E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10017947 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To test the hypotheses that interventions to restore intestinal microflora, such as probiotic administration, may decrease intestinal and extraintestinal inflammation, ultimately improving CF quality of life and clinical course. |
|
E.2.2 | Secondary objectives of the trial |
To test the hypotheses that: - Intestinal microflora is also modified and is characterized by bacterial species that are detected selectively or more frequently in CF, whereas other species that are detected in non CF subjects are not found in CF. - Intestinal inflammation is a very frequent feature of CF, possibly as a consequence of changes in intestinal microflora. - Intestinal inflammation contributes to extraintestinal inflammation, including the respiratory tract. - intestinal microenvironment is modified in cystic fibrosis (CF). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- 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; - boys and girls between 2 and 16 years of age; - in stable clinical condition at enrolment; - with pancreatic insufficiency; - with basal FEV1 above 50% of predicted value |
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E.4 | Principal exclusion criteria |
- pregnancy and fertile women taking oral contraceptives; - pancreatic sufficiency; - oral steroid therapy within one month before enrolment; - parenteral or oral antibiotics therapy within 2 weeks before enrolment ; - regular assumption of probiotics; - regular use of azythromicin |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- Incidence and severity of intestinal inflammation, as measured by fecal calprotectin concentration and by rectal nitric oxide concentration. -Quality of life (assessed by the revised Italian version of a questionnaire assessing Quality of Life (CFQoL). -Composition of colonic microflora before and after LGG administration and antibiotic therapy -Incidence, duration and severity of pulmonary exacerbations, as judged by the number of episodes and number of days free of intravenous antibiotic treatment. -Pulmonary function, as measured by FEV1 and FVC. -Hospital admissions. -Incidence, duration and severity of abdominal pain episodes. -Nutritional status -Systemic inflammation. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |