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 | 7.0 | E.1.2 | Level | PT | E.1.2 | Classification code | 10011762 | |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial | To assess the safety of Depelestat in CF patients with moderate pulmonary disease, particularly regarding the PFT evolution on treatment, compared to placebo. | |
E.2.2 | Secondary objectives of the trial | To compare two doses of Depelestat in terms of pharmacodynamic effect, by measuring change in hNE activity in sputum during the treatment period and the post-treatment period by comparison with the pre-treatment period. | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria | Male or female patient above 6 years of age Suffering from CF with a sweat chloride concentration above 60 mmol/L and/or proved by genotyping With a negative pregnancy test if female of childbearing potential, and taking adequate contraception precautions during the duration of the study With pulmonary disease of moderate severity defined as: less than 80% but at least 30% FEV1 predicted values at each baseline measurement (V-2, V-1 and V1 before treatment) With pulmonary disease in stable stage defined as: -maximal FEV1 variation by 10 %, between the 3 baseline values obtained during the pre-treatment period and on Day 1 before the first drug administration -no acute pulmonary exacerbation (APE) during the 6 previous weeks before treatment start (at least 4 weeks before V-2) Able to perform PFT (spirometry) with reproducible values | |
E.4 | Principal exclusion criteria | Suffering from a severe pulmonary disease : FEV <30% and /or FVC <40% of predicted values for any baseline measurement at V-2, -1 and V1 before nebulisation Having suffered from an acute exacerbation of pulmonary disease according to the definition of Fuchs with IV antibiotics treatment, within 6 weeks before start of treatment Having been admitted to hospital for treatment of their disease during the 6 weeks before start of treatment Suffering from allergic bronchopulmonary aspergillosis (ABPA) with related clinical signs as bronchospams or asthmatic manifestations Presenting an identified bronchial hyperresponsiveness with an history of asthma with episodes of wheezing Having an history of significant haemoptysis (except hemoptoïc expectoration) Presenting a bronchopulmonary colonisation by Bukholderia Cepacia, according to the last sputum bacterial examination Having changed their chronic therapy, including chest physiotherapy and drugs, less than 6 weeks (2 cycles for inhaled Tobramycin) before start of treatment Routine IV antibiotic treatment : patients receiving IV antibiotics at planned regular interval of time. Chronic use of oral corticosteroids (to be stopped at least 6 weeks before start of treatment) Taking a treatment with mucolytic drugs containing N-acetyl cystein (to be stopped at least 6 weeks before start of treatment | |
E.5 End points |
E.5.1 | Primary end point(s) | FEV1 % predicted relative change from baseline to week 8 | |
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 | Yes |
E.6.5 | Efficacy | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned | |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | The trial ends at the last visit of the last subject. | |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 12 |