Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
- Conditions
- Cystic Fibrosis in Children
- Interventions
- Dietary Supplement: Multistrain ProbioticDietary Supplement: Placebo - maltodextrin
- Registration Number
- NCT06284577
- Lead Sponsor
- Oslo University Hospital
- Brief Summary
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.
- Detailed Description
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
In WP1 participants will during routine examination before starting treatment with ETI be asked to deliver stool samples, and fill in QoL questionnaires. In WP2 participants will be randomized to intervention with probiotics or placebo, and the same parameters as in WP1 will also be collected.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 40
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Probiotics Multistrain Probiotic Participants will recieve a multi-strain probiotic daily for 6 months Placebo Placebo - maltodextrin Participants will recieve maltodextrin daily for 6 months
- Primary Outcome Measures
Name Time Method Changes in GI related QoL using the questionnaire PedsQL GI 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).
- Secondary Outcome Measures
Name Time Method Changes in microbiota 0-6 months Stool samples for microbiota will be collected using a collection kit provided to the participants at recruitment, and then after 6 month of ETI treamtment (WP1), or probiotic/placebo (WP2). Microbiota analysis will be done using 16s rRNA amplicon sequencing and reduced metagenome sequencing.
Changes in intestinal inflammation 0-6 months Stool samples for gut inflammation markers will be collected using a collection kit provided to the participants at recruitment and after 6 months of ETI treamtment (WP1), or probiotic/placebo (WP2. Levels of calprotectin, neopterin and myeloperoxidase will be measured.
Trial Locations
- Locations (1)
Oslo University Hospital
🇳🇴Oslo, Norway