# Nephrotic Syndrome Rare Disease Clinical Research Network III

> **NIH NIH U54** · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2020 · $1,574,136

## Abstract

ABSTRACT
Focal and Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD) and Membranous
Nephropathy (MN), presenting as Nephrotic Syndrome (NS), are rare diseases causing catastrophic
complications and end stage kidney disease, generating enormous individual, societal and economic burdens.
The clinical, histopathology-based taxonomy of NS is inadequate and fails to capture the molecular bases of
these diseases and does not adequately predict their natural history or response to therapy. A Precision
Medicine (PM) approach is necessary to define NS in molecular terms, to identify therapeutic targets and
match patients to treatments. NEPTUNE was applied an innovative, investigational strategy to improve the
diagnosis, management and treatment of NS. In the first two project periods, a translational and clinical
research infrastructure has been established, participants enrolled, biosamples collected, key collaborations
forged and an outreach strategy deployed. NEPTUNE has established a robust investigative infrastructure
encompassing 26 academic centers and two patient interest groups, has recruited more than 750 rigorously
phenotyped NS participants with detailed clinical, histological, genetic, transcriptomic and proteomic data sets.
This comprehensive information has been integrated in the NEPTUNE Knowledge Network for easy access by
the NS research community. With substantial support from the patient interest group NephCure Kidney
International, NEPTUNE has established robust training and ancillary study programs with 112 ancillary
studies ranging from methods development to successful Phase II clinical trials. These critical advances have
resulted in a significant interest in NS clinical trials, with more than 15 trials now in the advanced planning or
enrollment phase.
In this renewal application, we propose to leverage the NEPTUNE resources to catalyze discovery, training
and outreach as we strive to improve health outcomes for individuals affected by NS. The overarching goal is
to apply a PM approach to NS, leveraging the extensive NEPTUNE Knowledge Network established over the
past 9 years. NEPTUNE will implement this PM strategy to permit discovery of novel therapeutic targets and
deploy the patient stratification approach developed in the current funding cycle to help identify the right trial for
the right patient at the right time: Patient stratification approaches will be utilized for targeted enrollment into
clinical trials. Patients will undergo intense profiling at the time of disease presentation or at follow-up renal
biopsy in order to match the disease mechanism active with ongoing clinical trials in a precompetitive, public
private partnership with leading companies in the field. Training, pilot and ancillary study programs will
continue with significant funding support from NKI. NEPTUNE will maintain its engagement with lay
communities, clinicians, scientists, regulatory agencies and the pharmaceutical industry to ide...

## Key facts

- **NIH application ID:** 10017205
- **Project number:** 5U54DK083912-12
- **Recipient organization:** UNIVERSITY OF MICHIGAN AT ANN ARBOR
- **Principal Investigator:** Matthias Kretzler
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,574,136
- **Award type:** 5
- **Project period:** 2009-09-08 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10017205

## Citation

> US National Institutes of Health, RePORTER application 10017205, Nephrotic Syndrome Rare Disease Clinical Research Network III (5U54DK083912-12). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10017205. Licensed CC0.

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