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

Sponsor
NYU Langone Health (Other)
Overall Status
Completed
CT.gov ID
NCT03387176
Collaborator
(none)
13
1
42.4
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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
  • Other: Implementation of a gluten-free diet

Study Design

Study Type:
Observational
Actual Enrollment :
13 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker
Actual Study Start Date :
Dec 1, 2017
Actual Primary Completion Date :
Jun 14, 2021
Actual Study Completion Date :
Jun 14, 2021

Arms and Interventions

Arm Intervention/Treatment
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

  1. Change in disease activity measured by relapse rate [12 Months]

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

  2. 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

Ages Eligible for Study:
9 Months to 18 Years
Sexes Eligible for Study:
All
Accepts 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

Contacts and Locations

Locations

Site City State Country Postal Code
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.
Responsible Party:
NYU Langone Health
ClinicalTrials.gov Identifier:
NCT03387176
Other Study ID Numbers:
  • 17-01307
First Posted:
Dec 29, 2017
Last Update Posted:
Jun 22, 2021
Last Verified:
Jun 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Jun 22, 2021