E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Dilated airways which are characterized by chronic inflammation |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
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 |
Does Formoterol-beclomethasone inhalation in patients with bronchiectasis results in reduction of coughing |
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E.2.2 | Secondary objectives of the trial |
Improvement of quality of life reduction in exacerbations 24 hour sputum production (in mL); dyspnea score using Mahler Scale (basal dyspnea index and transition dyspnea index; safety and tolerability of long term formoterol-beclomethasone. local and systemic inflammation
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
· Age ≥ 18 years; · Symptomatic patient (wheezing, cough and dyspnoea); · Proven and documented diagnosis of BE by high resolution computed tomography · Stable pulmonary status as indicated by FEV1 (percent of predicted) ≥30% and <90% (post-bronchodilator); · Stable clinically phase (ie, subjects free from acute exacerbation for at least 6 weeks prior to the start of the study); · Stable regimen of standard treatment if used as chronic treatment for BE, at least for the past 4 weeks prior to screening. And/or macrolides if used as chronic treatment for BE at least for the past 6 months prior to screening; · Coughing on the majority of days.
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E.4 | Principal exclusion criteria |
- Possible asthma according to the definition of the Global Initiative for Asthma (GINA) with: o Positive bronchodilator reversibility test (increase in FEV1 of >12% and >200 mL from baseline, 10–15 minutes after 200–400 mcg salbutamol or equivalent) OR o Positive bronchial challenge test (fall in FEV1from baseline of ≥20% with standard doses of methacholine or histamine) - Known intolerance for ICS or LABA. - Other cardiopulmonary conditions (other than bronchiectasis) that could modify spirometric valeus. - Women who are pregnant, lactating, or in whom pregnancy cannot be excluded; - Cigarette smoking history of > 10 pack-years and/or current smokers; - Expected to die within 72 hours after enrolment. - Current ICS use |
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E.5 End points |
E.5.1 | Primary end point(s) |
Leicester cough questionnaire |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
· To evaluate the improvement of quality of life by Quality of Life Bronchiectasis (QOL-B) questionnaire’s respiratory symptom domain · To evaluate the efficacy of formoterol-beclomethasone on symptoms and pulmonary function (FEV1) and the frequency of exacerbation requiring an intervention with systemic antibiotics (oral/intravenous [i.v.]) in subjects with Bronchiectasis (BE). For this endpoint exacerbation events are defined as events with systemic antibiotic use and worsening of at least one sign/symptom; · To assess the 24 hour sputum production (in mL); · To assess the dyspnea score using the mMRC (Modified Medical Research Council) · To assess the safety and tolerability of long term formoterol-beclomethasone. . To asses local and systemic inflammation |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
E.6.5 | Efficacy | No |
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 | 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) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 2 |
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 | Yes |
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 | |
E.8.9.1 | In the Member State concerned days | |