A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 7
- 主要终点
- Safety - Adverse Events
研究概览
简要总结
This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.
详细描述
This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, 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
- •Males or females, ages 18 to 65 years
- •Confirmed diagnosis of Alport syndrome
- •eGFR between 40 and 90 mL/min/1.73m2
- •Proteinuria of at least 300 mg protein/g creatinine
- •For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
- •Willing to comply with contraception requirements
排除标准
- •Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
- •End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
- •Any other condition that may pose a risk to the subject's safety and well-being
- •Female subjects who are pregnant or lactating
研究组 & 干预措施
RG-012 Single Dose
1.5 mg/kg RG012 subcutaneous injection
干预措施: RG012 (Drug)
RG012 Every Other Week
1.5 mg/kg RG012 subcutaneous injections every other week
干预措施: RG012 (Drug)
结局指标
主要结局
Safety - Adverse Events
时间窗: 8 weeks
Incidence and severity of adverse events
Effect of RG-012 on renal microRNA-21 (miR-21)
时间窗: 8 weeks
Change in miR-21 expression in renal tissue
次要结局
- Pharmacokinetic (PK) parameter - Cmax(8 weeks)
- Pharmacokinetic (PK) parameter - Tmax(8 weeks)
- Pharmacokinetic (PK) parameter - AUC(8 weeks)