E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Exacerbation of asthma in children presenting to the Emergency Department |
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E.1.1.1 | Medical condition in easily understood language |
Worsening of asthma symptoms in children requiring their attendance at the Emergency Department |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003566 |
E.1.2 | Term | Asthmatic attack |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10015575 |
E.1.2 | Term | Exacerbation of asthma |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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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 test whether a single dose of oral dexamethasone (0.3mg/kg) is non-inferior to prednisolone (1mg/kg/day for 3 days) in the treatment of exacerbations of asthma in children, as measured by the Pediatric Respiratory Assessment Measure |
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E.2.2 | Secondary objectives of the trial |
Secondary hypotheses to be tested are related to:
•Relapse (this is defined as a return to care at an Emergency Department or other healthcare provider with worsening or unresolving acute asthma within 14 days of inclusion in this study) - we hypothesise that the dexamethasone group will experience similar or fewer episodes of relapse as the prednisolone group
•Number of salbutamol therapies given following enrolment – we hypothesise that patients in the dexamethasone group will require a similar number or fewer salbutamol therapies during the study period.
•Side effects (in particular vomiting) - we hypothesise that the dexamethasone group will experience similar or fewer episodes of vomiting as the prednisolone group.
•Compliance – we will measure compliance of each study participant in the prednisolone group with regard to taking the 2nd and 3rd dose of medication.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Ages 2 – 16 years
•Background history of asthma.
Asthma for the purpose of this study will be defined as either: at least one previous episode of ß2-agonist-responsive wheeze in a child two years of age or over; or a prior diagnosis of asthma, made by a paediatrician, or clinician of comparable experience.
•Presentation with an asthma exacerbation
An exacerbation of asthma, for the purpose of this study, will be defined as acute asthma which prompts assessment at the Emergency Department, and has any, or all, of the following clinical features:
o Dyspnoea
o Wheeze
o Acute cough
o Increased work of breathing
o Increased requirement for ß2-agonist from baseline use
o SpO2 <95%
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E.4 | Principal exclusion criteria |
•Less than 2 years old or over 16 years
•Critical or life-threatening asthma, defined as patients displaying one or more of the following clinical features:
o Confused/drowsy
o Maximal accessory muscle use/recession
o Poor respiratory effort (including bradypnoea)
o Exhaustion
o Silent chest
o Cyanosis
o O2 saturation < 90% in air
o Marked tachycardia
o Unable to verbalise normally
o Pneumothorax
•Known TB exposure
•Active varicella or herpes simplex infection
•Documented concurrent infection with RSV
•Fever >39.5 degrees Celcius
•Use of oral corticosteroids in the previous 4 weeks
•Concurrent stridor
•Significant co-morbid disease:
Lung, cardiac, immune, liver, endocrine, neurological or psychiatric
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E.5 End points |
E.5.1 | Primary end point(s) |
The Pediatric Respiratory Assessment Measure (PRAM), as the marker of asthma symptom severity will be the primary endpoint. The PRAM is a validated, responsive and reliable tool used to determine asthma severity in children aged 2 to 17 years. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The Pediatric Respiratory Assessment Measure as the primary end point will be completed on Day 4. |
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E.5.2 | Secondary end point(s) |
o Relapse rate, which will include any visit to a healthcare provider e.g. General Practitioner, Emergency Department
o Number of salbutamol therapies given following enrolment
oSide effects, in particular, vomiting
oCompliance with medication, assessed by interview and the return of medication bottles
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Relapse rate will be asessed at Day 4 and Day 14
Number of salbutamol therapies will be assessed Day 4 and Day 14
Side effects will be assessed at Day 4 and Day 14
Compliance will be assessed at Day 4 |
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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 | 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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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) | Yes |
E.8.2.2 | Placebo | No |
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
| 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 | 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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The definition of the end of the trial is 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 | 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 |