Polyethylene Glycol and Intestinal Inflammation in Cystic Fibrosis

NCT04458129 | Terminated Phase 2

• 2 other identifiers: CHUBX 2019/252019-004958-29
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

The main objective of the study is to evaluate the effectiveness of polyethylene glycol treatment on intestinal inflammation in children with cystic fibrosis. In this test, a method adapted from the Fleming one-step scheme will be used. The success rate is measured by the proportion of patients with fecal calprotectin levels \< 250 µg/g at 3 months after treatment initiation.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; intestinal inflammation; fecal calprotectin; microbiota; children; polyethylene glycol

Outcome Measures

Primary Outcomes (1)

• Impact of a 3-month polyethylene glycol treatment on intestinal inflammation assessed by measurement of fecal calprotectin (µg / g) by an ELISA biological test

  proportion of patients with fecal calprotectin \<250 µg / g measured by an ELISA test 3 months after initiation of treatment with polyethylene glycol, testifying to the absence of intestinal inflammation or slight inflammation.

  3 months after the inclusion visit i.e. 3 months after the initiation of treatment with polyethylene glycol

Secondary Outcomes (7)

• Evolution of the digestive inflammatory response, assessed by measurement of fecal calprotectin (µg / g)

  Change from Baseline fecal calprotectin measurement at 3 months

• Evolution of the digestive inflammatory response, assessed by Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response

  Change from Baseline measurement at 3 months

• Evolution of the digestive symptoms, assessed by the JenAbdomen CF-score

  Change from Baseline measurement at 3 months

• Evolution of the quality of life scores, assessed by the CFQ-R questionnaire

  Change from Baseline measurement at 3 months

• Evolution of the composition of the bacterial intestinal microbiota

  Change from Baseline measurement at 3 months

Study Arms (1)

Polyethylene glycol treatment

EXPERIMENTAL

Study participants will take a 3-month laxative treatment with polyethylene glycol for 3 months.

Drug: Treatment with polyethylene glycol (Macrogol 4000)

Interventions

Treatment with polyethylene glycol (Macrogol 4000) DRUG

3-month treatment with polyethylene glycol (Macrogol 4000), powder for oral solution, in 4g and 10g sachets. Dosage of 0.7 g/kg/day, with a maximum dose of 20 g/day.

Polyethylene glycol treatment

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age ≥ 4 years old and \<18 years old ;
• Patient with cystic fibrosis (sweat test \> 60 mmol/l and/or molecular biology identifying mutations in the CFTR gene) with associated pancreatic insufficiency (fecal elastase \<100 µg/g);
• With a rapid calprotectin assay result via the IBDoc test (Bühlmann®) superior or equal to 250 µg/g;
• Person affiliated or benefiting from a social security scheme;
• Free, informed and written consent signed by the holders of parental authority and the investigator before any examination required by the research and oral and/or written consent by the participant (depending on his/her age).

You may not qualify if:

• Ongoing processing that can modulate the functionality of the CFTR (such as lumacaftor-ivacaftor protein therapy);
• Cure of oral or intravenous antibiotics or antifungals in the month preceding the collection of samples;
• Transplanted patient (on immunosuppressants);
• Patient with IBD or celiac disease;
• Patient with digestive perforation or risk of digestive perforation;
• Patient with ileus or suspicion of intestinal obstruction, symptomatic stenosis;
• History of hypersensitivity to macrogol or any of the excipients
• Holders of parental authority enjoying judicial protection.

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

Study Sites (1)

CHU de Bordeaux

Bordeaux, 33000, France

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Therapeutics; Polyethylene Glycols

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

Intervention Hierarchy (Ancestors)

Ethylene Glycols; Glycols; Alcohols; Organic Chemicals; Polymers; Macromolecular Substances; Biomedical and Dental Materials; Manufactured Materials; Technology, Industry, and Agriculture

Study Officials

• Marie M MITTAINE, MD

  CHU de Toulouse - Hôpital des Enfants - Centre de Ressources et de Compétences de la Mucoviscidose (CRCM) pédiatrique

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Bi-centric, non-comparative, prospective, Phase II trial according to a Fleming scheme.

Study Record Dates

• First Submitted: June 30, 2020
• First Posted: July 7, 2020
• Study Start: September 29, 2020
• Primary Completion: April 8, 2022
• Study Completion: July 8, 2022
• Last Updated: June 22, 2026

Record last verified: 2020-06

Locations

FR (1)