E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult patient with cystic fibrosis |
Pazienti adulti con fibrosi cistica |
|
E.1.1.1 | Medical condition in easily understood language |
Adult patient with cystic fibrosis |
Pazienti adulti con fibrosi cistica |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 |
Evaluation of treatment efficacy in reducing bacterial load in sputum at visit 2 (day 7-9), visit 3 (day 14-16) and visit 4 (day 28-30) |
Primario: valutazione degli effetti del trattamento sulla carica batterica dell'espettorato alla visita 2 (giorno 7-9), visita 3 (giorno 14-16) e visita 4 (giorno 28-30) |
|
E.2.2 | Secondary objectives of the trial |
Evaluation of treatment efficacy in reducing inflammatory serum biomarkers at visit 2 (day 7-9), visit 3 (day 14-16) and visit 4 (day 28-30) |
Valutazione degli effetti del trattamento sui biomarcatori infiammatori sierici alla visita 2 (giorno 7-9), visita 3 (giorno 14-16) e visita 4 (giorno 28-30) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Out patient, both sexes, age 18-45. - diagnosys of cystic fibrosis - FEV1 >50% predicted - availability of sputum sample for microbiology - chest X ray negative for pneumonia and active tubercolosis - signed informed consent |
Pazienti ambulatoriali, maschi o femmine, con età compresa tra 18 e 45 anni. 2. Diagnosi di Fibrosi cistica 3. FEV1 >50% del predetto. 4. Disponibilità di un campione di secrezioni bronchiali per l’esame microbiologico. 5. Radiografia del torace negativo per polmonite e tubercolosi attiva. 6. Consenso informato scritto, datato e firmato dal paziente. |
|
E.4 | Principal exclusion criteria |
Hypersensibility or allergy to the study drug -use of systemic steroids (less than 2 weeks from the screening) - Pregnancy or lactation - partecipation in clinical trial with sperimental drug 4 weeks before enrollment |
Ipersensibilità o allergia ai farmaci in studio. 2. Uso di steroidi sistemici (trattamento da meno di 2 settimane prima dell’inizio dello studio), a meno che il paziente non sia in terapia cronica. L’uso di steroidi topici nasali è consentito per i primi tre giorni dello studio. 3. Gravidanza o allattamento. 4. Trattamento con farmaci sperimentali nelle 4 settimane precedenti l’arruolamento |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Microbiological efficacy |
Efficacia microbiologica |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Reduction in inflammatory serum biomarkers |
Riduzione biomarcatori infiammatori sierici |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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 | No |
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
|
last administration of study drug |
ultima somministrazione del farmaco |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |