Nephrotic Syndrome
Conditions
Brief summary
Elevated plasma zonulin levels, which are supportive of a diagnosis of CD (celiac disease) in children with gastrointestinal symptoms, may indicate patients with difficult-to-manage NS who will benefit from initiation of a GFD (gluten free diet). This pilot study will determine whether high plasma zonulin levels can be used as a screening tool to identify patients with NS (nephrotic syndrome) who are likely to demonstrate a beneficial response to a GFD. It will provide important information about the feasibility of testing the efficacy of a GFD for this condition and assist in the design and sample size calculation for a definitive trial to test the beneficial effect of this dietary intervention. Although NS is a rare condition in childhood, it is a chronic disease that can lead to short- and long-term disability especially in those with difficult-to-manage disease. There is an urgent need to develop safe and effective new therapies in this subgroup. This project may indicate the utility of a common dietary modification, a GFD, to treat these patients. The growing medical use of and greater access to gluten-free food items underscore the feasibility and timeliness of this approach.
Interventions
OTHERImplementation of a gluten-free diet
Patients will be placed on gluten free diet for 9-12 months.
Sponsors
NYU Langone Health
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
9 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* Steroid sensitive NS: complete remission of proteinuria in response to administration of a standard course of corticosteroids * Difficult-to-manage NS: disease that cannot be controlled without incurring intolerable side effects from currently available immunosuppressive agents, namely corticosteroids, calcineurin inhibitors, mycophenolate mofetil, or rituximab. Patients with biopsy-proven MCD or FSGS will be eligible as long as they have steroid sensitive disease. However, a renal biopsy will not be required for enrollment into the trial.
Exclusion criteria
* Any patient diagnosed with nephrotic syndrome that is not considered steroid sensitive or frequently relapsing * Pre-existing celiac disease or gastro-intestinal disorder that precludes use of a GFD
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in disease activity measured by change in dosage of corticosteroids and immunosuppressive medications | 12 Months | reduction by ≥1 drug in exposure to immunosuppressive medications in response to the GFD |
| Change in disease activity measured by relapse rate | 12 Months | Response is defined as a ≥50%decrease in relapse rate |
Countries
United States
Outcome results
None listed