Functional Respiratory Imaging (FRI) to Assess the Short-term Effect of the Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function in ORKAMBInaive Patients With Cystic Fibrosis Homozygous for Phe508del
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Change in specific image-based airway volumes (siVaw)
研究概览
简要总结
Open-label study to investigate the effects of Orkambi in CF patients homozygous for the F508del mutation by functional respiratory imaging. Primary endpoints in this study are the changes in Specific airway volumes (siVaw) and Specific Airway resistance (siRaw). A total of 20 ORKAMBI-naive patients with Cystic Fibrosis, homozygous for the F508del mutation will be included in this open label study and will be followed through 3 months of treatment. The treatment will be started after all assessments are performed at visit 1. After the start of the treatment some baseline measurements will be repeated throughout the 3-month treatment period. The patient will be asked to visit the hospital monthly. All study visits should be scheduled around the same time.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history).
- •Age ≥ 12 years
- •FEV1 > 50%
- •Signed informed consent. If patient is a minor, parents/guardians must give written informed consent
- •Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit
排除标准
- •FEV1 < 50%
- •Anticipated requirement for hospitalization within the next three weeks
- •History of pneumothorax within the past 6 months prior to Visit 1
- •History of haemoptysis requiring embolization within the past 12 months prior to Visit 1
- •Unable or unwilling to complete study visits or provide follow-up data as required per the study protocol
- •Has taken Intravenous (IV) antibiotics within the past 4 weeks prior to Visit 1
- •Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA)
- •Pregnant or lactating female
- •Posttransplant patients
- •Patients with severe hepatic impairment
研究组 & 干预措施
Orkambi open-label arm
Open-label study: all subjects will receive Orkambi during 3 months.
干预措施: Orkambi (Drug)
结局指标
主要结局
Change in specific image-based airway volumes (siVaw)
时间窗: baseline and after three months of therapy
change in CT-based airway volumes normalized by the lung volume
Change in specific image-based airway resistance (siRaw)
时间窗: baseline and after three months of therapy
Change in CFD-based airway resistance normalized by the lung volume
次要结局
- Exacerbation frequency(baseline and after three months of therapy)
- airway resistances(baseline and after three months of therapy)
- Lung clearance index(baseline and after three months of therapy)
- Digital lung auscultation(baseline and after three months of therapy)
- Internal Airflow Distribution(baseline and after three months of therapy)
- Dynamic lung volumes(baseline and after three months of therapy)
- Air Trapping(baseline and after three months of therapy)
- Airway Wall Volume(baseline and after three months of therapy)
- Aerosol Deposition(baseline and after three months of therapy)
- Static lung volumes(baseline and after three months of therapy)
- 6-minute walking test(baseline and after three months of therapy)
- Sweat chloride test(baseline and after three months of therapy)
- CFQ-R(baseline and after three months of therapy)
研究者
Stijn Verhulst
Director Department of Pediatrics
University Hospital, Antwerp