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Clinical Outcome Assessment Endpoints in Nephrotic Syndrome Drug Development: Research Partner Feedback on
John Devin Peipert1,2,3, Maja Kuharic3, Noelle E Carlozzi4
1Centre for Patient Reported Outcomes Research, Department of Applied Health Sciences, University of Birmingham, Birmingham, UK.
Glomerular Diseases
|May 11, 2026
Summary
Clinical outcome assessments (COAs) are needed for nephrotic syndrome (NS) trials to capture patient-reported swelling impacts. The Prepare-NS project surveyed experts to develop these vital patient-centered endpoints.
Area of Science:
- Nephrology
- Clinical Trials
- Patient-Reported Outcomes
Background:
- Nephrotic syndrome (NS) clinical trials often use biomarkers or clinical measures that may not fully reflect patient-experienced treatment benefits.
- Edema-related swelling in NS significantly impacts patients' quality of life, physical function, and daily activities, necessitating direct measurement of its severity and impact.
Purpose of the Study:
- To address the unmet need for clinical outcome assessments (COAs) that directly measure the severity and impact of edema-related swelling in nephrotic syndrome (NS).
- To inform the development and validation of standardized COAs for NS clinical trials through expert consultation.
Main Methods:
- Conducted qualitative interviews and surveys with clinical trialists from industry, academia, and patient advocacy organizations.
- Gathered expert opinions on the utility, timing, frequency, format, and alignment of COAs for demonstrating treatment benefit in NS trials, focusing on swelling impacts.
Main Results:
- Experts strongly supported using COA-based endpoints to complement existing clinical or biomarker endpoints in NS trials.
- Identified multiple potential endpoints for operationalizing swelling COAs and discussed key considerations like assessment timing, feasibility of daily diaries, confounding factors, and alignment with other trial endpoints.
Conclusions:
- Insights from this study will guide the ongoing development and validation efforts of the Prepare-NS initiative.
- Standardized COAs in NS clinical trials can expand efficacy endpoints and promote patient-centered drug development.
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