E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Wheezing in infants and young children (6-24 months old) |
Piepende ademhaling bij zuigelingen en jonge kinderen (6-24 maanden oud) |
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E.1.1.1 | Medical condition in easily understood language |
Wheeze in young children. |
Piepende ademhaling bij jonge kinderen |
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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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
What is the (cost-)effectiveness of salbutamol inhalations (4x200μg for 7 days) versus placebo in children aged 6-24 months presenting to their primary care physician with wheezing? |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Otherwise healthy children aged 6-24 months presenting to their primary care physician because of wheezing as confirmed by clinical examination (chest auscultation) and a baseline score of 7 or higher on a parent-reported respiratory symptom score. |
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E.4 | Principal exclusion criteria |
Prematurity, major congenital malformations, pre-existing pulmonary disease as diagnosed by a paediatrician, continuous use of inhalation medication, physician visit because of wheezing in previous two weeks, or use of asthma medication in the previous two weeks, wheezing as a result of upper airway obstruction (i.e. laryngitis subglottica/pseudocroup) and severe illness requiring inhalation medication, prescription of antibiotics, or hospital referral; during the consultation of inclusion. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome is the course of the mean parent reported respiratory symptom score over 5 days. |
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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) |
Secondary outcomes include [a] time to recovery (recovery defined as a respiratory symptom score of 5 or lower indicating only trivial symptoms), and adverse effects over the duration to the intervention (1 week), [b] healthcare utilisation (i.e. primary care physician re-consultations, medication prescriptions (e.g. antibiotics), specialist consultations and hospital admissions), and cost-effectiveness (4 weeks), and [c] the proportion of infants with persistent wheezing on auscultation at day 5 (included as objective outcome measure). |
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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 | 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) | 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 | 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.8.2.4 | 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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
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 | 10 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 10 |
E.8.9.2 | In all countries concerned by the trial days | 0 |