Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker

Completed

• Conditions: Nephrotic Syndrome
• Interventions: Other: Implementation of a gluten-free diet

• Registration Number: NCT03387176
• Lead Sponsor: NYU Langone Health

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-12-01
• Study First Submitted: 2017-12-21
• Study First Submitted QC: 2017-12-21
• Study First Posted: 2017-12-29
• Status Verified: 2021-06
• Primary Completion: 2021-06-14
• Study Completion: 2021-06-14
• Last Update Submitted: 2021-06-21
• Last Update Posted: 2021-06-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

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.

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
zonulin >17.5 ng/ml	Implementation of a gluten-free diet	Pediatric patients with difficult-to-manage nephrotic syndrome will be stratified based on the plasma zonulin concentration into two groups
zonulin ≤17.5 ng/ml	Implementation of a gluten-free diet	Pediatric patients with difficult-to-manage nephrotic syndrome will be stratified based on the plasma zonulin concentration into two groups

Primary Outcome Measures

Name	Time	Method
Change in disease activity measured by relapse rate	12 Months	Response is defined as a ≥50%decrease in relapse rate
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

Trial Locations

Locations (1)

New York University School of Medicine; 🇺🇸 New York, New York, United States