E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
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 |
The study has two co-primary objectives: (1) to assess whether the effects of Bronchitol are beneficial to the quality of life in subjects with bronchiectasis using the St George’s Respiratory Questionnaire; and (2) to assess the impact of Bronchitol on mucus clearance (24 hour sputum sample)
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E.2.2 | Secondary objectives of the trial |
Efficacy: To assess the impact of Bronchitol on: (1) bronchiectasis symptoms (2) cough severity (3) exercise capacity (4) lung function, including gas transfer (5) antibiotic use (6) bronchial wall thickening and inflammation and (7) peripheral airway function Safety: To demonstrate the safety profile of Bronchitol (adverse events, haematology, biochemistry, sputum microbiology)
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
(1) Evidence of confirmed diagnosis of (non CF) bronchiectasis by HRCT (2) Age: 15 – 80 years inclusive (3) FEV1 50% - 80% predicted and ≥1.0L (4) Chronic cough (defined as persistently troublesome cough present on the majority of days in the 3 months prior to enrolment) (5) Chronic sputum production of >10 mL per day on the majority of days in the 3 months prior to study entry (6) Chronic chest congestion ( defined as chronic excessive accumulation of mucus in the lungs) (7) Ability to perform all the techniques necessary to measure lung function, and (8) Stable clinical condition at the time of, and for a period of 14 days prior to recruitment; defined as lack of: (a) change in techniques for sputum clearance (b) change in sputum production (volume, colour, consistency) (c) new or increased haemoptysis (d) increased cough (e) worsening dyspnoea (f) increased malaise, fatigue or lethargy (g) reduction in exercise tolerance (h) fever (g) increase in weight loss or anorexia (h) sinus pain/tenderness or change in sinus discharge (i) gastrointestinal symptoms such as diarrhoea, constipation, nausea, vomiting or (j) additional antibiotics (routine antibiotics permitted) |
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E.4 | Principal exclusion criteria |
(1) Investigators, site personnel directly affiliated with this study, and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted (2) Bronchiectasis as a consequence of cystic fibrosis or focal endobronchial lesion (3) “Terminally ill” patients or those listed for transplantation (4) Use of hypertonic saline or mucolytic pharmacological agents concurrently or in the 4 weeks prior to study entry (5) Patients previously enrollment in a clinical trial using Bronchitol that was administered for more than a day (6) Significant episode of haemoptysis (>60 mL) in the previous 6 months (7) Exacerbation for which intravenous antibiotics were prescribed in the 4 weeks prior to study entry (8) Presence of airway hyperresponsiveness as defined by a positive Aridol challenge (9) Smoking history of >10 pack years or if more than 1 cigarette smoked per week within the previous 3 months (10) Likeliness of developing bronchoconstriction in response to Bronchitol in the opinion of the investigator (11) Myocardial Infarction in the three months prior to enrolment (12) Cerebral vascular accident in the three months prior to enrolment (13) Major ocular surgery in the three months prior to enrolment (14) Major abdominal, chest or brain surgery in the three months prior to enrolment (15) Presence of cerebral, aortic or abdominal aneurysm (16) Active tuberculosis (17) Active malignancy including melanoma (other skin carcinomas and remissions exempted) (18) Females who are breast feeding or pregnant or plan to become pregnant during the study (19) At risk females unwilling to use appropriate contraception to prevent pregnancy during the study (20) Patients participating in another investigative drug study, parallel to, or within 4 weeks of study entry (21) Known intolerance to mannitol or β-agonists (22) Uncontrolled hypertension – systolic BP > 190 and or diastolic BP > 100 or (23) Patient has a condition or is in a situation which in the Investigator’s opinion may put the subject at significant risk, may confound results or may interfere significantly with participation in the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Assessment of the effects of Bronchitol on quality of life as measured by a change in the score of the St George’s Respiratory Questionnaire; (2) Assessment of the impact of Bronchitol on mucus clearance (24 hour sputum sample) |
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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 | Yes |
E.6.4 | Safety | Yes |
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 | 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) | 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 | 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 | No |
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 |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |