Rilparencel (Renal Autologous Cell Therapy-REACT®) for Chronic Kidney Disease and Type 1 and 2 Diabetes: Phase 2 Trial Design Evaluating Bilateral Kidney Dosing and Re-Dosing Triggers.

INTRODUCTION: Autologous cell-based therapies (CBT) to treat Chronic Kidney Disease (CKD) with diabetes are novel and can potentially preserve renal function and decelerate disease progression. CBT dosing schedules are in early development and may benefit from individual bilateral organ dosing and kidney-dependent function to improve efficacy and durability. The objective of this open-label, phase 2 randomized controlled trial (RCT) is to evaluate participants' response to rilparencel (Renal Autologous Cell Therapy-REACT®) following bilateral percutaneous kidney injections into the kidney cortex with a prescribed dosing schedule versus re-dosing based on biomarker triggers.

METHODS: Eligible participants with type 1 or 2 diabetes and CKD, eGFR 20-50 ml/min/1.73 m2, UACR 30-5000 mg/g, Hemoglobin > 10 g/dL and glycated hemoglobin A1c < 10% were enrolled. After a percutaneous kidney biopsy and bioprocessing ex vivo expansion of Selected Renal Cells, participants were randomized 1:1 into two cohorts determined by the dosing scheme. Cohort 1 receives two cell injections, one in each kidney three months apart, and Cohort 2 receives one injection and the second dose only if there is a sustained eGFR decline of ≥20 ml/min/1.73 m2 and/or UACR increase of ≥ 30% confirmed by re-testing.

CONCLUSION: This multicenter phase 2 RCT is designed to investigate the efficacy and safety of rilparencel with bilateral kidney dosing and comparing two injection schedules with the potential of preserving or improving kidney function and delaying kidney disease progression among patients with Stages 3a-4 CKD with diabetes.