Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Multistrain Probiotic, Placebo - maltodextrin.
- Who it may be relevant to
- Registry conditions: Cystic Fibrosis in Children. Basic parameters: 2 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Norway
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Probiotic Supplementation, Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
Overview
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.
Interventions
- Dietary supplement Multistrain Probiotic
Participants will recieve a multi-strain probiotic daily for 6 months - Dietary supplement Placebo - maltodextrin
Participants will recieve placebo/ maltodextri daily for 6 months
Primary outcome measures
- Changes in GI related QoL using the questionnaire PedsQL GI [Time frame: 0-6 months]
Secondary outcome measures (2)
- Changes in microbiota [Time frame: 0-6 months]
- Changes in intestinal inflammation [Time frame: 0-6 months]
Eligibility criteria
Inclusion criteria for WP1:
- 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 2024.
- Included in the Norwegian CF Register and consented to participation in CF general research biobank
Exclusion criteria for WP1:
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
Norway · 1 center
- Oslo University Hospital — Oslo
Identifiers
NCT: NCT06284577 · 677186