What is Nephrotic Syndrome?
Current Research in this Space
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 https://neptune-study.org
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 https://curegn.org
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 https://www.kidneyresearchnetwork.org
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 www.kidneyresearchnetwork.org/patient-reported-outcomes
Treatments 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
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:
A Guidance Series on Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making
FDA-led Patient-Focused Meetings, and Externally-Led Patient-Focused Meetings designed to engage patients and elicit their perspectives on their symptoms, quality of life, and approaches to treatment
A Pilot Grant Program to support the development of publicly available Clinical Outcome Assessments (COAs) for specific disease indications. Prepare-NS is one of two 2021 awardees in this program.
The FDA encourages stakeholders to submit written comments on grantee milestone documents and public meeting content: FDA-2020-N-1727-0003
Links to FDA resources and public meetings
FDA- Center for Drug Evaluation and Research (CDER): Patient-Focused Drug Development
FDA- Center for Drug Evaluation and Research (CDER): Clinical Outcome Assessment (COA) Qualification Program
FDA- Center for Drug Evaluation and Research (CDER): Drug Development Tool Qualification Project Search
FDA- Center for Drug Evaluation and Research (CDER): List of Qualified Clinical Outcome Assessments (COAs)