Cystic fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: FORLAX 4 g, poudre pour solution buvable en sachet
FORLAX 10 g, poudre pour solution buvable en sachet-dose
Pharmaceutical Form: Powder for oral solution in sachet
Sponsors
CHU de Bordeaux
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age = 4 years old and 60 mmol/l and/or molecular biology identifying mutations in the CFTR gene) with associated pancreatic insufficiency (fecal elastase =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Ongoing processing that can modulate the functionality of the CFTR (such as lumacaftor-ivacaftor protein therapy); - Patient already on polyethylene glycol or other laxative within 3 months before the inclusion visit; - Patient with diarrhea at inclusion (diarrhea will be defined as the presence of 3 or more stools / day in the 7 days prior to the inclusion visit); - Acute viral or bacterial diarrhea in the month prior to the inclusion visit (associated with fever); - Cure of oral or intravenous antibiotics or antifungals in the month preceding the collection of samples; - Change in background treatment in the month prior to the inclusion visit (oral or inhaled corticosteroid therapy, azithromycin, inhaled antibiotic therapy, inhaled antifungal agent, proton pump inhibitors); - Taking probiotics in the month before the inclusion visit; - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Study the impact of a 3-month polyethylene glycol treatment on intestinal inflammation associated with cystic fibrosis in children aged 4 to 18 years, as measured by the fecal calprotectin assay.;Secondary Objective: Study the impact of a 3-month treatment with polyethylene glycol: - On digestive symptoms and quality of life, - On the intestinal microbiota (bacterial and fungal) Study the co-evolution of inflammation between the gut and lungs ("the gut-lung axis") during the 3 months of polyethylene glycol treatment. ;Primary end point(s): proportion of patients with faecal 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.;Timepoint(s) of evaluation of this end point: 3 months after initiation of treatment with polyethylene glycol | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The evolution between the initiation of treatment (D0) and 3 months of treatment with polyethylene glycol (M3): - Digestive symptoms and quality of life, assessed respectively by the validated questionnaire JenAwinen CF score and CFQ-R; - The digestive inflammatory response, assessed by: - The dosage of fecal calprotectin, interpreted as a quantitative variable (measured by an ELISA test), - Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response, using NanoString Technology and the nCounters® PanCancer Immune Profiling Panel and the production of the main cyto / chemokines produced by the digestive mucosa in the stool samples .. - The composition of the bacterial and fungal intestinal microbiota, with: - High-speed sequencing and phylogenetic assignment of the bacterial flora (on MiSeq, from Illumina®) - High-speed sequencing of the region and phylogenetic affectation of the fungal flora (on MiSeq, from Illumina®) - Confirmation by quantitative PCR of the species or genera whose relative abundance will be significantly modified - Calculation of the MD-Index dysbiosis score for the intestinal microbiota. - Pulmonary inflammation, assessed by the dosage of calprotectin in the sputum. ;Timepoint(s) of evaluation of this end point: at the initiation of treatment (D0) and at 3 months of treatment with polyethylene glycol (M3): | — |
Countries
France
Contacts
Public ContactClinical Trials Manager
CHU de Bordeaux
Outcome results
None listed