Steroid Sensitive Nephrotic Syndrome in Children

NCT ID: NCT03878914

Last Updated: 2022-05-02

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-08-06
• Study Completion Date: 2021-10-31

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.

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.

Conditions

Nephrotic Syndrome in Children

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Corticosteroids

Intervention Type: DRUG

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.

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.

Group Type: ACTIVE_COMPARATOR

Corticosteroids

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital of Fudan University

OTHER

Sponsor Role: collaborator

Wayne State University

OTHER

Sponsor Role: lead

Responsible Party

Tej Mattoo, MD

Professor of Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Tej Mattoo, MD

Role: PRINCIPAL_INVESTIGATOR

Wayne State University

Locations

Wayne Pediatrics

Detroit, Michigan, United States

Children's Hospital of Fudan University

Shanghai, , China

Countries

United States; China

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Other Identifiers

1901002013

Identifier Type: -

Identifier Source: org_study_id