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

NCT03387176 | Completed

• 1 other identifier: 17-01307
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (2)

• Change in disease activity measured by relapse rate

  Response is defined as a ≥50%decrease in relapse rate

  12 Months

• Change in disease activity measured by change in dosage of corticosteroids and immunosuppressive medications

  reduction by ≥1 drug in exposure to immunosuppressive medications in response to the GFD

  12 Months

Study Arms (2)

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

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

Interventions

Implementation of a gluten-free diet OTHER

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

zonulin >17.5 ng/ml; zonulin ≤17.5 ng/ml

Eligibility Criteria

• Age: 9 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children age 9 months to 18 years of age with steroid sensitive Nephrotic Syndrome

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

New York University School of Medicine

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Nephrotic Syndrome

Condition Hierarchy (Ancestors)

Nephrosis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Officials

• Howard Trachtman, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2017
• First Posted: December 29, 2017
• Study Start: December 1, 2017
• Primary Completion: June 14, 2021
• Study Completion: June 14, 2021
• Last Updated: June 22, 2021

Record last verified: 2021-06

Locations

US (1)