NCT05844449Enrolling By Invitation3 期
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 174
- 试验地点
- 54
- 主要终点
- Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parts A and B:
- •Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)
- •Meets at least 1 of the following criteria:
- •Completed study drug treatment in Part A
- •Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A
排除标准
- •Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
- •History of solid organ, hematological transplantation, or cancer
- •History of drug intolerance in the parent study
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
VNZ/TEZ/D-IVA
Experimental
Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study.
Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.
干预措施: VNZ/TEZ/D-IVA (Drug)
结局指标
主要结局
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
时间窗: From Baseline up to Week 100
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
时间窗: From Baseline up to Week 196
次要结局
- Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)(From Baseline Through Week 96)
- Part B: Absolute Change in Sweat Chloride (SwCl)(From Baseline Through Week 192)
- Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)(From Baseline Through Week 100)
- Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)(From Baseline Through Week 196)
- Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)(From Baseline Through Week 100)
- Part B: Absolute Change in Body Mass Index (BMI)(From Baseline Through Week 196)
- Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score(From Baseline Through Week 100)
- Part B: Number of Pulmonary Exacerbation (PEx)(From Baseline Through Week 196)
- Part A (All Cohorts): Number of CF- Related Hospitalizations(From Baseline Through Week 100)
- Part B: Number of CF- Related Hospitalizations(From Baseline Through Week 196)
- Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score(From Baseline Through Week 100)
- Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score(From Baseline Through Week 196)
- Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)(From Baseline Through Week 100)
- Part B: Absolute Change in BMI-for-age Z-score(From Baseline Through Week 196)
- Part A (Cohort 3): Absolute Change in Weight-for-length(From Baseline Through Week 100)
- Part B: Absolute Change in Weight-for-length(From Baseline Through Week 196)
- Part A (Cohort 3): Absolute Change in Weight-for-length Z-score(From Baseline Through Week 100)
- Part B: Absolute Change in Weight-for-length Z-score(From Baseline Through Week 196)
- Part A (All Cohorts): Absolute Change in Weight(From Baseline Through Week 100)
- Part B: Absolute Change in Weight(From Baseline Through Week 196)
- Part A (All Cohorts): Change in Weight-for-age Z-score(From Baseline Through Week 100)
- Part B: Change in Weight-for-age Z-score(From Baseline Through Week 196)
- Part A (Cohorts 1 and 2): Absolute Change in Height(From Baseline Through Week 100)
- Part B: Absolute Change in Height(From Baseline Through Week 196)
- Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score(From Baseline Through Week 100)
- Part B: Absolute Change in Height-for-age Z-score(From Baseline Through Week 196)
- Part A (Cohort 3): Absolute Change in Length(From Baseline Through Week 100)
- Part B: Absolute Change in Length(From Baseline Through Week 196)
- Part A (Cohort 3): Absolute Change in Length-for-age Z-score(From Baseline Through Week 100)
- Part B: Absolute Change in Length-for-age Z-score(From Baseline Through Week 196)
研究者
研究点 (54)
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