NCT02971839终止2 期
A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations.
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 11
- 试验地点
- 14
- 主要终点
- Change From Baseline in Sweat Chloride at Day 28
研究概览
简要总结
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
详细描述
This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years of age or older
- •Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.
- •Has been stable on Kalydeco therapy for at least 3 months prior to screening
- •Has FEV1 ≥ 60% of predicted normal for age, sex, and height at screening and baseline (Day 1) assessments
- •Weighs at least 40 kg at screening
- •Patients of either gender and women of childbearing potential must be willing to use a medically highly effective form of birth control during the treatment period and 30 days after the last dose of study treatment.
排除标准
- •Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment
- •Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes
- •History of hepatitis C or chronic active hepatitis B infection
- •History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening
- •Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening
- •Abnormal liver function
- •History of abnormal renal function
- •History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females
- •History of solid organ or hematological transplantation
- •Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening
- •Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose
研究组 & 干预措施
VX-561 150 mg
Experimental
干预措施: VX-561 (Drug)
Ivacaftor
Active Comparator
干预措施: IVA (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
VX-561 100 mg
Experimental
干预措施: VX-561 (Drug)
VX-561 20 mg
Experimental
干预措施: VX-561 (Drug)
结局指标
主要结局
Change From Baseline in Sweat Chloride at Day 28
时间窗: From baseline at Day 28
次要结局
- Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28(From baseline at Day 28)
- Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28(From baseline at Day 28)
研究者
研究点 (14)
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