# Study B- cNEPTUNE

> **NIH NIH U54** · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2022 · $536,324

## Abstract

ABSTRACT
Current approaches to childhood nephrotic syndrome (NS) are based on routine clinical and laboratory findings
that are unable to accurately define the underlying biologic mechanisms, predict clinical course, or select optimal
therapy. Treatment of childhood NS lacks a rational mechanistic basis and is associated with significant morbidity
and is responsible for 12% of the pediatric end stage kidney disease in the USA. A precision medicine approach
is warranted to identify mechanistically discrete disease subgroups, refine biomarkers to assist with molecular
classification, identify relevant treatment targets, and improve trial design for pediatric NS. The children's
nephrotic syndrome cohort in the Nephrotic Syndrome Study Network (Cohort B) is inclusive of 16 enrolling
pediatric centers and associated investigators. In this project, Cohort B will expand to 200 children with incident
NS, prospectively followed to define the longitudinal disease course, collect biospecimens to enable
comprehensive molecular profiling (phenotype, genotype, immune profiling) for treatment target identification.
cNEPTUNE will develop and improve clinical trial methods, endpoints, and systems to enable the conduct of
precision medicine trials inclusive of children with nephrotic syndrome. In this renewal proposal, we propose to
(a) define genotype-phenotype subgroups of children with incident NS; (b) define non-invasive biomarkers,
inclusive of immunological profiles, in functionally defined subgroups of NS; (c) identify candidate therapeutic
targets based on the mechanistically defined NS; and (d) develop validated endpoints, trial designs, and systems
to foster efficient and effective precision medicine clinical trials for patients with NS.

## Key facts

- **NIH application ID:** 10480855
- **Project number:** 5U54DK083912-14
- **Recipient organization:** UNIVERSITY OF MICHIGAN AT ANN ARBOR
- **Principal Investigator:** DEBBIE S GIPSON
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $536,324
- **Award type:** 5
- **Project period:** 2009-09-08 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10480855

## Citation

> US National Institutes of Health, RePORTER application 10480855, Study B- cNEPTUNE (5U54DK083912-14). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10480855. Licensed CC0.

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