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

NCT06284577 | Recruiting

• 1 other identifier: 677186
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis in Children

Keywords

Cystic Fibrosis; Children; Microbiota; Quality of life; Probiotic supplement; CFTR modulators; elexacaftor-tezacaftor-ivacaftor

Outcome Measures

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).

  0-6 months

Secondary Outcomes (2)

• Changes in microbiota

  0-6 months

• Changes in intestinal inflammation

  0-6 months

Study Arms (2)

Probiotics

EXPERIMENTAL

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

Dietary Supplement: Multistrain Probiotic

Placebo

SHAM COMPARATOR

Participants will recieve maltodextrin daily for 6 months

Dietary Supplement: Placebo - maltodextrin

Interventions

Multistrain Probiotic DIETARY_SUPPLEMENT

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

Probiotics

Placebo - maltodextrin DIETARY_SUPPLEMENT

Participants will recieve placebo/ maltodextri daily for 6 months

Placebo

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• Use of probiotics or prebiotics last 2 months
• Current pulmonary exacerbation
• 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
• Use of probiotics or prebiotics last 2 months
• Current pulmonary exacerbation

Sponsors & Collaborators

• Oslo University Hospital: lead
• Norwegian National Advisory Unit on Rare Disorders, Oslo University Hospital: collaborator

Study Sites (1)

Oslo University Hospital

Oslo, Norway

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, CARE PROVIDER
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dietitian, PhD

Study Record Dates

• First Submitted: February 12, 2024
• First Posted: February 29, 2024
• Study Start: January 2, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: June 13, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

NO (1)