatment. The total duration of treatment ranges anywhere from two to six months, generally about 3 months. The main objective of our study is to test the feasibility of a shorter total duration (two months) of corticosteroid therapy in patients who show a quicker treatment response to the initial treatment.

Read the detailed description

Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in children and corticosteroid therapy is its most effective treatment. The main objective of our prospective, open-label, observational clinical cohort study is to test the feasibility of a shorter duration of corticosteroid therapy in patients who show a quicker treatment response. We hypothesize that the clinical outcomes in children with time to remission of ≤10 days and treated with only 8 weeks of corticosteroid therapy will not be significantly different as compared to those with time to remission of >10 days and treated with ≥12 weeks of standard corticosteroid therapy. Our specific aims are as follows: First, we will evaluate the time to first relapse after 8-week corticosteroid therapy in quick responders in comparison to the standard treatment of ≥12 weeks in slow responders. Second, we will assess the frequency of relapses during one year follow-up after completion of 8-week corticosteroid therapy in quick responders in comparison to the standard treatment of ≥12 weeks in slow responders. To complete the study successfully during the funding period of two years and to increase the generalizability of its results, the study will recruit 66 patients at six study participating sites in five countries, including U.S., India, China, Egypt, and Qatar. The sites have been carefully selected on the basis of their reputation, patient volume, research experience, and PI's personal rapport with the site investigators. The proposed study is innovative because it seeks a paradigm shift from 'one-size-fits-all' to an entirely new concept of individualized treatment duration based on "time to remission" with initial corticosteroid therapy. The proposed study is the first precision medicine initiative in the management of INS. The project is significant because of the potential to improve public health by decreasing the side effects of prolonged corticosteroid administration in about half of the patients diagnosed with INS. Our long-term objective is to develop additional novel therapeutic strategies to optimize the use of corticosteroids in the management of initial episode and relapses in children with INS.

Being tested
Corticosteroids
Keywords
Nephrotic sydrome, Steroid sensitive nephrotic syndrome
Medical subject headings
Nephrotic Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CorticosteroidsDrug

    Patients will be divided into two groups based on time to remission with initial standard dose of corticosteroids. Patients who respond within 10 days (Group A) will receive a total of 8 weeks of corticosteroid therapy whereas those who respond between 10 days to 28 days (Group B) will receive ≥12 weeks ((maximum of 16 weeks) of corticosteroid therapy.

Study groups in NCT03878914
Study groupRoleWhat they receive
Quick responders (Group A)ExperimentalPatients will be divided into two groups based on time to remission with initial standard dose of corticosteroids. Patients who respond within 10 days (Group A) will receive a total of 8 weeks of corticosteroid therapy whereas those who respond between 10 days to 28 days (Group B) will receive ≥12 weeks ((maximum of 16 weeks) of corticosteroid therapy. CORTICOSTEROID THERAPY FOR INITIAL EPISODE Group A (Total duration of therapy 8 weeks) * 60mg/m2/day or 2mg/kg/day (maximum 60mg) day for 2 weeks * 40mg/m2 or 1.5mg (maximum 40mg) every other day for 2 weeks. * Wean off in 4 weeks CORTICOSTEROID THERAPY FOR A RELAPSE * 60mg/m2/day or 2mg/kg/day (maximum 60mg) until remission * 40mg/m2 or 1.5mg (maximum 40mg) every other day for one week followed by continued weaning until discontinued in 6-8 weeks.
Receives: Drug: Corticosteroids
Slow responders (Group B)Active comparatorCORTICOSTEROID THERAPY FOR INITIAL EPISODE Group B: (Total duration of therapy ≥ 12 weeks) * 60mg/m2/day or 2mg/kg/day (maximum 60mg) day for 4 weeks * 40mg/m2 or 1.5mg (maximum 40mg) every other day for 4 weeks. * Wean off in 4-6 weeks CORTICOSTEROID THERAPY FOR A RELAPSE * 60mg/m2/day or 2mg/kg/day (maximum 60mg) until remission * 40mg/m2 or 1.5mg (maximum 40mg) every other day for one week followed by continued weaning until discontinued in 6-8 weeks.
Receives: Drug: Corticosteroids

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Time to first relapse.

    The study will evaluate the time in weeks for patients to relapse after completion of initial treatment and if there is any difference between Group A and Group B.

    Measured: 60-64 weeks

Secondary outcomes (8)
  1. Number of relapses

    Number of relapses per patient after completion of treatment.

    Measured: 52 weeks

  2. Number of frequent relapses

    Number of frequent relapses per patient after completion of treatment.

    Measured: 52 weeks

  3. Number of patients with steroid dependence

    Number of patients who show steroid dependence after completion of treatment.

    Measured: 52 weeks

  4. Number of patients with late steroid resistance

    Number of patients who show late steroid resistance after completion of treatment.

    Measured: 52 weeks

  5. Cumulative steroid dose in two groups

    The total dose of corticosteroids received in Group A patients versus Group B patients

    Measured: 60 to 64 weeks

  6. Number of episodes of upper respiratory infection (URI) and other infections.

    The total number of URI or other infections in Group A patients versus Group B patients after completion of treatment.

    Measured: 52-weeks

  7. Weight profile

    Weight profile in Group A patients versus Group B patients

    Measured: 60-64 weeks

  8. Height Profile

    Height profile in Group A patients versus Group B patients

    Measured: 60-64 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Mar 2019
  2. Study start6 Aug 2019
  3. Primary completion31 Oct 2021
  4. Study completion31 Oct 2021

Why it stopped: The study was terminated at all but one site because of the pandemic

Resources

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 4
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Patients will be divided into two groups based on time to remission with initial standard dose of corticosteroids. Patients who respond within 10 days (Group A) will receive a total of 8 weeks of corticosteroid therapy whereas those who respond between 10 days to 28 days (Group B) will receive ≥12 weeks ((maximum of 16 weeks) of corticosteroid therapy.
Masking
None (open label)
Enrolment
34 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03878914
Sponsor's protocol ID
1901002013
Status last verified
Apr 2022
First submitted
12 Mar 2019
Last updated
2 May 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Depending on available resources, we may share data without any patient identifier with the study site investigators as well as others who might be interested.

Publications

  • Hahn D, Hodson EM, Willis NS, Craig JC. Corticosteroid therapy for nephrotic syndrome in children. Cochrane Database Syst Rev. 2015 Mar 18;2015(3):CD001533. doi: 10.1002/14651858.CD001533.pub5.

    PubMed 25785660
  • Hodson EM, Willis NS, Craig JC. Corticosteroid therapy for nephrotic syndrome in children. Cochrane Database Syst Rev. 2007 Oct 17;(4):CD001533. doi: 10.1002/14651858.CD001533.pub4.

    PubMed 17943754
  • Yoshikawa N, Nakanishi K, Sako M, Oba MS, Mori R, Ota E, Ishikura K, Hataya H, Honda M, Ito S, Shima Y, Kaito H, Nozu K, Nakamura H, Igarashi T, Ohashi Y, Iijima K; Japanese Study Group of Kidney Disease in Children. A multicenter randomized trial indicates initial prednisolone treatment for childhood nephrotic syndrome for two months is not inferior to six-month treatment. Kidney Int. 2015 Jan;87(1):225-32. doi: 10.1038/ki.2014.260. Epub 2014 Jul 23.

    PubMed 25054775
  • Sinha A, Saha A, Kumar M, Sharma S, Afzal K, Mehta A, Kalaivani M, Hari P, Bagga A. Extending initial prednisolone treatment in a randomized control trial from 3 to 6 months did not significantly influence the course of illness in children with steroid-sensitive nephrotic syndrome. Kidney Int. 2015 Jan;87(1):217-24. doi: 10.1038/ki.2014.240. Epub 2014 Jul 16.

    PubMed 25029428
  • Lombel RM, Hodson EM, Gipson DS; Kidney Disease: Improving Global Outcomes. Treatment of steroid-resistant nephrotic syndrome in children: new guidelines from KDIGO. Pediatr Nephrol. 2013 Mar;28(3):409-14. doi: 10.1007/s00467-012-2304-8. Epub 2012 Oct 5.

    PubMed 23052648
  • Vivarelli M, Moscaritolo E, Tsalkidis A, Massella L, Emma F. Time for initial response to steroids is a major prognostic factor in idiopathic nephrotic syndrome. J Pediatr. 2010 Jun;156(6):965-971. doi: 10.1016/j.jpeds.2009.12.020. Epub 2010 Mar 10.

    PubMed 20223477
  • Constantinescu AR, Shah HB, Foote EF, Weiss LS. Predicting first-year relapses in children with nephrotic syndrome. Pediatrics. 2000 Mar;105(3 Pt 1):492-5. doi: 10.1542/peds.105.3.492.

    PubMed 10699098
  • Letavernier B, Letavernier E, Leroy S, Baudet-Bonneville V, Bensman A, Ulinski T. Prediction of high-degree steroid dependency in pediatric idiopathic nephrotic syndrome. Pediatr Nephrol. 2008 Dec;23(12):2221-6. doi: 10.1007/s00467-008-0914-y. Epub 2008 Jul 11.

    PubMed 18618150
  • Srivastava RN, Mayekar G, Anand R, Choudhry VP, Ghai OP, Tandon HD. Nephrotic syndrome in indian children. Arch Dis Child. 1975 Aug;50(8):626-30. doi: 10.1136/adc.50.8.626.

    PubMed 973
  • Elzouki AY, Amin F, Jaiswal OP. Primary nephrotic syndrome in Arab children. Arch Dis Child. 1984 Mar;59(3):253-5. doi: 10.1136/adc.59.3.253.

    PubMed 6712274
  • Sharples PM, Poulton J, White RH. Steroid responsive nephrotic syndrome is more common in Asians. Arch Dis Child. 1985 Nov;60(11):1014-7. doi: 10.1136/adc.60.11.1014.

    PubMed 4073933
  • Banh THM, Hussain-Shamsy N, Patel V, Vasilevska-Ristovska J, Borges K, Sibbald C, Lipszyc D, Brooke J, Geary D, Langlois V, Reddon M, Pearl R, Levin L, Piekut M, Licht CPB, Radhakrishnan S, Aitken-Menezes K, Harvey E, Hebert D, Piscione TD, Parekh RS. Ethnic Differences in Incidence and Outcomes of Childhood Nephrotic Syndrome. Clin J Am Soc Nephrol. 2016 Oct 7;11(10):1760-1768. doi: 10.2215/CJN.00380116. Epub 2016 Jul 21.

    PubMed 27445165
  • Mattoo TK, Mahmood MA, al-Harbi MS. Nephrotic syndrome in Saudi children clinicopathological study of 150 cases. Pediatr Nephrol. 1990 Sep;4(5):517-9. doi: 10.1007/BF00869837.

    PubMed 2242321
  • Bagga A, Hari P, Srivastava RN. Prolonged versus standard prednisolone therapy for initial episode of nephrotic syndrome. Pediatr Nephrol. 1999 Nov;13(9):824-7. doi: 10.1007/s004670050708.

    PubMed 10603129
  • Hiraoka M, Tsukahara H, Matsubara K, Tsurusawa M, Takeda N, Haruki S, Hayashi S, Ohta K, Momoi T, Ohshima Y, Suganuma N, Mayumi M; West Japan Cooperative Study Group of Kidney Disease in Children. A randomized study of two long-course prednisolone regimens for nephrotic syndrome in children. Am J Kidney Dis. 2003 Jun;41(6):1155-62. doi: 10.1016/s0272-6386(03)00346-9.

    PubMed 12776266

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Corticosteroids in people with Nephrotic Syndrome in Children, run by Wayne State University at 2 sites in 2 countries.

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 4 inclusion and 5 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: stopped early, as of the sponsor's last update on 2 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States, China. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: The study was terminated at all but one site because of the pandemic). Results are not available in the source record.

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?

Time to first relapse. (time frame: 60-64 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03878914, which also lists 16 linked publications.

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 2 May 2022.

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