A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 466
- 试验地点
- 608
- 主要终点
- Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 10 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •APOL1 genotype of G1/G1, G2/G2, or G1/G2
- •Proteinuric kidney disease
- •- Completion of Treatment Period in Part A and no permanent discontinuation of study drug.
排除标准
- •Solid organ or bone marrow transplant
- •Uncontrolled hypertension
- •History of diabetes mellitus
- •Known underlying cause of kidney disease including but not limited to sickle cell disease
- •ESKD (End Stage Kidney Disease) as defined in the protocol.
- •Any lab abnormality that may pose a safety risk to the participant, as judged by the investigator.
- •Other protocol defined Inclusion/Exclusion criteria will apply.
研究组 & 干预措施
Part A (Phase 2/3): Placebo
Participants will receive placebo matched to VX-147.
干预措施: Placebo (Drug)
Part A (Phase 2/3): VX-147
Participants will be randomized to receive different doses of VX-147. Participants will receive the selected dose of VX-147 for atleast 96 weeks.
干预措施: VX-147 (Drug)
Part B (Phase 3): VX-147
Participants who complete Part A will receive VX-147 for an additional 96 weeks.
干预措施: VX-147 (Drug)
结局指标
主要结局
Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)
时间窗: From Baseline to Week 48
Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis
时间窗: From Baseline Through >= Week 48
Part A: eGFR Slope Assessed at Final Analysis
时间窗: From Baseline Through Study Completion (At least 2 years of eGFR data assessed at the final analysis)
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)
次要结局
- Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)(Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls))
- Part A: Maximum Plasma Concentration (Cmax) of VX-147(Day 1 and Week 40)
- Part A: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147(Day 1 and Week 40)
- Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-147(Day 1 up to Week 40)
- Part A: Acceptability Tablet Formulation of VX-147 in Pediatric Participants using the Convenience Domain of the Treatment Satisfaction Questionnaire for Medication (TSQM) Version 1.4(Day 1 and Week 48)
- Part B: Percent Change From Baseline in UPCR Over Time(From Baseline Through Study Completion (Approximately 4 Years After the Last Participant Enrolls))
- Part B: eGFR Slope Assessment(Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls))