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.
Brief Title
Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker
Completion Date
Completion Date Type
Actual
Conditions
Nephrotic Syndrome
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
* 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
Inclusion 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.
* 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.
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
18 Years
Minimum Age
9 Months
NCT Id
NCT03387176
Org Class
Other
Org Full Name
NYU Langone Health
Org Study Id
17-01307
Overall Status
Completed
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker
Primary Outcomes
Outcome Description
Response is defined as a ≥50%decrease in relapse rate
Outcome Measure
Change in disease activity measured by relapse rate
Outcome Time Frame
12 Months
Outcome Description
reduction by ≥1 drug in exposure to immunosuppressive medications in response to the GFD
Outcome Measure
Change in disease activity measured by change in dosage of corticosteroids and immunosuppressive medications
Outcome Time Frame
12 Months
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Study Population
Children age 9 months to 18 years of age with steroid sensitive Nephrotic Syndrome
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
18
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Frederick Kaskel
Investigator Email
FKASKEL@montefiore.org
Investigator Phone
718-655-1120