The Nephrotic Syndrome Study Network (NEPTUNE)

Kidney Disease Clinical Trial

Official title: The Nephrotic Syndrome Study Network (NEPTUNE)

Status Recruiting

Clinical Trial Summary

Background:

• The Nephrotic Syndrome Study Network (NEPTUNE) is a network of multidisciplinary researchers who are investigating why kidney disease happens. NEPTUNE researchers will collect kidney tissue and other samples (for example, blood and urine) from individuals who are scheduled to have kidney biopsies to determine the cause of protein in the urine (only one kidney biopsy is necessary).

Objectives:

• To collect kidney tissue, other samples, and data /information for continuing research into kidney diseases.

Eligibility:

• Individuals at least 18 years of age who need to have a kidney biopsy to determine the cause of protein in the urine, do not have a systemic disease that is the cause of the their kidney disease, and have not received specific treatment for kidney disease.

Design:

• This study involves a screening and baseline visit and additional followup visits after the kidney biopsy.

• Participants will be screened with a medical history and physical examination, as well as blood and urine samples and collection of fingernail clippings. Participants will also complete questionnaires about their history of kidney problems.

• During the kidney biopsy, performed at the NIH Clinical Center, researchers will take an additional tissue sample for research.

• Participants will return for followup visits at NIH every 4 months in the first year, and every 6 months in the second through fifth years after the biopsy. Additional blood and urine samples will be collected at each visit, and fingernail clippings will also be collected annually by the study researchers.

• Treatment for kidney disease will not be provided as part of this protocol and instead will generally be provided by the patient s own physician.

Compensation:

Subjects received compensation for each visit to the NIH Clinical Center.

Clinical Trial Description

Idiopathic Nephrotic Syndrome (NS) is a rare disease syndrome responsible for approximately 12% of all causes of end-stage kidney disease (ESRD) and up to 20% of ESRD in children. Treatment strategies for Focal and Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD) and Membranous Nephropathy (MN), the major causes of NS, include high dose prolonged steroid therapy, cyclophosphamide, cyclosporine A, tacrolimus, mycophenolate mofetil and other immunosuppressive agents, which all carry significant side effects. Failure to obtain remission using the current treatment approaches frequently results in progression to ESRD with its associated costs, morbidities, and mortality (1). In the North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) registry, half of the pediatric patients with Steroid Resistant Nephrotic Syndrome required renal replacement therapy within two years of being enrolled in the disease registry. FSGS also has a high recurrence rate following kidney transplantation (30-40%) and is the most common recurrent disease leading to allograft loss. The prevailing classification of Nephrotic Syndrome categorizes patients into FSGS, MCD, and MN, if in the absence of other underlying causes, glomerular histology shows a specific histological pattern. This classification does not adequately predict the heterogeneous natural history of patients with FSGS, MCD, and MN. Major advances in understanding the pathogenesis of FSGS and MCD have come over the last ten years from the identification of several mutated genes responsible for causing Steroid Resistant Nephrotic Syndrome (SRNS) presenting with FSGS or MCD histopathology in humans and model organisms. These functionally distinct genetic disorders can present with indistinguishable FSGS lesions on histology confirming the presence of heterogeneous pathogenic mechanisms under the current histological diagnoses . The limited understanding of FSGS, MCD, and MN biology in humans has necessitated a descriptive classification system in which heterogeneous disorders are grouped together. This invariably consigns these heterogeneous patients to the same therapeutic approaches, which use blunt immunosuppressive drugs that lack a clear biological basis, are often not beneficial, and are complicated by significant toxicity. The foregoing shortcomings make a strong case that concerted and innovative investigational strategies combining basic science, translational, and clinical methods should be employed to study FSGS, MCD, and MN. It is for these reasons that the Nephrotic Syndrome Study Network is established to conduct clinical and translational research in patients with FSGS/MCD and MN. ;

Study Design

Time Perspective: Prospective

Related Conditions & MeSH terms

• Glomerular Disease
• Glomerulonephritis
• Glomerulosclerosis, Focal
• Glomerulosclerosis, Focal Segmental
• Kidney Disease
• Kidney Diseases
• Nephrosis
• Nephrotic Syndrome
• Syndrome

• NCT number: NCT01240564
• Study type: Observational
• Source: National Institutes of Health Clinical Center (CC)
• Contact: Anaida Widell
• Phone: (301) 451-9946
• Email: awidell@cc.nih.gov
• Status: Recruiting
• Phase: N/A
• Start date: October 2010