Clopidogrel for Proteinuria Reduction in Focal Segmental Glomerulosclerosis: Phase 2 Trial Design

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  Figure 1. ClopiD4FSGS trial overview. Patients enrolled in the ARREST NEPHROSIS registry are screened for eligibility. A total of up to 22 patients will be recruited. After the recruitment of 10 patients (= cohort 1), an interim analysis for early futility will be performed after visit 2 (= 84 days after baseline visit). The futility assessment will be based on a secondary parameter only. If no more than 1 patient reaches the focal segmental glomerulosclerosis partial remission end point, the study will be stopped for early futility. Otherwise, recruitment of the remaining patients will be completed, and the primary end point be tested in the final analysis of 22 patients. The primary outcome is the percentage change of the urinary protein-to-creatinine ratio from baseline to week 24. Focal segmental glomerulosclerosis partial remission end point is defined as a urinary protein-to-creatinine ratio <1.5 and a >40% reduction in urinary protein-to-creatinine ratio compared to baseline, which will be reported for each visit. Registry data will be continuously obtained during and following the ClopiD4FSGS trial.

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  Figure 2. ClopiD4FSGS study design. The baseline visit will be conducted 7 weeks after the screening assessment (= baseline period), followed by 24-week treatment period with 75 mg clopidogrel daily (= active study period). UPCR as assessed by 24-hour-urine collections will be assessed at baseline and at each study visit. The primary end point is defined as the percent proteinuria change from baseline to visit 4 (week 24). Individual patient trajectories will be reported. The secondary end point is the proportion of patients who achieve the FPRE after visit 4 (week 24). As tertiary end points, core outcomes as defined by the “Standardized Outcomes in Nephrology” (SONG) initiative will be assessed, including PROM for life participation. Patients are recruited from the ARREST-NEPHROSIS registry (green triangle, green box), registry data will be continuously obtained during and following the ClopiD4FSGS trial. IMP, investigational medicinal product; PROM, patient reported outcome measures; UPCR, urinary protein-to-creatinine ratio. FPRE is defined as UPCR <1.5 and a >40% reduction in UPCR.