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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
40
Participants will recieve a multi-strain probiotic daily for 6 months
Participants will recieve placebo/ maltodextri daily for 6 months
Oslo University Hospital
Oslo, Norway
RECRUITINGChanges in GI related QoL using the questionnaire PedsQL GI
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).
Time frame: 0-6 months
Changes in microbiota
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.
Time frame: 0-6 months
Changes in intestinal inflammation
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.
Time frame: 0-6 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.