NCT07161037进行中（未招募）2 期
A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 26
- 试验地点
- 93
- 主要终点
- htTKV on MRI Over Time
研究概览
简要总结
The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •A pre-existing diagnosis of ADPKD as defined in the protocol
- •Willing and able to comply with scheduled visits and other study procedures
- •Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdominal MRI obtained during screening
- •Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73 square meter (m^2)
排除标准
- •History of kidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD
- •History of solid organ or bone marrow transplantation or nephrectomy
- •Ongoing renal replacement therapy or planning to start renal replacement therapy within 12 months of screening
- •Other protocol defined Inclusion/Exclusion criteria will apply.
研究组 & 干预措施
VX-407
Experimental
Participants will receive VX-407 for up to 52 weeks.
干预措施: VX-407 (Drug)
结局指标
主要结局
htTKV on MRI Over Time
时间窗: Baseline up to End of Study (Week 52)
次要结局
- Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(From Day 1 up to Week 54)
- Maximum Observed Plasma Concentration (Cmax) of VX-407(From Day 1 up to Week 52)
- Area Under the Concentration Versus Time Curve (AUC) of VX-407(From Day 1 up to Week 52)
研究者
研究点 (93)
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