Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior: an Evaluation of Triple Combination Therapy in Children Aged 6 to 11 Years
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Delta Small Bowel Water (DeltaSBW)
研究概览
简要总结
Recently trial data has shown that the medicine KaftrioTM (Elexacaftor/ Tezacaftor/ Ivacaftor) improves lung function in children aged 6 to 11 years who have cystic fibrosis (CF). This has led to it being licensed for use in the UK in 2022 and is now being prescribed in this age group. There is little information in trials however that shows the effect KaftrioTM (ETI) has on the gut or liver in this age group.
Previous studies in the GIFT-CF series (NCT 03566550, NCT04006873 and NCT04618185) has found differences in the functioning of the gut between adults with CF and healthy controls but it is not known whether these differences are present in those aged 6 to 11 years.
This study is a significant amendment of the GIFT-CF3 protocol (NCT04618185) that aims to measure gut function using magnetic resonance imaging (MRI) in children with CF before and after starting ETI. This study also aims to opportunistically measure lung function and structure using MRI and explore how the liver can be measured using MRI in this age group.
The study is split into 2 stages. The first is a pilot stage using the modified GIFT-CF protocol recruiting 3 children with CF before starting ETI and 3 healthy volunteers. This is to determine we are able to successfully perform these scans in these age groups. If successful, the second stage will recruit a further 12 children with CF before they start ETI. This will take our cohort up to 15 children with CF. This cohort will then be rescanned 6 months after starting ETI using the same scan protocol.
详细描述
This is an observational cohort study using MRI to measure gastrointestinal (GI) function, lung structure and function and liver endpoints in children with CF aged 6 to 11 years. The study will use a modified version of the protocol used in the GIFT-CF3 study (NCT04618185) to measure GI physiology in addition to MRI measures of lung and liver structure and function. The aim is to identify whether these measures are changed before and after starting ETI.
For the CF participants, to be enrolled in the study they must be homozygous or have at least 1 copy of the Phe508del mutation, which would mean they are eligible for ETI treatment.
The modified MRI protocol, taken from NCT04618185, has been shortened to include scans that are taken over three timepoints. These timepoints are determined around the ingestion of a test meal which has been previously described. Participants will arrive fasted before having a baseline scan. After their first scan they will eat their first standardised high fat meal followed by a second standardised high fat meal around 240 minutes after.
MRI scans to assess GI function will be taken at baseline (fasted), 240 minutes post ingestion of a breakfast meal (T240) and 300 minutes post breakfast (T300). Liver scans will also be taken at baseline and lung scans taken opportunistically at the T240 timepoint. In total, participants will be asked to remain in the MRI scanner for approximately 30 minutes at a time. For comfort, participants will be able to watch multimedia programmes through the use of a specially adapted television while in the scanner.
Participants will also be asked to complete validated gastrointestinal questionnaires throughout the study day and recall their GI symptoms over the previous 2 weeks and complete a 3 day food diary.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Children with CF aged 6 to 11 years. Must have at least one copy of the Phe508del mutation.
- •Healthy volunteers must have no previous history of gastrointestinal disease.
排除标准
- •Healthy volunteers with a history of gastrointestinal disease.
研究组 & 干预措施
Children with cystic fibrosis aged 6 to 11 years
Group of children who have at least one copy of Phe508del gene mutation and are eligible for starting on the modulator elexacaftor/ tezacaftor/ ivacaftor (ETI). This group will have baseline MRI scans before starting ETI and aiming to have subsequent scans post starting ETI (6 months to 1 year post starting ETI)
干预措施: Elexacaftor / Ivacaftor / Tezacaftor (Drug)
结局指标
主要结局
Delta Small Bowel Water (DeltaSBW)
时间窗: This a measure of the change in small bowel water between study time points 240 minutes and 300 minutes post first study meal.
This measurement relates to the postprandial fall in small bowel water content post second study meal. This will be adjusted to body surface area (mL/m2)
次要结局
- Small bowel water content(Will be measured at study timepoints: baseline, 240 minutes and 300 minutes and then area under the curve calculated using the three timepoints (L.min/m2))
- Liver volume(Measured during fasted, baseline MRI scan)
- Liver elastography(Measured during fasted, baseline MRI scan)
- Lung ventilation(Measured at 240 minute time point)