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

NCT ID: NCT03387176

Last Updated: 2021-06-22

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 13 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-12-01
• Study Completion Date: 2021-06-14

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.

Conditions

Nephrotic Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

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

Implementation of a gluten-free diet

Intervention Type: OTHER

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

Implementation of a gluten-free diet

Intervention Type: OTHER

Patients will be placed on gluten free diet for 9-12 months.

Interventions

Implementation of a gluten-free diet

Patients will be placed on gluten free diet for 9-12 months.

Intervention Type: OTHER

Eligibility Criteria

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

• Minimum Eligible Age: 9 Months
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NYU Langone Health

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Howard Trachtman, MD

Role: PRINCIPAL_INVESTIGATOR

NYU Langone Health

Locations

New York University School of Medicine

New York, New York, United States

Countries

United States

Other Identifiers

17-01307

Identifier Type: -

Identifier Source: org_study_id