Improved Quality of Life in Children With Intestinal Failure

Improved Quality of Life in Children With Intestinal Failure - a Randomised Intervention Trial

Children with intestinal failure have a lack of tolerance for food in the intestine. The children are dependent on intravenous nutrition over a long period of time period to ensure growth and development. The condition is characterized by bacterial overgrowth in the intestine, with nausea, vomiting, diarrhea and flatulence as physical symptoms. Mental health is affected in the form of lower quality of life, lack of school participation and less social contact with peers. The investigators own (unpublished) data show that children with intestinal failure have a lower quality of life than healthy people. Standard treatment is antibiotics, but the effect of these is short-lived, and many must have repeated courses. Prebiotics are indigestible carbohydrates (fiber) in foods that positively affect the bacterial flora and promote intestinal health. In this project the investigators want to see if supply of prebiotics can change the bacterial balance, reduce symptoms of bacterial overgrowth and increase quality of life. The study is unique, as Prebiotics have not previously been used in the treatment of intestinal failure. If successful, it can pave the way for a new and better treatment method that can potentially be transferred to other conditions with imbalance in the intestinal flora. The study is a randomized intervention study and is consist of two phases. In phase 1, the effect of 4 weeks of open intervention with prebiotics is studied to establish so-called "proof of concept". Data from phase 1 are used to look at connections between the composition of intestinal flora, nutritional status and bowel function. The intervention involves the use of a prebiotic product (Stimulance, Nutricia), which is added to childs regular food. In phase 2, patients are randomized into two groups. One group will continue with the product for 6 months, while the other group does not receive prebiotics.

Study Overview

• Status: Completed
• Conditions: Short Bowel Syndrome; Intestinal Pseudo-Obstruction; Bacterial Overgrowth; Malabsorption Syndrome Autoimmune Enteropathy
• Intervention / Treatment: Dietary supplement: Stimulance

Detailed Description

Quality of life, gastrointestinal symptoms and family impact are measured using validated forms for four weeks before intervention phase 1, at start-up and after phase 1, as well as after phase 2. Stool samples are collected at the same time points. Weight, height is measured and blood tests are collected at the start and end of phases 1 and 2. Data on diet and nutritional treatment are collected at the start and end of the two phases. The stool samples are analyzed for short fatty acids (marker for good intestinal health), the composition of the intestinal flora and inflammation markers. Blood samples are analyzed for infection markers, intestinal health markers and nutritional markers. Nutritional data are analyzed for nutrient content and the dependence on nutritional support is calculated.

The investigators expect that the study will lead to an improved quality of life for the patient group through increased tolerance to food in the intestine, reduced antibiotic use and reduction of gastrointestinal symptoms. The project is patient-oriented.

• Study Type: Interventional
• Enrollment (Actual): 55
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Rut Anne Thomassen; Phone Number: + 4722118765; Email: uxruom@ous-hf.no
• Study Contact Backup: Name: Anne Charlotte Brun, MD PhD; Phone Number: +4722118765; Email: bruan@ous-hf.no

Study Locations

• Norway
  • Oslo, Norway, 0424
    • Oslo University Hopspital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria are:

• Diagnosed with congenital malformations or diseases requiring intestinal surgery leading to short bowel syndrome or
• Diagnosed with conditions and diseases leading to intestinal failure e.g pseudo obstruction.

And Treated with parenteral nutrition for minimum 60 days within a 74 day period.

Exclusion criteria:

• Children in need of temporary advanced nutrition intervention due to illness, e.g. infections.
• Children with temporary malfunctioning gut due to advanced medical treatment, for example cancer treatment or transplantation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Stimulance; 4 weeks intervention with Stimulance for all participants	Dietary supplement: Stimulance; Intervention with Prebiotic supplement
Experimental: Stimulance 6 months; 6 months intervention	Dietary supplement: Stimulance; Intervention with Prebiotic supplement
No Intervention: No intervention; 6 months control group

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Quality of life scores before and after treatment with prebiotics	Changes in Pediatric quality of life inventory scores on an analog scale from 0-100 where higher scores means better outcome	6 months
Changes in gastro intestinal symptoms scores before and after treatment with prebiotics	Pediatric quality of life inventory Gastro intestinal symptom scale scores on an analog scale from 0-100 where higher scores means better outcome	6 months
Changes in familt impact scores before and after treatment with prebiotics	Pediatric quality of life inventory Family Impact Module scores on an analog scale from 0-100 where higher scores means better outcome	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Microbiota	Measured by Short Chain Fatty Acids and microbiota composition	6 months

Collaborators and Investigators

• Sponsor: Oslo University Hospital
• Collaborators: University of Oslo
• Investigators: Principal Investigator: Rut Anne Thomassen, Oslo University Hospital; Study Director: Anne Charlotte Brun, MD PhD, Oslo University Hospital; Study Chair: Christine Henriksen, PhD, University of Oslo

Study record dates

Study Major Dates

• Study Start (Actual): September 2, 2021
• Primary Completion (Actual): December 2, 2023
• Study Completion (Actual): December 15, 2023

Study Registration Dates

• First Submitted: June 22, 2021
• First Submitted That Met QC Criteria: July 26, 2021
• First Posted (Actual): July 28, 2021

Study Record Updates

• Last Update Posted (Estimated): April 26, 2024
• Last Update Submitted That Met QC Criteria: April 25, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Metabolic Diseases; Postoperative Complications; Disease; Gastrointestinal Diseases; Intestinal Diseases; Intestinal Obstruction; Ileus; Syndrome; Malabsorption Syndromes; Short Bowel Syndrome; Intestinal Pseudo-Obstruction; Intestinal Failure
• Other Study ID Numbers: 170851

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