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What is Nephrotic Syndrome?

Clinical Overview

Nephrotic syndrome is a group of symptoms that show your kidneys are not working as well as they should. These symptoms include too much protein in your urine, not enough protein in your blood, too much fat or cholesterol in your blood, and swelling.


The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) has information about nephrotic syndrome in adults and nephrotic syndrome in children.

Patient Organizations
Current Research in this Space
(Observational Studies)


The Nephrotic Syndrome Study Network (NEPTUNE) is a 24-site consortium designed to foster clinical and translational research to redefine nephrotic syndrome on a mechanistic level and to translate new discoveries to the bedside. The NIH sponsored consortium is coordinated at the University of Michigan with a focus on patients with Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), Membranous Nephropathy (MN) or childhood onset nephrotic syndrome. 

NEPTUNE includes a) longitudinal cohort study with bio-specimens and data collection for use in scientific


discovery and b) a biology-based patient to clinical trial matching program, NEPTUNE MATCH.

Learn more at


Glomerulonephropathy (CureGN) is a 66-center consortium that fosters research through a prospective observational study and biobank with 2,400 patients with glomerular disease.

Funded by the NIH-NIDDK, University of Michigan manages the CureGN data coordinating center. Participating children and adults with FSGS, MCD, MN, or IgA nephropathy graciously share their clinical data, patient-reported data, biospecimens and insight to support clinical and translational research.


Learn more at



The mission of the KRN is to improve kidney disease treatment options and optimize patient health. KRN engages patients, medical teams, researchers, patient advocates, regulatory, and industry partners to accelerate research to find better treatments with a focus on rare kidney diseases.

The KRN, coordinated by the University of Michigan Pediatric Nephrology Research Program, includes a Patient Registry of 1,200 participants with proteinuric kidney disease who consent to share data from their electronic health records; Clinical Trials Consulting and Endpoints Consortium to improve clinical trial designs and trial endpoint options; and Quality


Improvement Program to improve the care provided to patients with kidney disease.

Learn more at

The Nephrotic Syndrome Patient Reported Outcomes Consortium is a collaboration between academia, patient foundation partners, and industry to develop measures that collect information directly from patients about how they feel and function. These measures can be used in clinical and research settings.

Learn more at

Treatments and Therapies

Treatment and Therapies

Clinical trials for nephrotic syndrome may focus on:

  • Drugs, biologics, or devices for the modification of disease. Examples of therapies tested for disease modification include immunosuppression agents and specific biologic pathway inhibitors.


  • Symptom control. Examples of therapies tested for symptom control may include agents such as diuretics.


A summary of active clinical trials in Nephrotic Syndrome can be found online. Searches including specific diagnoses such as Membranous Nephropathy, Focal Segment Glomerulosclerosis (FSGS), Minimal Change Disease, Frequently Relapsing Nephrotic Syndrome, etc. can help to refine the search.


Check out the online clinical trial search engines:

Patient-focused Drug Development

Patient Focused Drug Development

As experts in what it is like to live with their condition, patients should play a major role in helping to define the therapeutic context for drug development and evaluation. Patient-focused drug development (PFDD) ensures that patients’ experiences, perspectives, needs, and priorities are prioritized when developing and evaluating drugs.

The FDA describes several requirements of PFDD:

  • Facilitating and advancing use of systematic approaches to collecting and utilizing robust and meaningful patient and caregiver input to more consistently inform drug development and regulatory decision-making

  • Encouraging identification and use of approaches and best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials

  • Enhancing understanding and appropriate use of methods to capture information on patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes


  • Identifying the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to support their decision making.

 FDA’s Efforts To Advance Patient-focused Drug Development

The FDA’s Patient-focused Drug Development Program includes several initiatives to support PFDD in drug development and evaluation.  These include:

Links to FDA resources and public meetings

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