E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
First acute wheezing episode |
|
E.1.1.1 | Medical condition in easily understood language |
First acute expiratory difficulty |
|
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 |
The overall objective of the study is to determine the efficacy of corticosteroids in preventing recurrent wheezing and asthma in high-risk, first-time severe wheezing children with rhinovirus infection. |
|
E.2.2 | Secondary objectives of the trial |
The secondary objectives are to determine duration and severity in each acute episode with acute expiratory breathing difficulty, the number and duration of episodes with acute expiratory breathing difficulty, quality of life, degree of pulmonary hyperreactivity and safety measures including se-cortisol, height and weight, within 24 months after study entry. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Age 3-24 months, 2) first acute severe wheezing episode, 3) rhinovirus positive PCR-test in nasopharyngeal aspirate 4) written informed consent.
|
|
E.4 | Principal exclusion criteria |
1) Previous episodes of expiratory breathing difficulties 2) gestational age < 37 weeks 3) Chronic illness other than atopy (eczema) 4) previous systemic or inhaled corticosteroid treatment 5) COVID-19 related disease 6) participation to another trial 7) varicella infection or contact during the last 2-3 weeks 8) need for intensive care unit treatment during the present infection except for respiratory support with non-invasive ventilation methods (high frequency nasal cannula ventilation, CPAP or BiPAP). 8) need for intensive care unit treatment, except for respiratory support with high frequency nasal cannula ventilation
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoints are the time to a new physician-confirmed wheezy episode, and time to need for a regular controller medication for asthma within 24 months after study entry.
Primary interaction analyses: Age, gender, atopic predisposition as indicated by presence of atopic eczema, elevated blood-eosinophile count and allergic sensitization, risk genotype [the 17q locus or CDHR3], rhinovirus genome load and co-dectection of respiratory syncytial virus and other airway viruses. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Continuously (symptom diary, scheduled calls and visits) for two years; using questionnaire, patient charts and final visit until 6 year follow-up, and national registry data of drug purchases until 14 years of age. |
|
E.5.2 | Secondary end point(s) |
Determined at the first episode of acute breathing difficulty within 24 months of study entry: - duration and severity of symptoms Determined at each scheduled follow-up visit within 24 months of study entry: -number, duration and severity of episodes with acute breathing difficulty since start of study medication -degree of pulmonary hyperreactivity -quality of life -height and weight
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Continuously (symptom diary, scheduled calls and visits) for two years. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
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 | 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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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
|
After LVLS, national registry data of drug purchases will be analysed until age 14 years. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 16 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 16 |