Nephrotic Syndrome in Children
Conditions
Keywords
Nephrotic sydrome, Steroid sensitive nephrotic syndrome
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.
Interventions
DRUGCorticosteroids
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.
Sponsors
Children's Hospital of Fudan University
Wayne State University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
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.
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to first relapse. | 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
| Measure | Time frame | Description |
|---|---|---|
| Number of episodes of upper respiratory infection (URI) and other infections. | 52-weeks | The total number of URI or other infections in Group A patients versus Group B patients after completion of treatment. |
| Number of relapses | 52 weeks | Number of relapses per patient after completion of treatment. |
| Number of frequent relapses | 52 weeks | Number of frequent relapses per patient after completion of treatment. |
| Cumulative steroid dose in two groups | 60 to 64 weeks | The total dose of corticosteroids received in Group A patients versus Group B patients |
| Number of patients with late steroid resistance | 52 weeks | Number of patients who show late steroid resistance after completion of treatment. |
| Weight profile | 60-64 weeks | Weight profile in Group A patients versus Group B patients |
| Height Profile | 60-64 weeks | Height profile in Group A patients versus Group B patients |
| Number of patients with steroid dependence | 52 weeks | Number of patients who show steroid dependence after completion of treatment. |
Countries
China, United States
Outcome results
None listed