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

NCT ID: NCT06284577

Last Updated: 2024-06-13

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-02
• Study Completion Date: 2027-07-01

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.

Conditions

Cystic Fibrosis in Children

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Probiotics

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

Group Type: EXPERIMENTAL

Multistrain Probiotic

Intervention Type: DIETARY_SUPPLEMENT

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

Placebo

Participants will recieve maltodextrin daily for 6 months

Group Type: SHAM_COMPARATOR

Placebo - maltodextrin

Intervention Type: DIETARY_SUPPLEMENT

Participants will recieve placebo/ maltodextri daily for 6 months

Interventions

Multistrain Probiotic

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

Intervention Type: DIETARY_SUPPLEMENT

Placebo - maltodextrin

Participants will recieve placebo/ maltodextri daily for 6 months

Intervention Type: DIETARY_SUPPLEMENT

Eligibility Criteria

Inclusion Criteria

• 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

• 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

• Other CFTR modulators commenced the last 6 months before inclusion
• Use of probiotics or prebiotics last 2 months
• Current pulmonary exacerbation

• CFTR modulators commenced the last 6 months before inclusion
• Use of probiotics or prebiotics last 2 months
• Current pulmonary exacerbation

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Norwegian National Advisory Unit on Rare Disorders, Oslo University Hospital

UNKNOWN

Sponsor Role: collaborator

Oslo University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Magnhild L. P. Kolsgaard

Dietitian, PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Oslo University Hospital

Oslo, , Norway

Site Status: RECRUITING

Countries

Norway

Facility Contacts

Magnhild P Kolsgaard, PhD

Role: primary

uxpoma@ous-hf.no

0047 41514045

Camilla Styffe Sæland, Master

Role: backup

casael@ous-hf.no

004793257798

Other Identifiers

677186

Identifier Type: -

Identifier Source: org_study_id