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PRISTINE - Personalised Approach to Improve aSThma prescrIbing iN childrEn (PRISTINE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
Sponsor:
Information provided by (Responsible Party):
Brighton and Sussex University Hospitals NHS Trust

Brief 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 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Randomized
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)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Asthma

Arm Intervention/treatment
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)



Primary Outcome Measures :
  1. 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.

  2. 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

  3. 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

  4. 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.


Secondary Outcome Measures :
  1. Childhood Asthma Control Test [ Time Frame: Baseline to 3 months ]
    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.

  2. 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)

  3. 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.

  4. 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.

  5. 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.

  6. Beliefs about medicine questionnaire [ Time Frame: Baseline to 3 months ]
    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.

  7. 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 [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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

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


Locations
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United Kingdom
BrightonNHS
Brighton, East Sussex, United Kingdom, BN2 5BE
Sponsors and Collaborators
Brighton and Sussex University Hospitals NHS Trust
Investigators
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Principal Investigator: Somnath Prof Mukhopadhyay Brighton and Sussex University Hospital NHS Trust

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Responsible Party: Brighton and Sussex University Hospitals NHS Trust
ClinicalTrials.gov Identifier: NCT03269318    
Other Study ID Numbers: 183898
First Posted: August 31, 2017    Key Record Dates
Last Update Posted: December 18, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Asthma
Bronchial Diseases
Respiratory Tract Diseases
Lung Diseases, Obstructive
Lung Diseases
Respiratory Hypersensitivity
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Theophylline
Salmeterol Xinafoate
Montelukast
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Leukotriene Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Cytochrome P-450 CYP1A2 Inducers
Cytochrome P-450 Enzyme Inducers
Phosphodiesterase Inhibitors