A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults With Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 160
- 试验地点
- 1
- 主要终点
- The change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value
研究概览
简要总结
This is a dose-finding study for the investigational product Lancovutide (Moli1901) in the exploratory phase IIb to establish minimum effective dose, optimal dose, and maximum safe dose. Additionally, the tolerability of Moli1901 shall be investigated.
详细描述
Study Moli1901-010B is a multi center, parallel group, placebo controlled, double-blind efficacy and safety evaluation of three different dosage schedules of aerosolized Moli1901 in adolescents (12 years of age or older), and adults. Study will start with a screening period (visit 1) followed by an 8 weeks double blind comparative treatment period (visit 2-7). Thereafter, subjects will be observed for additional 4 weeks without treatment (follow-up period and visit 8). Spirometry, pulse oximetry and other safety measurements will be performed up to 120 min after study medication inhalation. The study will be conducted in 30 sites in 9 European countries.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Body mass index
- •Confirmed diagnosis of cystic fibrosis
- •FEV1 between 50% and 85% of predicted
- •Oxygen saturation level measured by pulse oximetry (SpO2) >90 % on room air
排除标准
- •Bronchial hyperresponsiveness
- •Unstable lung function
- •Pulmonary disease such as pneumonia, tuberculosis, or lung cancer
- •Acute upper respiratory tract infection within the last 2 weeks
- •Acute lower respiratory tract infection (requiring antibiotics or hospitalization) within the last 4 weeks
- •Pulmonary exacerbation within the last 4 weeks
- •Changes from routine maintenance therapy within the last 4 weeks
- •Scheduled changes to inhaled antibiotics regimen during the course of the study
- •Receive or are planned to receive any treatment via "on-off" regimen (e.g. Tobramycin - TOBI®); last dose of any "on-off" treatment within the last 6 weeks
- •Any clinically significant liver, renal, cardiac, neurological, or hematological disease
- •ABPA or colonization with Burkholderia cepacia
- •Poorly controlled diabetes mellitus
研究组 & 干预措施
1
daily inhalation
干预措施: Moli1901 (Drug)
2
inhalation every other day
干预措施: Moli1901 (Drug)
3
inhalation twice a week
干预措施: Moli1901 (Drug)
4
daily inhalation
干预措施: Moli1901 placebo (Drug)
结局指标
主要结局
The change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value
时间窗: at every study visit
次要结局
- The ensemble of two subject reported clinical scores, which are physical dimensions of the self-report, disease-specific quality of life test, the cystic fibrosis questionnaire in the revised version(every 2 weeks)