Severe paediatric asthma Respiratory Asthma
Conditions
Interventions
The trial will be undertaken at 11 specialist paediatric asthma centres across the UK. All children who are referred to the centres with poor asthma control despite being prescribed maximal inhaled th
Sponsors
Imperial College London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Current inclusion criteria as of 02/02/2023: 1. Written informed consent given 2. Aged 6 to 16 years 3. Confirmed diagnosis of asthma 4. Poor asthma control despite being prescribed high dose therapy Inclusion criteria for RCT phase: 1. Written informed consent 2. Children aged 6-17 years 3. Confirmed diagnosis of asthma with one of the following: 3.1. Persistent poor control =1 attack after adherence assessment with =80% adherence during run-in (STRA) 3.2. Persistent poor control and poor adherence despite optimal efforts to improve adherence, including enhanced monitoring (Refractory DA) 4. Female patients capable of becoming pregnant must agree to use hormonal contraception, intrauterine device, intrauterine hormone-releasing system, or to complete abstinence for the duration of the trial and up to 100 days after the last dose of IMP _____ Previous participant inclusion criteria as of 10/09/2020: Inclusion criteria for run-in phase: 1. Written informed consent given 2. Aged 6 to 16 years 3. Patients capable of becoming pregnant must agree to use hormonal contraception, intrauterine device, intrauterine hormone-releasing system, or to complete abstinence for the duration of the trial and up to 100 days after the last dose of IMP 4. Confirmed diagnosis of asthma 5. Poor asthma control despite being prescribed high dose therapy Inclusion criteria for RCT phase: 1. Children aged 6-17 years 2. Confirmed diagnosis of asthma with one of the following: 2.1. Persistent poor control =1 attack after adherence assessment with =80% adherence during run-in (STRA) 2.2. Persistent poor control and poor adherence despite optimal efforts to improve adherence, including enhanced monitoring (Refractory DA) _____ Previous participant inclusion criteria: 1. Confirmed diagnosis of asthma 2. On high dose maintenance treatment (>/=800mcg per day budesonide/equivalent and long-acting beta agonist) 3. At least 4 severe attacks (defined as the need for a short course of oral steroids, or hospitalisation) in the previous 12 months, OR if on maintenance oral steroids then at least 2 severe attacks in the last 12 months +/- 4. Poor asthma control defined as a score of < 20 on the Asthma Control Test (ACT) [children aged 12-16 years], or childhood Asthma Control Test (cACT) [children aged 6-11 years]
Exclusion criteria
Exclusion criteria: Current participant exclusion criteria as of 10/09/2020: 1. Considered unfit for the study or risk of noncompliance with study procedures by the responsible physician as a result of medical interview, physical examination or screening investigation 2. Known hypersensitivity to Omalizumab or Mepolizumab or to any of the excipients 3. History of drug or other allergy, which, in the opinion of the responsible physician, contra-indicates their participation 4. Pregnant, lactating or within 6 weeks post-partum or breast feeding 5. Participated within 3 months in a study using a new molecular entity, another study investigating drugs or in a study with invasive procedures 6. Significant alternative diagnoses that may mimic or complicate asthma, in particular dysfunctional breathing, panic attacks, and overt psychosocial problems (if these are thought to be the main diagnosis; they can be present in addition to asthma major problem rather than in addition to severe asthma) 7. Significant other primary pulmonary disorders in particular cystic fibrosis, or interstitial lung disease 8. Diagnosis of chronic inflammatory diseases other than asthma (e.g. inflammatory bowel disease) Previous participant exclusion criteria: 1. As a result of medical interview, physical examination or screening investigation the physician responsible considers the child unfit for the study or has a risk of non-compliance with study procedures 2. The child has a history of drug or other allergy, which, in the opinion of the responsible physician, contra-indicates their participation 3. Participant is female who is pregnant, lactating or within 6 weeks post-partum or breastfeeding 4. The child has participated within 3 months in a study using a new molecular entity, another study investigating drugs or in a study with invasive procedures 5. Significant alternative diagnoses that may mimic or complicate asthma, in particular dysfunctional breathing, panic attacks, and overt psychosocial problems (if these are thought to be the major problem rather than in addition to severe asthma) 6. Significant other primary pulmonary disorders in particular cystic fibrosis, or interstitial lung disease 7. Diagnosis of chronic inflammatory diseases other than asthma (e.g. inflammatory bowel disease)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measure as of 10/09/2020: 52-week asthma exacerbation rate; defined as the number of asthma attacks requiring high dose systemic steroids (oral, intravenous, or intramuscular) or asthma related admission to hospital (=4 hours in hospital) measured at 52 weeks _____ Previous primary outcome measure: 52-week asthma exacerbation rate, defined as the number of asthma attacks requiring a short course of high-dose oral steroids (for 3 or more days) or admission to hospital, measured at 52 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Current secondary outcome measures as of 30/07/2024: 1. Asthma severity measured using the composite asthma severity index (CASI) questionnaire at 4, 16, 32, and 52 weeks 2. Quality of life measured using paediatric asthma quality of life (PAQLQ) questionnaire at 4, 16, and 52 weeks 3. Lung function (FEV1, bronchodilator reversibility) measured by spirometry (blowing and breathing tests) at 4, 16, 32, and 52 weeks 4. Exhaled nitric oxide measured by blowing and breathing tests at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 5. Asthma control measured using asthma control test (ACT) questionnaire at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 6. Inhaled corticosteroid dose (dose determined by the manufacturer that makes the inhaled steroid) at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 7. Sputum inflammatory cell count and eosinophil peroxidase measured by sputum test at4, 16, and 52 weeks 8. Patient burden (number of visits and injections) measured using a patient-assessed visual analogue scale at 4, 16, 32, and 52 weeks _____ Previous secondary outcome measures as of 10/09/2020: 1. Asthma severity measured using the composite asthma severity index (CASI) questionnaire at 4, 16, 32, and 52 weeks 2. Quality of life measured using paediatric asthma quality of life (PAQLQ) questionnaire at 4, 16, and 52 weeks 3. Lung function (FEV1, bronchodilator reversibility) measured by spirometry (blowing and breathing tests) at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 4. Exhaled nitric oxide measured by blowing and breathing tests at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 5. Asthma control measured using asthma control test (ACT) questionnaire at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 6. Inhaled corticosteroid dose (dose determined by the manufacturer that makes the inhaled steroid) at 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52 weeks 7. Sputum inflammatory cell | — |
Countries
England, Scotland, United Kingdom
Outcome results
None listed