Nephrotic Syndrome Study Network
Study Design: Cohort
Conditions: Kidney Diseases, Nephrotic Syndrome, Renal Insufficiency, Chronic
Duration: 2010 – Present
# Recruitment Centers: 18
Treatment: None, observational only
Available Genotype Data: No
Image Summary: No
Transplant Type: None
Does it have dialysis patients: No
Clinical Trials URL: http://www.clinicaltrials.gov/show/NCT01209000
Pro non-inflammatory glomerular diseases, including minimal change disease (MCD), focal and segmental glomerulosclerosis (FSGS) and membranous nephropathy (MN), account for approximately 15% of prevalent end-stage renal disease (ESRD) cases in the United States. Development of successful therapeutic interventions is hindered by a limited knowledge of underlying glomerular disease mechanisms. In response to the need for research concerning these conditions, the Nephrotic Syndrome Study Network (NEPTUNE) was established to investigate the underlying disease mechanisms, elucidate pathogenesis, and identify therapeutic targets for clinical trials. The NEPTUNE consortium also aims to bank long-term observational data and corresponding biological specimens for future studies. NEPTUNE is part of the NIH Rare Disease Center Research Network (RDCRN).
The NEPTUNE cohort study, one of the projects initiated by the consortium, is a prospective, observational study that enrolls children and adults with FSGS, MCD, and MN. Information on demographics, clinical history, physical examination, as well as tissue samples from a renal biopsy visit, is collected at baseline. Questionnaires are given to assess quality of life and self-reported health. Participants are assigned into a specific study cohort (FSGS, MCD, MN) based on renal biopsy. After baseline assessment is complete, participants are followed over 30 months to collect data concerning health, quality of life, and outcomes. The primary outcome is a composite measure of change in urinary protein excretion and change in renal function. Secondary outcome measures include quality of life assessment, development of new-onset diabetes, malignancies, infections, thromboembolic events, hospitalization, acute kidney injury, and death. Molecular profiles and gene expression data will be linked to phenotypic, genetic, and histologic data for systems biology analysis.
This study is ongoing.
The NEPTUNE observational study was established to investigate the underlying disease mechanisms, elucidate pathogenesis, and identify therapeutic targets for clinical trials.
The primary outcome is a composite measure of change in urinary protein excretion and change in renal function, which is defined as remission, partial remission, and non-remission. The rate of change of renal function will also be evaluated, defined as a 25 mL/min/1.73m2 reduction in follow-up estimated GFR (eGFR) compared to baseline eGFR; 50% decline in follow-up eGFR compared to baseline measurement; and ESRD defined as eGFR ≤ 10cc/min, initiation of maintenance dialysis or preemptive kidney transplantation.
Secondary outcome measures include quality of life assessment, development of new-onset diabetes, malignancies, infections, thromboembolic events, hospitalization, acute kidney injury, and death.
Adults and children with a diagnosis of FSGS, MCD, or MN or with an incipient diagnosis of these syndromes are eligible for the study. Individuals must have a proteinuria ≥ 500 mg/day, estimated from a 24 hour or protein to creatinine ratio urine collection, to participate. Patients with sub-nephrotic proteinuria are included to capture the broad spectrum of clinical presentation.
Exclusion criteria are documented in the study protocol.
This study is ongoing.