Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker
Study Details
Study Description
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.
Condition or Disease | Intervention/Treatment | Phase |
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Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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zonulin ≤17.5 ng/ml Pediatric patients with difficult-to-manage nephrotic syndrome will be stratified based on the plasma zonulin concentration into two groups |
Other: Implementation of a gluten-free diet
Patients will be placed on gluten free diet for 9-12 months.
|
zonulin >17.5 ng/ml Pediatric patients with difficult-to-manage nephrotic syndrome will be stratified based on the plasma zonulin concentration into two groups |
Other: Implementation of a gluten-free diet
Patients will be placed on gluten free diet for 9-12 months.
|
Outcome Measures
Primary Outcome Measures
- 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
Eligibility Criteria
Criteria
Inclusion Criteria:
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Steroid sensitive NS: complete remission of proteinuria in response to administration of a standard course of corticosteroids
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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:
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Any patient diagnosed with nephrotic syndrome that is not considered steroid sensitive or frequently relapsing
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Pre-existing celiac disease or gastro-intestinal disorder that precludes use of a GFD
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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1 | New York University School of Medicine | New York | New York | United States | 10016 |
Sponsors and Collaborators
- NYU Langone Health
Investigators
- Principal Investigator: Howard Trachtman, MD, NYU Langone Health
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 17-01307