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

June 12, 2024 updated by: Magnhild L. P. Kolsgaard, Oslo University Hospital

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Norway
      • Oslo, Norway
        • Recruiting
        • Oslo University Hospital
        • Contact:
        • Contact:
          • Camilla Styffe Sæland, Master
          • Phone Number: 004793257798
          • Email: casael@ous-hf.no
        • Principal Investigator:
          • Magnhild P Kolsgaard, PhD
        • Sub-Investigator:
          • Camilla Styffe Sæland, Master
        • Sub-Investigator:
          • Linh Dieu Ngo, Master

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Probiotics
Participants will recieve a multi-strain probiotic daily for 6 months
Dietary supplement: Multistrain Probiotic
Participants will recieve a multi-strain probiotic daily for 6 months
Sham Comparator: Placebo
Participants will recieve maltodextrin daily for 6 months
Dietary supplement: Placebo - maltodextrin
Participants will recieve placebo/ maltodextri daily for 6 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in GI related QoL using the questionnaire PedsQL GI
Time Frame: 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).
0-6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in microbiota
Time Frame: 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.
0-6 months
Changes in intestinal inflammation
Time Frame: 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.
0-6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Oslo University Hospital

Collaborators

Norwegian National Advisory Unit on Rare Disorders, Oslo University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 2, 2024

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

July 1, 2027

Study Registration Dates

First Submitted

February 12, 2024

First Submitted That Met QC Criteria

February 21, 2024

First Posted (Actual)

February 29, 2024

Study Record Updates

Last Update Posted (Actual)

June 13, 2024

Last Update Submitted That Met QC Criteria

June 12, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 677186

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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