A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 33
- 试验地点
- 24
- 主要终点
- Incidence, severity and dose relationship of adverse events
研究概览
简要总结
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
详细描述
This is an open-label, multiple-ascending dose study of ARCT-032 in adults with CF who are not on CFTR modulator therapy. After successful screening, eligible participants enrolled in cohorts 1-3 will receive nebulized ARCT-032 daily for 4 weeks and then will be followed for safety for a total of 12 weeks. In cohort 4, eligible participants will receive nebulized ARCT-032 for 12 weeks and will then be followed for safety for an additional 12 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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
- •Confirmed diagnosis of Cystic Fibrosis
- •Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators)
- •FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height
排除标准
- •History of illness or medical condition that might pose an additional risk or may confound study results
- •Recent moderate or severe hemoptysis
- •Recent major surgery
- •Solid organ or hematologic transplant
- •Requirement of supplemental oxygen while awake or > 2L per minute while sleeping.
- •Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day
- •Adequate liver and kidney function as determined by lab tests
研究组 & 干预措施
Cohort 1
Dose Level A of ARCT-032, inhaled daily for 28 days
干预措施: ARCT-032 (Biological)
Cohort 3
Dose Level C of ARCT-032, inhaled daily for 28 days
干预措施: ARCT-032 (Biological)
Cohort 2
Dose Level B of ARCT-032, inhaled daily for 28 days
干预措施: ARCT-032 (Biological)
Cohort 4
Dose level B or C of ARCT-032, inhaled once daily for 12 weeks
干预措施: ARCT-032 (Biological)
结局指标
主要结局
Incidence, severity and dose relationship of adverse events
时间窗: 16 weeks
Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level
次要结局
- Pharmacokinetics(Up to 6 weeks)
- Pharmacodynamics--Lung Function(4 weeks)
- Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)(4 weeks)