UG3FD007308

Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome [PREPARE-NS] - Abstract

The PREPARE-NS program aims to develop and establish the psychometric properties of a core set of clinical outcome assessments (COAs) for nephrotic syndrome, with a primary focus on fluid overload (FO).

During the UG3 phase, a diverse team of experts and stakeholders will be convened, in cooperation with the FDA, to confirm conditions, measures, and anchors. The team will also design the studies necessary to identify a rigorous and patient-centered FO endpoint definition and measure set.

The UH3 phase will focus on testing and implementing the core set of COAs identified in the UG3 phase. The research conducted in the UH3 phase will allow us to refine the FO COAs. The final activities will be focused on making the final core set of COAs widely available to the public for use in trials.

For both phases, we will work in close consultation with the project stakeholder engagement and clinician expert panels. We will draw from the perspectives of patients, clinicians, regulators, payers, and drug developers. By tapping into these resources and relying on our team's wide-ranging clinical, COA development, and psychometric expertise, we will generate a core set of highly-relevant COAs that will advance patient-focused drug development in nephrotic syndrome.

Regents Of The University Of Michigan

93.103 - Food and Drug Administration Research

FDA Office of Acquisition and Grant Services (OAGS) [HHS - FDA]

Center for Drug Evaluation and Research (CDER) [HHS - FDA]

Michigan United States

State-Wide

Development of Standard Core Clinical Outcomes Assessments (COAs) and Endpoints (UG3/UH3 Clinical Trial Optional) (RFA-FD-21-004)

Amendment Since initial award the total obligations have increased 278% from $675,017 to $2,553,894.