Kidney Week

Abstract: INFO21-FR

Field Testing and Validation of Patient-Reported and Observer-Reported Measures for Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome (Prepare-NS)

Session Information

• Informational Posters - 2
  October 23, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• No subcategory defined

Authors

• Salmon, Eloise, University of Michigan, Ann Arbor, Michigan, United States

Eloise Salmon, MD

• Hashemi, Somayeh, University of Michigan, Ann Arbor, Michigan, United States

Somayeh Hashemi, MA, MSc, MPH

• Hurt, Courtney N., Northwestern University, Evanston, Illinois, United States

Courtney N. Hurt, MSW

• Rahimi, Ashley E., University of Michigan, Ann Arbor, Michigan, United States

Ashley E. Rahimi, MS

• Scherr, Rebecca, University of Michigan, Ann Arbor, Michigan, United States

Rebecca Scherr, MA

• Zhai, Yan, University of Michigan, Ann Arbor, Michigan, United States

Yan Zhai, MS

• Carlozzi, Noelle E., University of Michigan, Ann Arbor, Michigan, United States

Noelle E. Carlozzi, PhD

• Lai, Jin-Shei, University of Michigan, Ann Arbor, Michigan, United States

Jin-Shei Lai, PhD

• Peipert, John Devin, University of Birmingham, Birmingham, England, United Kingdom

John Devin Peipert, PhD

Description

Nephrotic syndrome (NS) affects children and adults with rare kidney diseases and includes swelling that substantially impairs health-related quality of life, but patient-centered clinical outcome assessments (COAs) to evaluate treatment benefit in trials of NS therapeutics are lacking. The PREPARing a clinical outcomes assessment set for Nephrotic Syndrome (Prepare-NS, FD007308) initiative created a standardized set of COAs specifically to measure the effects of NS-related swelling impact (NS-SIM). The COA set includes both patient- (PRO – for affected individuals 8 and older) and observer-reported outcomes (ObsRO – for caregivers of affected children age 2-11) in multiple formats. For the field testing and validation study, participants complete NS-SIM-PRO (21 items) or NS-SIM-ObsRO (19 items) via REDCap software or paper administration at enrollment, three months, and nine months. If also participating in the optional daily diary, NS-SIM-PRO-DD or NS-SIM-ObsRO-DD (6 items) delivered via LifeData software on study days 1-21, 83-104, and 249-270. Image 1 shows sample items from NS-SIM-PRO and NS-SIM-PRO-DD. Key inclusion criteria are current edema, NS diagnosis of focal segmental glomerulosclerosis, minimal change disease, membranous nephropathy or childhood-onset not biopsied, and preserved kidney function. Recruitment targets are 200 impacted individuals for NS-SIM-PRO and 100 caregivers for NS-SIM-ObsRO. Upon completion of field testing and validation, the COA set will be publicly available to increase the pateint voice in both drug trials and clinical care.

Funding

• U.S. Food and Drug Administration