A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients With IgA Nephropathy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 31
- 试验地点
- 1
- 主要终点
- Percent Change From Baseline in UPCR as Measured in 24-hour Urine at Week 32
研究概览
简要总结
The purpose of this study is to evaluate the effect of cemdisiran on proteinuria in adults with immunoglobulin A nephropathy (IgAN), who excrete >1 gram (gm) of protein per day despite standard of care, which includes treatment with angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARB). These participants are at high risk for progression of kidney disease, which can result in end-stage renal failure.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with primary IgAN
- •Currently being treated for IgAN with stable, optimal therapy, including an ACE inhibitor or ARB.
- •Has urine protein greater than or equal to 1 gram/24-hour
- •Has hematuria (blood cells present in urine)
排除标准
- •Has renal disease other than IgAN
- •Has a diagnosis of rapidly progressive glomerulonephritis
- •Has a diagnosis of Henoch-Schonlein Purpura (IgA Vasculitis)
- •Has poor kidney function with estimated glomerular filtration rate (eGFR) <30 milliliters per minute per 1.73 meters square (mL/min/1.73 m^2)
- •Has known human immunodeficiency virus (HIV) infection, hepatitis C virus (HCV) infection or hepatitis B virus (HBV) infection
- •Has on-going high blood pressure
- •Treated with systemic corticosteroids for more than 7 days, or other immunosuppressant agents in the past 6 months
- •Received an organ transplant
研究组 & 干预措施
Double-Blind Treatment (DBT) Period: Cemdisiran
Participants received cemdisiran, 600 milligrams (mg), subcutaneous (SC) injection, once every 4 weeks (Q4W) in combination with standard of care treatment up to a maximum of 38 weeks in the DBT period.
干预措施: Cemdisiran (Drug)
DBT Period: Placebo
Participants received cemdisiran matching placebo, SC injection, Q4W in combination with standard of care treatment up to a maximum of 38 weeks in the DBT period.
干预措施: Placebo (Drug)
DBT Period: Cemdisiran to Open-Label Extension (OLE) Period: Cemdisiran
Participants who were randomized to receive cemdisiran in the DBT period continued receiving cemdisiran, 600 mg, SC injection, Q4W in combination with standard of care treatment up to a maximum of 88 weeks in the OLE treatment period.
干预措施: Cemdisiran (Drug)
DBT Period: Placebo to OLE Period: Cemdisiran
Participants who were randomized to receive cemdisiran matching placebo in the DBT period started receiving cemdisiran, 600 mg, SC injection, Q4W in combination with standard of care treatment up to a maximum of 88 weeks in the OLE treatment period.
干预措施: Cemdisiran (Drug)
结局指标
主要结局
Percent Change From Baseline in UPCR as Measured in 24-hour Urine at Week 32
时间窗: Baseline to Week 32
UPCR is a way of assessing the amount of protein in the urine. The primary analysis for UPCR was performed using Mixed-Effect Model Repeated Measures (MMRM) approach. Geometric mean (GM)ratios were obtained by exponentially back-transforming the arithmetic mean of change in log-transformed 24h UPCR. Standard error of the mean (SEM) was calculated as exponential (mean of change in log-transformed data) \* (standard error of change in log-transformed data). Adjusted GM ratio to baseline and 90% confidence interval (CIs) were calculated by exponentially back-transforming the model-based least square (LS) mean and the corresponding 90% CI.
次要结局
- Percent Change From Baseline in 24-hour Proteinuria at Week 32(Baseline to Week 32)
- Number of Participants With Adverse Events (AEs)(Up to 126 weeks)
- Percentage of Participants With >50% Reduction in 24-hour Proteinuria at Week 32(Week 32)
- Percentage of Participants With Partial Clinical Remission at Week 32(Week 32)
- Change From Baseline in UPCR as Measured in a Spot Urine at Week 32(Baseline to Week 32)
- Number of Participants With Change From Baseline in Hematuria at Week 32(Baseline to Week 32)