Cystic Fibrosis
Conditions
Keywords
Pulmonary exacerbation, Inflammation, Intestinal microbiota
Brief summary
Cystic fibrosis (CF) is a complex systemic disease that mainly involves the respiratory and gastrointestinal (GI) tracts. The polymicrobial community composition of respiratory and GI tracts is influenced by both genetic and environmental factors. Children with CF may harbor an abnormal intestinal microflora, because of altered cystic fibrosis transmembrane conductance regulator (CFTR) function and heavy drug load (antibiotics, pancreatic enzymes and acid suppressors). The investigators have previously demonstrated that intestinal inflammation is highly frequent in CF children, being a major feature of intestinal involvement. In addition, specific probiotics significantly improved airway and GI inflammation in a preliminary trial. The investigators aim to characterize intestinal and respiratory microflora in CF patients and to investigate the effects of daily Lactobacillus GG (LGG) supplementation on both GI and airway microflora and the eventual relationship between probiotic assumption and clinical and inflammation markers. The investigators aim is to eventually improve the quality of life of CF patients, who often suffer from intestinal and respiratory progressive disease, through a non invasive intervention consisting in the supplementation of probiotic bacteria.
Interventions
DIETARY_SUPPLEMENTLactobacillus rhamnosus GG
Capsules containing lyophilized 6x10\^9 Colony Forming Units (CFU)/die LGG, (60mg) maltodextrin (163 mg), gelatine capsule (75 mg), magnesium stearate (2 mg) 1 cps/die for 12 months
DIETARY_SUPPLEMENTplacebo
Capsules containing maltodextrin (163 mg), gelatine capsule (75 mg), magnesium stearate (2 mg) 1 cps/die for 12 months
Sponsors
Federico II University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. A confirmed diagnosis of CF documented by sweat chloride test over 60 mmol/L and confirmed by genotype analysis with the presence of F508del/F508del or F508del/other 2. Boys and girls between 2 and 16 years of age 3. Clinical stability at enrolment, defined as no clinical evidence of acute exacerbation, no modifications in the therapeutic regimen and no hospitalization in the last 2 weeks 4. Pancreatic insufficiency 5. Basal Forced Expiratory Volume 1 second above 50% of predicted value
Exclusion criteria
1. Colonization of respiratory tract with Burkholderia cepacia spp. 2. Steroid therapy within one month before enrolment 3. Pregnancy and fertile women taking oral contraceptives 4. Parenteral or oral antibiotics therapy within 2 weeks before enrolment 5. Regular assumption of probiotics 6. Regular assumption of azythromycin
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in the incidence of pulmonary exacerbations from baseline to 12 months of treatment | every six months up to 18 months | The incidence of pulmonary exacerbation is assessed every six months. First evaluation from baseline to 6 months of observation. Second evaluation from randomization ( placebo/LGG) to 6 months of treatment and third evaluation after 12 months of treatment |
| Change of intestinal inflammation from baseline to 12 months of treatment | every six months up to 18 months | Assessment of intestinal inflammation is performed four times. First time at enrollment, second time at the end of six months of observation. Third time after six months of treatment and fourth time after 12 months of treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the incidence of hospital admission from baseline to 12 months of treatment | every six month up to 18 months | The incidence of hospital admission is assessed every six months. First evaluation from baseline to 6 months of observation. Second evaluation from randomization ( placebo/LGG) to 6 months of treatment and third evaluation after 12 months of treatment |
| change in pulmonary function from baseline to 12 months of treatment (measured by Forced Expiratory Volume 1 sec (FEV1)) | every six months up to 18 months | Assessment of pulmonary function is performed four times. First time at enrollment, second time at the end of six months of observation. Third time after six months of treatment and fourth time after 12 months of treatment. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change in systemic inflammation from baseline to 12 months of treatment | At baseline and after 12 months of treatment | Assessment of intestinal microflora composition is performed 2 times. First time at randomization (placebo/LGG), second time at the end of 12 months of treatment. |
| Change of intestinal microflora composition from baseline to 12 months of treatment | baseline and 12 months after treatment | Assessment of intestinal microflora composition is performed 2 times. First time at randomization (placebo/LGG), second time at the end of 12 months of treatment. |
| Change in the incidence of abdominal pain episodes from baseline to 12 months of treatment | every six months up to 18 months | The incidence of abdominal pain episodes is assessed every six months. First evaluation from baseline to 6 months of observation. Second evaluation from randomization ( placebo/LGG) to 6 months of treatment and third evaluation after 12 months of treatment |
Countries
Italy
Outcome results
None listed