A Phase 2, Open-Label, Multicenter, Multi-cohort Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 216
- 试验地点
- 27
- 主要终点
- PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL)
研究概览
简要总结
This study will have 2 parts: Pharmacokinetics (PK) Lead-in Phase and the Treatment Phase.
The primary objective of the PK Lead-in Phase is to evaluate the steady state PK and confirm the dose of sofosbuvir/velpatasvir (SOF/VEL) fixed-dose combination (FDC) in pediatric participants with chronic hepatitis C virus (HCV) infection.
The primary objective of the Treatment Phase is to evaluate the safety and tolerability of SOF/VEL for 12 weeks in pediatric participants with chronic HCV.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronic HCV-infected, treatment-naive and treatment-experienced adolescent and pediatric individuals aged 3 to < 18 as determined at Day
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
排除标准
- 未提供
研究组 & 干预措施
12 to < 18 Years Old
PK Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) 400/100 mg once daily for 7 days. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase.
Treatment Phase: SOF/VEL 400/100 mg once daily for 12 weeks.
干预措施: SOF/VEL (Drug)
6 to < 12 Years Old
PK Lead-in Phase: SOF/VEL 200/50 mg once daily for 7 days. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase.
Treatment Phase: SOF/VEL 200/50 mg once daily for 12 weeks.
干预措施: SOF/VEL (Drug)
3 to < 6 Years Old
PK Lead-in Phase: SOF/VEL 200/50 mg once daily for 7 days for participants who weigh ≥ 17 kg. SOF/VEL 150/37.5 mg once daily for 7 days for participants who weigh < 17 kg. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase.
Treatment Phase: SOF/VEL 200/50 mg once daily for 12 weeks for participants who weigh ≥ 17 kg. SOF/VEL 150/37.5 mg once daily for 12 weeks for participants who weigh < 17 kg.
干预措施: SOF/VEL (Drug)
结局指标
主要结局
PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL)
时间窗: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).
PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of GS-331007 (Metabolite of SOF)
时间窗: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).
PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Sofosbuvir (SOF)
时间窗: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).
Treatment Phase: Percentage of Participants Who Discontinued Study Drug Due to Any Treatment-Emergent Adverse Event (TEAE)
时间窗: From first dose through last dose of the study drug (Up to 12 weeks) plus 30 days
An AE is any untoward medical occurrence in a clinical study participant administered a medicinal product, which does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and/or unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. TEAEs were defined as 1 or both of the following: Any AEs with an onset date on or after the study drug start date and no later than 30 days after permanent discontinuation of study drug and/or Any AEs leading to premature discontinuation of study drug.
次要结局
- PK Lead-in Phase: Change From Baseline in Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Day 7(Baseline; Day 7)
- Treatment Phase: Percentage of Participants Who Develop Viral Resistance to SOF and/or VEL During Treatment and After Discontinuation of Treatment(First dose date up to Posttreatment Week 24)
- Treatment Phase: Change From Baseline in HCV RNA at Weeks 1, 4, 8, and 12(Baseline; Weeks 1, 4, 8, and 12)
- Treatment Phase: Changes in Growth and Development as Measured by Tanner Stage Assessment From Baseline(Baseline; EOT, FU-12 and FU-24)
- Treatment Phase: Growth and Development as Measured by Parental Height(Day 1)
- Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Day 1(Day 1)
- Treatment Phase: Percentage of Participants With Virologic Failure(Up to Posttreatment Week 24)
- Treatment Phase: Change From Baseline in Growth and Development as Measured by Bone Age(Baseline; FU-24)
- Treatment Phase: Swallowability of SOF/VEL as Assessed by the Participant's Ability to Swallow SOF/VEL Placebo Tablets at Baseline(Baseline)
- Treatment Phase: Change From Baseline in Growth and Development as Measured by Weight Percentiles(Baseline; Weeks 1, 4, 8, 12, FU-4, FU-12, and FU-24)
- PK Lead-in Phase: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event (AE)(First dose date up to Day 7)
- Treatment Phase: Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)(Posttreatment Week 24)
- Treatment Phase: Change From Baseline in Growth and Development as Measured by Height Percentiles(Baseline; Weeks 1, 4, 8, 12, Follow-up (FU) Week 4 (FU-4), FU-12, and FU-24)
- Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Week 12(Week 12)
- Treatment Phase: Percentage of Participants With Sustained Virologic Response (SVR) at 12 Weeks After Discontinuation of Therapy (SVR12)(Posttreatment Week 12)
- Treatment Phase: Percentage of Participants With SVR at 4 Weeks After Discontinuation of Therapy (SVR4)(Posttreatment Week 4)
- Treatment Phase: Percentage of Participants With HCV RNA < LLOQ On Treatment(Weeks 1, 4, 8, and 12)
- Treatment Phase: Quality of Life (QoL) and Neuropsychiatric Assessments as Measured by PedsQL™ Pediatric QoL Survey(Baseline; Week 12, End of Treatment (EOT), Posttreatment/Follow-up (FU) Week-12 (FU-12), and FU Week-24 (FU-24))