[Translation] An additional extended portion of a randomized, double-blind, placebo-controlled, parallel-group phase IIa study evaluating the efficacy and safety of BAY 3401016 in participants aged 18 to 45 years with Alport syndrome.
本研究旨在在18至45岁的Alport综合征(AS)的参与者中探究BAY 3401016能否减少有快速疾病进展风险的AS参与者人群的肾功能衰退。该研究还将研究在此类人群中BAY 3401016重复给药的药代动力学(PK)和安全性。本研究的主要目的为评估BAY 3401016对AS 参与者白蛋白尿的影响,评估指标为治疗16、20和24周时UACR较基线的比值的平均值;次要目的为考察BAY 3401016的安全性和耐受性,将监测治疗期出现的不良事件(TEAE)和治疗期出现的严重不良事件(TESAE);研究的其他目的包括评价其他安全性变量,包括生命体征、ECG、安全性实验室参数等,还将研究 BAY 3401016 的药代动力学及其靶点相互作用,特别是关注血浆中的 Sema3A 浓度,此外还将分析与Alport综合征和肾功能相关的各种生物标志物。
[Translation] This study aims to investigate whether BAY 3401016 can reduce renal function decline in AS participants aged 18 to 45 years with Alport syndrome (AS). The study will also investigate the pharmacokinetics (PK) and safety of repeated administration of BAY 3401016 in this population. The primary objective is to assess the effect of BAY 3401016 on albuminuria in AS participants, using the mean ratio of UACR to baseline at weeks 16, 20, and 24 of treatment. Secondary objectives include examining the safety and tolerability of BAY 3401016, monitoring for treatment-induced adverse events (TEAEs) and treatment-induced serious adverse events (TESAEs). Other objectives include evaluating other safety variables, including vital signs, ECG, and safety laboratory parameters. The pharmacokinetics and target interactions of BAY 3401016 will also be investigated, with particular focus on plasma Sema3A concentrations. Furthermore, various biomarkers associated with Alport syndrome and renal function will be analyzed.