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Steroid Sensitive Nephrotic Syndrome in Children

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ClinicalTrials.gov Identifier: NCT03878914
Recruitment Status : Not yet recruiting
First Posted : March 18, 2019
Last Update Posted : March 18, 2019
Sponsor:
Collaborators:
Children's Hospital of Fudan University
Mansoura University Children Hospital
Lady Hardinge Medical College
Sawai Mansingh Medical College
Sidra Medical and Research Center
Information provided by (Responsible Party):
Tej Mattoo, MD, Wayne State University

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Nephrotic Syndrome in Children Drug: Corticosteroids Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 66 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention 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)
Primary Purpose: Treatment
Official Title: A Multinational Prospective Study on the Duration of Steroid Therapy in Steroid Sensitive Nephrotic Syndrome
Estimated Study Start Date : May 2019
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : April 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Allergy Steroids

Arm Intervention/treatment
Experimental: Quick responders (Group A)

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.

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.
Drug: Corticosteroids
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.

Active Comparator: Slow responders (Group B)

CORTICOSTEROID 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.
Drug: Corticosteroids
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.




Primary Outcome Measures :
  1. Time to first relapse. [ Time Frame: 60-64 weeks ]
    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.


Secondary Outcome Measures :
  1. Number of relapses [ Time Frame: 52 weeks ]
    Number of relapses per patient after completion of treatment.

  2. Number of frequent relapses [ Time Frame: 52 weeks ]
    Number of frequent relapses per patient after completion of treatment.

  3. Number of patients with steroid dependence [ Time Frame: 52 weeks ]
    Number of patients who show steroid dependence after completion of treatment.

  4. Number of patients with late steroid resistance [ Time Frame: 52 weeks ]
    Number of patients who show late steroid resistance after completion of treatment.

  5. Cumulative steroid dose in two groups [ Time Frame: 60 to 64 weeks ]
    The total dose of corticosteroids received in Group A patients versus Group B patients

  6. Number of episodes of upper respiratory infection (URI) and other infections. [ Time Frame: 52-weeks ]
    The total number of URI or other infections in Group A patients versus Group B patients after completion of treatment.

  7. Weight profile [ Time Frame: 60-64 weeks ]
    Weight profile in Group A patients versus Group B patients

  8. Height Profile [ Time Frame: 60-64 weeks ]
    Height profile in Group A patients versus Group B patients



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03878914


Contacts
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Contact: Tej Mattoo, MD (313)745-5604 tmattoo@med.wayne.edu

Locations
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United States, Michigan
Children's Hospital of Michigan Not yet recruiting
Detroit, Michigan, United States, 48201
Principal Investigator: Tej Mattoo, MD         
China
Children's Hospital of Fudan University Not yet recruiting
Shanghai, China, 201102
Contact: Hong Xu, MD         
Principal Investigator: Hong Xu, MD         
Egypt
Mansoura University Children Hospital Not yet recruiting
Mansoura, Egypt, 35516
Contact: Ahmed El-Refaey, MD         
Principal Investigator: Ahmed El-Refaey, MD,PhD         
India
Sawai Man Singh Medical College Hospital Not yet recruiting
Jaipur, India, 302004
Contact: Amarjeet Mehta, MD         
Principal Investigator: Amarjeet Mehta, MD         
Lady Hardinge Medical College Not yet recruiting
New Delhi, India, 110001
Contact: Rachita Dhull, MD         
Principal Investigator: Rachita Dhull, MD         
Qatar
Sidra Medical and Research Center Not yet recruiting
Doha, Qatar, 26999
Contact: Abubakr Imam, MD         
Principal Investigator: Abubakr Imam, MD         
Sponsors and Collaborators
Wayne State University
Children's Hospital of Fudan University
Mansoura University Children Hospital
Lady Hardinge Medical College
Sawai Mansingh Medical College
Sidra Medical and Research Center
Investigators
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Principal Investigator: Tej Mattoo, MD Wayne State University

Publications:

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Responsible Party: Tej Mattoo, MD, Professor of Pediatrics, Wayne State University
ClinicalTrials.gov Identifier: NCT03878914     History of Changes
Other Study ID Numbers: 1901002013
First Posted: March 18, 2019    Key Record Dates
Last Update Posted: March 18, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: 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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Tej Mattoo, MD, Wayne State University:
Nephrotic sydrome
Steroid sensitive nephrotic syndrome

Additional relevant MeSH terms:
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Syndrome
Hypersensitivity
Nephrotic Syndrome
Nephrosis
Disease
Pathologic Processes
Immune System Diseases
Kidney Diseases
Urologic Diseases