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Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis

ConditionCystic Fibrosis in Children
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age2-18
SponsorOslo University Hospital

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

Other CFTR modulators commenced the last 6 months before inclusion

Use of probiotics or prebiotics last 2 months

Current pulmonary exacerbation

Age 3-18 years

Trial design

Design model

Parallel

Treatments tested in this trial

  • Multistrain Probiotic

    Dietary supplement

    Participants will recieve a multi-strain probiotic daily for 6 months

  • Placebo - maltodextrin

    Dietary supplement

    Participants will recieve placebo/ maltodextri daily for 6 months

Treatment groups

40 Participants
are divided into 2 treatment groups
Group A: ProbioticsExperimental treatment 1 intervention
Group B: PlaceboSham comparator 1 intervention

Trial outcomes

Primary outcomes

1

Changes 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

Secondary outcomes

1

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
2

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

Other outcomes

Sponsors and contacts

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Oslo University Hospital

Lead sponsor

Norwegian National Adivisory Unit on Rare Disorders

Collaborator

Source ClinicalTrials.gov
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