Protocol for an open-label, single-arm, multicentre clinical study to evaluate the efficacy and safety of rituximab in the first episode of paediatric idiopathic nephrotic syndrome.

ABSTRACT

INTRODUCTION: Rituximab (RTX) effectively prevents relapses in patients with complicated steroid-sensitive nephrotic syndrome (SSNS). The 1-year relapse-free survival rate is approximately 30% in children after the first episode of SSNS treated with standardised corticosteroids. Whether the benefits of RTX extend to the first relapse are unknown. The efficacy and safety of RTX in the first episode of paediatric idiopathic nephrotic syndrome (RTXFIRPedINS) trial (NCT04783675) will assess its effect on the risk of subsequent relapse. METHODS AND ANALYSIS: RTXFIRPedINS is an open-label, single-arm, multicentre trial targeting patients aged 1-18 years with a first episode of SSNS. All patients will receive standardised corticosteroid treatment for 12 weeks. A sample size of 44 patients provides 80% power to detect a 20% increase in the 1-year relapse-free rate, assuming a dropout rate of 10%. After obtaining informed consent and screening, eligible patients will be treated with a single intravenous infusion of 375 mg/m2 RTX within 1 week after achieving remission. Trimethoprim-sulfamethoxazole will be administered for 3 months after RTX administration to prevent Pneumocystis carinii infection. The follow-up period will be 1 year. The primary outcome is the 1-year relapse-free survival rate after RTX infusion. The secondary study outcomes are the number of days from the infusion of RTX to the occurrence of the first relapse, 6-month relapse-free survival rate, the B cell recovery time and treatment-related adverse events. Immunological factors will be studied as predictors of response. ETHICS AND DISSEMINATION This trial was approved by the Ethics Committee of the Children's Hospital of Fudan University and seven local ethics committees. We will publish our study results in peer-reviewed journals and present them at international scientific meetings. TRIAL REGISTRATION NUMBER NCT04783675.