AsthmaPhase 4PRISTINENCT03269318
PRISTINE - Personalised Approach to Improve aSThma prescrIbing iN childrEn
A Phase 4 study testing Montelukast or Salmeterol or Theophylline or Steroid in people with Asthma, run by Brighton and Sussex University Hospitals NHS Trust at 1 site in United Kingdom.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 5 to 11 years
- Conditions the study is for
- Asthma
- What is being tested?
- Montelukast or Salmeterol or Theophylline or Steroid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Jul 2017to 30 Aug 2019
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent/Guardian/Participant is willing and able to give informed consent/assent
- Physician-diagnosed asthma that is inadequately controlled as per view of doctor (for example, history of at least two emergency visits to GP or hospital over the previous year, frequent use of blue inhaler (three times or more per week))
- Aged 5-11 years (inclusive)
- Children who are already on at least 400 micrograms per day inhaled beclomethasone or equivalent and hence ready to be prescribed inhaled long-acting beta2 agonists and/or other add-on medication or are already on inhaled long-acting beta2 agonists and/or other add-on medication
Who cannot join
- Parent/Guardian/Participant is unwilling or unable to give informed consent/assent
- Known contraindication to montelukast or salmeterol
- Other known significant airway or lung disease (e.g. chronic lung disease of prematurity, cystic fibrosis or congenital airway abnormalities) or other co-existing serious disease such as congenital cardiac disease
- Poor inhaler technique and/or history of poor adherence on checking following standard procedure at the clinic
- Participating in another clinical trial (other than observational trials and registries) concurrently or within 30 days prior to screening for entry into this study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Feasibility of a Personalised Medicine Clinic for Children With Asthma Aged 5-11 Years
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".
Read the detailed description
Asthma is a common chronic illness in children and young people. It affects, for example, an average of two children in every UK classroom. Initial treatment usually consists of salbutamol used on demand at step 1 of British Thoracic Society (BTS) guidelines. At step 2, regular anti-inflammatory 'controller' therapy starts with the regular use of inhaled corticosteroids such as beclomethasone. Therapeutic efficacy with inhaled steroids usually peaks around 400 micrograms per day of beclomethasone (or equivalent). With inadequate asthma control at step 2, inhaled long-acting β2 agonists (LABA) such as salmeterol, or leukotriene receptor antagonists (LTRA) such as montelukast are added or inhaled corticosteroids are increased; this represents BTS step 3 for asthma management.
Overall, in children with asthma managed on step 3, salmeterol appears to provide better asthma control than montelukast in the setting of a randomized controlled trial. However, in real life, the efficacy of salmeterol at step 3 for improving asthma control in individual children appears rather variable, and some children continue to experience day-to-day symptoms and exacerbations.
In this study of 1182 UK children and young adults (4-22 years), 50% of those on regular salmeterol experienced asthma exacerbations over a 6-month period, and 18% required inhaled salbutamol at least daily for symptom relief. Indeed, the investigators reported a step-wise increase in the risk of asthma attacks related to each copy of the Arg16 allele on the β2 receptor gene (1.7-fold) in asthmatic children and young adults exposed to regular salmeterol in conjunction with inhaled corticosteroids. This led the investigators to hypothesize that, contrary to the observations on the overall population of children and young adults where salmeterol is superior in efficacy to montelukast at step 3, those possessing susceptible Arg16 β2 receptor genotype may experience better asthma control with the addition of montelukast rather than salmeterol as second-line controller medication, in addition to inhaled corticosteroids. As such the investigators elected to identify from the database those children with two copies of the Arg16 polymorphism [i.e. homozygous Arg genotype (∼15% of overall population) who would potentially be at greatest risk]. The mechanism for worse control with regular salmeterol involves a greater susceptibility to agonist-induced down-regulation and uncoupling of airway β2 receptors and associated sub-sensitivity of response in the Arg16 genotype.
The investigators therefore performed a proof-of-concept randomized controlled trial to determine whether genetically susceptible children with homozygous Arg16 genotype experience superior long-term asthma control with montelukast compared with salmeterol when used as tailored second-line controller therapy as add-on to the inhaled steroid fluticasone. The purpose of this preliminary study was to provide evidence to support the potential for personalised medicine based on the individual genotype to improve asthma-related quality-of-life and control. This study was published in 2013, and represents the first prospective randomized controlled study in children with asthma that addresses personalised medicine based on genotype. The results of this study showed that in children expressing the homozygous Arg 16 genotype, in comparison with salmeterol, adding montelukast to inhaled fluticasone significantly improved asthma-related quality-of-life and clinical symptoms, while reducing school absences and inhaled reliever use. The relative benefits of montelukast in comparison with salmeterol became evident within the first 3 months and persisted throughout the whole year.
Subsequently, the investigators used Pubmed to search the Medline database for other randomised controlled trials comparing the effects of salmeterol (or other long-acting beta2 agonist) with montelukast (or other leukotriene antagonist) within the context of Arg/Gly variation, in children with asthma. No studies could be identified. In particular, there are no trials in either adults or children that have studied quality-of-life, which is a key outcome of interest in the context of asthma-related disability, and which is often unrelated to outcomes such as lung function. This led to the development of the Personalised Medicine for Asthma Control (PACT)-study, a randomised controlled trial to determine if personalised medicine improves quality of life and asthma control in 12-18 years olds. Results of this trial when published, will provide more conclusive evidence as to the effectiveness of personalised medicine in this age group. However, there is an absence of trials in a younger age group of children with asthma (5-11 years) and no evidence to determine if a personalised medicine clinic is feasible within a hospital setting, which underscores the need for this study.
There is an absence of trials in a younger age group of children with asthma (5-11 years) and no evidence to determine if a personalised medicine clinic is feasible within a hospital setting and this underscores the need for this study.
This research proposes two stages of work and has two main objectives:
- Feasibility study: Conduct a feasibility study to determine important parameters (standard deviation of outcomes, recruitment and retention) to inform the design of a definitive randomised controlled trial Research questions:
- Are children with asthma and their parents willing to be recruited and randomised to a trial of genotyping and personalised management for asthma?
- Are there retention issues? If yes, at what stages did these occur? What were the reasons?
- Are follow-up data complete?
- Can the intervention (genotyping plus medication) be delivered with sufficient fidelity?
- Is there sufficient evidence for scaling up to a definitive randomised controlled trial?
- What sample size is needed to power a full scale randomised controlled trial?
- Are there any safety issues or adverse events?
- What are the associated costs of running a personalised asthma clinic and is it cost effective?
- Qualitative aspect: Assess the acceptability of a personalised asthma clinic for children with asthma
Research questions:
- How acceptable do children with asthma and their parents find genotyping and the personalised approach? How does this compare with their views on acceptability of conventional clinics?
- Was there any aspect of genotyping and personalised medicine clinic which children with asthma and their parents thought was particularly good or worked well?
- Was there any aspect of genotyping and personalised medicine clinic which children with asthma and their parents thought was particularly bad or difficult?
- How satisfied were children with asthma and their parents with the genotyping and personalised medicine clinic?
- How did the genotyping and personalised medicine clinic differ from usual care?
- What would encourage other children with asthma and their parents to participate in a genotyping and personalised medicine clinic?
- What would participants change about the personalised clinic?
- How has the genotyping and personalised medicine clinic impacted on the child and their parent?
- Were there any outcomes which weren't measured which should have been?
- What did the health professionals involved think about the clinic? (in primary and secondary care)
Two arm, randomised controlled feasibility trial of genotyping and personalised medicine versus usual care with qualitative aspect to assess acceptability and impact.
The genotyping and personalised medicine clinic is based in the Royal Alexandra Children's Hospital in Brighton, England. Participants are referred to the research team by their health care professional (primary and secondary care).
The intervention and follow up period will last 4 months per participant. Outcomes will be measured at baseline and 3-months.
- Conditions
- Asthma
- Being tested
- Montelukast or Salmeterol or Theophylline or Steroid
- Medical subject headings
- Asthma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Montelukast or Salmeterol or Theophylline or SteroidDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Personalised Medicine | Experimental | Personalised Medicine who will be prescribed controller medication based on genetic test, Arg/Arg or Arg/Gly - montelukast (LTRA) or Gly/Gly -salmeterol (LABA). Receives: Drug: Montelukast or Salmeterol or Theophylline or Steroid |
| Standard Care | No intervention | Standard of care (Standard Care will be prescribed controller medication based on guidelines) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: Baseline to 3 months
Are there retention issues? If yes, at what stages did these occur? What were the reasons? Qualitative interview
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
Measured: Baseline to 3 months
Are follow-up data complete?
Suitability of outcome measures will be evaluated based on completion rates and rates of missing data
Measured: Baseline to 3 months
Acceptability of personalised approach
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.
Measured: Baseline to 3 months
Secondary outcomes (7)
Childhood Asthma Control Test
The Childhood Asthma Control Test (C-ACT) [10], 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.
Measured: Baseline to 3 months
Lung Function
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)
Measured: Baseline to 3 months
Days unable to complete usual activities
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.
Measured: Baseline to 3 months
Use of medication
Number of courses of oral corticosteroids for asthma and any other medication use will be recorded.
Measured: Baseline to 3 months
Use of health services
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.
Measured: Baseline to 3 months
Beliefs about medicine questionnaire
The Beliefs About Medicine Questionnaire (BMQ) [11] 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.
Measured: Baseline to 3 months
Experience of service
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 [12]. 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.
Measured: 3 month visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2017
- Study start1 Jul 2017
- Primary completion30 Aug 2019
- Study completion30 Aug 2019
Why it stopped: Change to Primary Endpoint resulted in development of new protocol
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AsthmaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brighton and Sussex University Hospitals NHS Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Somnath Prof Mukhopadhyay, Principal investigator, Brighton and Sussex University Hospital NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03269318
- Sponsor's protocol ID
- 183898
- Acronym
- PRISTINE
- Status last verified
- Dec 2019
- First submitted
- 11 Jul 2017
- Last updated
- 18 Dec 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility of a Personalised Medicine Clinic for Children With Asthma Aged 5-11 Years
- ClinicalTrials.gov ID
- NCT03269318
- Acronym / sponsor ID
- PRISTINE / 183898
- Conditions
- Asthma
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Change to Primary Endpoint resulted in development of new protocol
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Brighton and Sussex University Hospitals NHS TrustUniversity, hospital or other
- Study start
- 1 Jul 2017 (actual)
- Primary completion
- 30 Aug 2019 (actual)
- Study completion
- 30 Aug 2019 (actual)
- First posted
- 31 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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".
Detailed description
Asthma is a common chronic illness in children and young people. It affects, for example, an average of two children in every UK classroom. Initial treatment usually consists of salbutamol used on demand at step 1 of British Thoracic Society (BTS) guidelines. At step 2, regular anti-inflammatory 'controller' therapy starts with the regular use of inhaled corticosteroids such as beclomethasone. Therapeutic efficacy with inhaled steroids usually peaks around 400 micrograms per day of beclomethasone (or equivalent). With inadequate asthma control at step 2, inhaled long-acting β2 agonists (LABA) such as salmeterol, or leukotriene receptor antagonists (LTRA) such as montelukast are added or inhaled corticosteroids are increased; this represents BTS step 3 for asthma management.
Overall, in children with asthma managed on step 3, salmeterol appears to provide better asthma control than montelukast in the setting of a randomized controlled trial. However, in real life, the efficacy of salmeterol at step 3 for improving asthma control in individual children appears rather variable, and some children continue to experience day-to-day symptoms and exacerbations.
In this study of 1182 UK children and young adults (4-22 years), 50% of those on regular salmeterol experienced asthma exacerbations over a 6-month period, and 18% required inhaled salbutamol at least daily for symptom relief. Indeed, the investigators reported a step-wise increase in the risk of asthma attacks related to each copy of the Arg16 allele on the β2 receptor gene (1.7-fold) in asthmatic children and young adults exposed to regular salmeterol in conjunction with inhaled corticosteroids. This led the investigators to hypothesize that, contrary to the observations on the overall population of children and young adults where salmeterol is superior in efficacy to montelukast at step 3, those possessing susceptible Arg16 β2 receptor genotype may experience better asthma control with the addition of montelukast rather than salmeterol as second-line controller medication, in addition to inhaled corticosteroids. As such the investigators elected to identify from the database those children with two copies of the Arg16 polymorphism [i.e. homozygous Arg genotype (∼15% of overall population) who would potentially be at greatest risk]. The mechanism for worse control with regular salmeterol involves a greater susceptibility to agonist-induced down-regulation and uncoupling of airway β2 receptors and associated sub-sensitivity of response in the Arg16 genotype.
The investigators therefore performed a proof-of-concept randomized controlled trial to determine whether genetically susceptible children with homozygous Arg16 genotype experience superior long-term asthma control with montelukast compared with salmeterol when used as tailored second-line controller therapy as add-on to the inhaled steroid fluticasone. The purpose of this preliminary study was to provide evidence to support the potential for personalised medicine based on the individual genotype to improve asthma-related quality-of-life and control. This study was published in 2013, and represents the first prospective randomized controlled study in children with asthma that addresses personalised medicine based on genotype. The results of this study showed that in children expressing the homozygous Arg 16 genotype, in comparison with salmeterol, adding montelukast to inhaled fluticasone significantly improved asthma-related quality-of-life and clinical symptoms, while reducing school absences and inhaled reliever use. The relative benefits of montelukast in comparison with salmeterol became evident within the first 3 months and persisted throughout the whole year.
Subsequently, the investigators used Pubmed to search the Medline database for other randomised controlled trials comparing the effects of salmeterol (or other long-acting beta2 agonist) with montelukast (or other leukotriene antagonist) within the context of Arg/Gly variation, in children with asthma. No studies could be identified. In particular, there are no trials in either adults or children that have studied quality-of-life, which is a key outcome of interest in the context of asthma-related disability, and which is often unrelated to outcomes such as lung function. This led to the development of the Personalised Medicine for Asthma Control (PACT)-study, a randomised controlled trial to determine if personalised medicine improves quality of life and asthma control in 12-18 years olds. Results of this trial when published, will provide more conclusive evidence as to the effectiveness of personalised medicine in this age group. However, there is an absence of trials in a younger age group of children with asthma (5-11 years) and no evidence to determine if a personalised medicine clinic is feasible within a hospital setting, which underscores the need for this study.
There is an absence of trials in a younger age group of children with asthma (5-11 years) and no evidence to determine if a personalised medicine clinic is feasible within a hospital setting and this underscores the need for this study.
This research proposes two stages of work and has two main objectives:
- Feasibility study: Conduct a feasibility study to determine important parameters (standard deviation of outcomes, recruitment and retention) to inform the design of a definitive randomised controlled trial Research questions:
- Are children with asthma and their parents willing to be recruited and randomised to a trial of genotyping and personalised management for asthma?
- Are there retention issues? If yes, at what stages did these occur? What were the reasons?
- Are follow-up data complete?
- Can the intervention (genotyping plus medication) be delivered with sufficient fidelity?
- Is there sufficient evidence for scaling up to a definitive randomised controlled trial?
- What sample size is needed to power a full scale randomised controlled trial?
- Are there any safety issues or adverse events?
- What are the associated costs of running a personalised asthma clinic and is it cost effective?
- Qualitative aspect: Assess the acceptability of a personalised asthma clinic for children with asthma
Research questions:
- How acceptable do children with asthma and their parents find genotyping and the personalised approach? How does this compare with their views on acceptability of conventional clinics?
- Was there any aspect of genotyping and personalised medicine clinic which children with asthma and their parents thought was particularly good or worked well?
- Was there any aspect of genotyping and personalised medicine clinic which children with asthma and their parents thought was particularly bad or difficult?
- How satisfied were children with asthma and their parents with the genotyping and personalised medicine clinic?
- How did the genotyping and personalised medicine clinic differ from usual care?
- What would encourage other children with asthma and their parents to participate in a genotyping and personalised medicine clinic?
- What would participants change about the personalised clinic?
- How has the genotyping and personalised medicine clinic impacted on the child and their parent?
- Were there any outcomes which weren't measured which should have been?
- What did the health professionals involved think about the clinic? (in primary and secondary care)
Two arm, randomised controlled feasibility trial of genotyping and personalised medicine versus usual care with qualitative aspect to assess acceptability and impact.
The genotyping and personalised medicine clinic is based in the Royal Alexandra Children's Hospital in Brighton, England. Participants are referred to the research team by their health care professional (primary and secondary care).
The intervention and follow up period will last 4 months per participant. Outcomes will be measured at baseline and 3-months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Personalised Medicine | Experimental | Drug: Montelukast or Salmeterol or Theophylline or SteroidPersonalised Medicine who will be prescribed controller medication based on genetic test, Arg/Arg or Arg/Gly - montelukast (LTRA) or Gly/Gly -salmeterol (LABA). |
| Standard Care | No intervention | –Standard of care (Standard Care will be prescribed controller medication based on guidelines) |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Parent/Guardian/Participant is willing and able to give informed consent/assent
- Physician-diagnosed asthma that is inadequately controlled as per view of doctor (for example, history of at least two emergency visits to GP or hospital over the previous year, frequent use of blue inhaler (three times or more per week))
- Aged 5-11 years (inclusive)
- Children who are already on at least 400 micrograms per day inhaled beclomethasone or equivalent and hence ready to be prescribed inhaled long-acting beta2 agonists and/or other add-on medication or are already on inhaled long-acting beta2 agonists and/or other add-on medication
Key exclusion criteria (5 listed)
- Parent/Guardian/Participant is unwilling or unable to give informed consent/assent
- Known contraindication to montelukast or salmeterol
- Other known significant airway or lung disease (e.g. chronic lung disease of prematurity, cystic fibrosis or congenital airway abnormalities) or other co-existing serious disease such as congenital cardiac disease
- Poor inhaler technique and/or history of poor adherence on checking following standard procedure at the clinic
- Participating in another clinical trial (other than observational trials and registries) concurrently or within 30 days prior to screening for entry into this study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Montelukast or Salmeterol or Theophylline or SteroidMedication 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. | Drug | Personalised Medicine |
Primary outcome
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.
7 secondary outcomes
Childhood Asthma Control Test
Time frame: Baseline to 3 months
Lung Function
Time frame: Baseline to 3 months
Days unable to complete usual activities
Time frame: Baseline to 3 months
Use of medication
Time frame: Baseline to 3 months
Use of health services
Time frame: Baseline to 3 months
Beliefs about medicine questionnaire
Time frame: Baseline to 3 months
Experience of service
Time frame: 3 month visit
Results and safety
The study was stopped early (reason given: Change to Primary Endpoint resulted in development of new protocol). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2019.
- Individual participant dataSharing planned: Undecided
Related trials for Asthma
Active, not recruitingPhase 1
A Comparative Study to Investigate the Difference in Blood Levels of Two Different Formulations of GSK5784283
Asthma
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingObservational study
A Study Evaluating Disease Characteristics and Outcomes in Participants With Asthma in Routine Clinical Practice
Asthma
RecruitingPhase 3
A Study to Compare Safety of 4 Weeks Exposure to Propellants Hydrofluoroalkane 1,1-difluoroethane (HFA-152a) and HFA-1,1,1,2-tetrafluoroethane (134a)
Asthma
Not yet recruitingPhase 4
Novel Method for Removal of Orally Deposited Inhaled Fluticasone Propionate
Asthma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Somnath Prof Mukhopadhyay principal investigator
Brighton and Sussex University Hospital NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Montelukast or Salmeterol or Theophylline or Steroid in people with Asthma, run by Brighton and Sussex University Hospitals NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Change to Primary Endpoint resulted in development of new protocol). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2019.