E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Asthma is a chronic inflammatory disorder in which many cells play a role, in particular mast cells, eosinophils and T lymphocytes. In susceptible individuals this inflammation causes recurrent episodes of wheezing, breathlessness, chest tightness and cough. These symptoms are usually associated with widespread but variable airflow limitation that is at least partly reversible either spontaneously or with treatment. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003553 |
E.1.2 | Term | Asthma |
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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 investigate the effect of treatment with nebulized rhDNase on pulmonary function in children with persistent asthma and persistent peripheral airflow limitation. |
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E.2.2 | Secondary objectives of the trial |
To investigate the effect of treatment with nebulized rhDNase on lung clearance index (LCI), FENO values and symptom scores in children with asthma and persistent peripheral airway obstruction. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Age 6 – 18 years; - Asthma diagnosed according to GINA guidelines; - Attending the outpatient clinic for at least one year; - Treatment with at least 400 mg/day inhaled Budesonide or equivalent (dose constant for at least 6 months) and bronchodilators as needed or daily; - Clinically stable asthma while using a constant dose of ICS; - Ability to perform lung function tests (assessed by trained lung function technician); - Persistent peripheral airways obstruction as assessed by pulmonary function testing, defined as: * Dissociation between FVC and FEF75 values: FEF75 at least 20% (absolute % predicted) lower than FVC. - FVC within normal limits (for this study defined as FVC > 80% pred).
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E.4 | Principal exclusion criteria |
- Asthma exacerbation with hospital admission in last 3 months; - Intensive Care Unit (ICU) admission for asthma in the last year; - Current respiratory tract infection; - Inability to follow instructions of the investigator; - Inability to inhale rhDNase; - Concomitant medical conditions that effect inhaled treatment (e.g. cleft palate, severe malacia); - Neuromuscular disease; - Smoking.
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint will be the change in FEF75 as a result of treatment. FEF75 is the most suitable endpoint since it is sensitive to peripheral airways obstruction. |
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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 | 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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | 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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The end of the study is defined as the last patient’s last visit. |
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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 | 6 |
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 | 6 |