PRISTINE - Personalised Approach to Improve aSThma prescrIbing iN childrEn (PRISTINE)
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|ClinicalTrials.gov Identifier: NCT03269318|
Recruitment Status : Terminated (Change to Primary Endpoint resulted in development of new protocol)
First Posted : August 31, 2017
Last Update Posted : December 18, 2019
Asthma is one of the most common chronic diseases affecting children in the UK. Poorly controlled asthma manifests with chronic cough, wheeze and shortness of breath which in-turn has a significant negative impact on a child's quality of life, interfering with sleep, impairing exercise ability and resulting in frequent school absences and hospital admissions.
Management of paediatric asthma in the UK is directed by the British Thoracic Society (BTS) Guidelines, which recommend a stepwise (one to five) treatment plan. Step three of the management guideline for children aged 5-12 years of age recommends the addition of the preventer inhaled medication, including long-acting β2 agonists such as salmeterol. However, there is a wide variation in response to this medication with approximately one in seven people, with a specific genetic change, found to have an increase in asthma symptoms in association with the use of thisiss medication. A related medicine, formoterol, is used less commonly in children with asthma.
In this study, the investigators will aim to identify children with asthma whose symptoms are poorly controlled on inhaled long-acting beta2 agonists. Via a simple saliva test, the investigators will identify the presence or absence of the specific genetic change potentally influencing the effectiveness of treatment with salmeterol or related longacting beta2 agonists thus enabling the investigators to recommend either salmeterol or an alternative medication for the treatment plan such as montelukast. The investigators will randomise the patients into two groups; to receive "personalised care" where the choice of controller medication would be based on the child's gene test results and predicted response to long-acting beta2 agonists, or "standard care" following the BTS guidelines at the clinician's discretion without knowledge of the gene test results. The investigators aim to measure whether this individualized approach to asthma prescribing results in improved control of asthma symptoms and overall quality of life. Targeting treatment to a child's specific genetic make-up is a concept known as "personalised medicine".
|Condition or disease||Intervention/treatment||Phase|
|Asthma||Drug: Montelukast or Salmeterol or Theophylline or Steroid||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Participants will be allocated to one of two groups as per block randomisation, with no stratification or minimisation to Group 1; Personalised Medicine who will be prescribed controller medication based on genetic test, Arg/Arg or Arg/Gly - montelukast (LTRA) or Gly/Gly -salmeterol (LABA). While Group 2, Standard Care will be prescribed controller medication based on guidelines.|
|Masking:||None (Open Label)|
|Official Title:||Feasibility of a Personalised Medicine Clinic for Children With Asthma Aged 5-11 Years|
|Actual Study Start Date :||July 1, 2017|
|Actual Primary Completion Date :||August 30, 2019|
|Actual Study Completion Date :||August 30, 2019|
Experimental: Personalised Medicine
Personalised Medicine who will be prescribed controller medication based on genetic test, Arg/Arg or Arg/Gly - montelukast (LTRA) or Gly/Gly -salmeterol (LABA).
Drug: Montelukast or Salmeterol or Theophylline or Steroid
Medication will be patient specific according to their current medication, clinical symptoms and genotype. It will be from a choice of; leukotriene receptor antagonist (montelukast), long-acting beta2 agonist (salmeterol), theophylline or increase dose of inhaled steroid.
No Intervention: Standard Care
Standard of care (Standard Care will be prescribed controller medication based on guidelines)
- Are children with asthma and their parents willing to be recruited and randomised to a trial of genotyping and personalised management for asthma? Qualitative interview [ Time Frame: Baseline to 3 months ]Recruitment rates will be measured as rate of invited participants who are eligible and consenting and will be reported in a Consolidated Standards of Reporting Trials (CONSORT) participant flowchart.
- Are there retention issues? If yes, at what stages did these occur? What were the reasons? Qualitative interview [ Time Frame: Baseline to 3 months ]Acceptability of allocation procedures will be assessed by examining reasons for dropout in discontinuing participants and comparing attrition rates between the two study groups and between participants who did and did not receive their preferred allocation. Attrition rates will be established as discontinuation of intervention and loss to follow-up measurement for both groups
- Are follow-up data complete? [ Time Frame: Baseline to 3 months ]Suitability of outcome measures will be evaluated based on completion rates and rates of missing data
- Acceptability of personalised approach [ Time Frame: Baseline to 3 months ]All participants and their parents/guardians will be invited to have a semi-structured interview with a member of the research team in order to discuss their experiences of living with and managing their asthma. To enhance communication and ensure the child's perspective is captured, children will be invited to make a drawing of what it is like to have asthma and what it feels like when they take their asthma medication. The research team interviewing will then discuss the drawings (as a visual cue) in simple language with each child to understand what the child means.
- Childhood Asthma Control Test [ Time Frame: Baseline to 3 months ]The Childhood Asthma Control Test (C-ACT) , a 7-item validated questionnaire capturing the frequency of asthma symptoms and their effect on daily function in children 4 to 11 years of age. It uses a 4-point Likert scale with higher scores indicating better control. The C-ACTuses a single cut point of a score of ≤19 to identify children whose asthma is not well controlled.
- Lung Function [ Time Frame: Baseline to 3 months ]Lung function will be measured by a nurse trained in collecting spirometry data in the Royal Alexandra Children's Hospital. Measure of PEF (litres/second), FEV1 (litres) and FVC (litres)
- Days unable to complete usual activities [ Time Frame: Baseline to 3 months ]Participants and parents will be asked to report how many days in the last month they have been unable to complete usual activities as a result of their asthma.
- Use of medication [ Time Frame: Baseline to 3 months ]Number of courses of oral corticosteroids for asthma and any other medication use will be recorded.
- Use of health services [ Time Frame: Baseline to 3 months ]Participants and parents will be asked to report how many times they have had to see their GP or asthma nurse (outside of routine asthma review), been to A&E or been admitted to hospital as a result of their asthma.
- Beliefs about medicine questionnaire [ Time Frame: Baseline to 3 months ]The Beliefs About Medicine Questionnaire (BMQ)  is an 18-item validated questionnaire which will capture parental beliefs about asthma, asthma medication and how these may have affected their child's life. Respondents indicate their degree of agreement with each individual statement about medicines on a 5-point Likert scale, (1=strongly disagree to 5=strongly agree). Scores obtained are summed to give a scale score with higher scores indicating stronger beliefs.
- Experience of service [ Time Frame: 3 month visit ]At the final follow up, participants and parents will be asked to comment on their experience of the service received in the personalised medicine clinic. The validated Commission for Health Improvement Experience of Service Questionnaire will be used as the outcome measure . The ESQ consists of 12 items rated on a 3-point Likert scale (3=Certainly True to 1=Not true) and three free-text sections looking at what the respondent liked about the clinic, what they felt needed improving, and any other comments. Higher scores indicate more positive experiences.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03269318
|Brighton, East Sussex, United Kingdom, BN2 5BE|
|Principal Investigator:||Somnath Prof Mukhopadhyay||Brighton and Sussex University Hospital NHS Trust|