A Multicentre, Randomised, Double-blind, Parallel Group, Placebo-controlled Trial to Assess the Effects of Oral TRPC6 Inhibitor BI 764198 Taken Over a 104 Week Treatment Period in Adult and Adolescent Participants With Primary Focal Segmental Glomerulosclerosis (pFSGS) or Genetic FSGS Related to TRPC6 Gene Variants
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 286
- 试验地点
- 487
- 主要终点
- Relative change in 24-hour UPCR (measured in mg/g) from baseline to Week 104
研究概览
简要总结
PODOMOUNT-pFSGS
This study is open to adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS). The purpose of this study is to find out whether a medicine called BI 764198 helps people with FSGS.
Participants are put into 2 groups randomly, which means by chance. Every participant has an equal chance of being in each group. One group takes BI 764198 tablets, and the other group takes placebo tablets. Placebo tablets look like BI 764198 tablets but do not contain any medicine.
Participants take a tablet once a day for up to 2 years. All participants also continue their standard medication for FSGS.
Participants are in the study for up to 2 years. During this time, they visit the study site about every 3 months. Participants regularly collect urine samples. This is done to check their kidneys. The results are compared between the two groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants ≥12 years old on the day of signing informed consent/assent (Visit 1)
- •Weight of ≥40 kg at the screening visit (Visit 1)
- •Body mass index (BMI) of ≤40 kg/m² at the screening visit (Visit 1)
- •Participants with a diagnosis prior to the screening visit (Visit 1) of either:
- •Biopsy-confirmed primary focal segmental glomerulosclerosis (pFSGS) (based on Investigator's judgement) OR
- •Genetic focal segmental glomerulosclerosis (FSGS) resulting from a gain-of-function mutation in the transient receptor potential cation subfamily C member 6 (TRPC6) gene (based on historical genetic test)
- •Urine protein-creatinine ratio (UPCR) ≥1500 mg/g based on the mean of the spot urine sample and first morning void (FMV) urine sample (both assessed by central laboratory) at the screening visit (Visit 1)
- •Estimated glomerular filtration rate (eGFR)
- •For adult participants (≥18 years): ≥25 mL/min/1.73 m² (chronic kidney disease epidemiology collaboration (CKD-EPI) formula based on serum cystatin C) at the screening visit (Visit 1)
- •For adolescent participants (12 to <18 years): ≥25 mL/min/1.73 m² based on chronic kidney disease under 25 years (CKiD U25) formula using serum cystatin C at the screening visit (Visit 1) Further inclusion criteria apply.
排除标准
- •Known monogenic or syndromic causes of FSGS (with the exception of TRPC6 gain-of-function gene mutations)
- •Clinical or histologic evidence of secondary adaptive or toxic forms of FSGS (based on Investigator's judgement)
- •FSGS of undetermined cause (FSGS-UC) with a diagnosis prior to the screening visit (Visit 1) (based on Investigator's judgement)
- •A history of organ transplantation or planned organ transplantation during the course of the trial
- •Use of intravenous immunosuppressive agents (e.g. cyclophosphamide, rituximab, obinutuzumab) in the last 6 months prior to screening (Visit 1) Further exclusion criteria apply.
研究组 & 干预措施
Placebo treatment
干预措施: Placebo (Drug)
BI 764198 treatment
干预措施: BI 764198 (Drug)
结局指标
主要结局
Relative change in 24-hour UPCR (measured in mg/g) from baseline to Week 104
时间窗: Baseline and Week 104
24-hour urinary protein-to-creatinine ratio (24-hr UPCR)
Relative change in 24-hour urinary protein-to-creatinine ratio (UPCR, measured in mg/g) from baseline to Week 104
时间窗: Baseline and Week 104
次要结局
- Key secondary endpoint: Absolute change in eGFRcys in mL/min/1.73m2 from baseline to Week 104(Baseline and Week 104)
- Key secondary endpoint: Treatment response, defined as 24-hr UPCR <1000 mg/g at Week 104(At Week 104)
- Treatment response, defined as 24-hr UPCR <1000 mg/g and eGFRcys ≥85% vs. baseline at Week 104 and no treatment failure between randomisation and Week 104 (combined or multi-component endpoint)(At Week 104)
- Complete remission, defined as 24-hr UPCR <300 mg/g at Week 104(At Week 104)
- Change from baseline across disease-specific clinical outcome assessment (COA) NS-SIM-PRO at Week 104(Baseline and Week 104)
- Change from baseline across clinical outcome assessment (COA) KDQOL-36 at Week 104(Baseline and Week 104)