Probiotic Supplementation, Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Changes in GI related QoL using the questionnaire PedsQL GI
研究概览
简要总结
The aim of the present study is to investigate the effect of probiotic supplementation on GI related quality of life, through a randomised placebo-controlled clinical trial. Moreover, the invetigators wish to study CF microbiota and intestinal inflammation in the setting of probiotic supplementation and newly started treatment with a highly effective CF-specific treatment, elexacaftor-tezacaftor-ivacaftor (ETI). The proposed project has the potential to increase QoL and decrease GI morbidity in children with CF. If successful, the results of this study can contribute to alter the care of CF patients by including supplementation of probiotics in routine CF care. Morever, the study can provide much needed insights to GI microbiota and inflammation in pediatric CF patients.
详细描述
The project targets pediatric patients with cystic fibrosis (CF), and has to work packages (WP). WP1 is an observational study, and WP2 is a randomised placebo-controlled clinical trial. The goal of the study is to investigate the effect of probiotics on pediatric CF patients' quality of life (QoL). Moreover the investigators wish to explore effects of both a highly effective CFTR modulator and probiotics on gut microbiota and intestinal inflammation.
The primary question it aims to answer are:
• Can probiotics improve GI related QoL in children with CF?
Secondary aims are to:
- Investigate GI microbiota and GI inflammation before and after commencement of the highly effective triple-combination elexacaftor-tezacaftor-ivacaftor (ETI)
- Explore GI microbiota before and after treatment with probiotics vs. placebo
- Study intestinal inflammation before and after treatment with probiotics vs. placebo
- Examine body composition and its relation to lung function
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
- Double (Participant, Care Provider)
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •CFTR mutations eligible for treatment with ETI
- •Age 2-18 years. Majority of patients will be 2-6 years of age as ETI was approved from 6 years of age in 2022, and will be available for children above 2 years from
- •Included in the Norwegian CF Register and consented to participation in CF general research biobank
排除标准
- •Other CFTR modulators commenced the last 6 months before inclusion
- •Use of probiotics or prebiotics last 2 months
- •Current pulmonary exacerbation
- •Inclusion criteria for WP2:
- •Age 3-18 years
- •CFTR modulator treatment naïve or treated with CFTR modulator for at least 6 months
- •Included in the Norwegian CF Register and consented to participation in CF general research biobank
- •Exclusion criteria for WP2:
- •CFTR modulators commenced the last 6 months before inclusion
- •Use of probiotics or prebiotics last 2 months
- •Current pulmonary exacerbation
结局指标
主要结局
Changes in GI related QoL using the questionnaire PedsQL GI
时间窗: 0-6 months
PEDsQL GI has been validated for use in the CF population. Participants will be scored before and 6 months after treatment start with ETI (WP2), and before and after 6 months treatment with probiotics vs placebo (WP2).
次要结局
- Changes in microbiota(0-6 months)
- Changes in intestinal inflammation(0-6 months)
研究者
Magnhild L. P. Kolsgaard
Dietitian, PhD
Oslo University Hospital