Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12618001497291
Ethics application status
Approved
Date submitted
7/08/2018
Date registered
5/09/2018
Date last updated
29/10/2024
Date data sharing statement initially provided
22/04/2021
Type of registration
Retrospectively registered

Titles & IDs
Public title
Australian Mepolizumab Registry (AMR) for Severe Asthma
Scientific title
Australian Mepolizumab Registry for Severe Asthma
Secondary ID [1] 295747 0
Protocol number 207455
Secondary ID [2] 313267 0
Protocol number 213520
Universal Trial Number (UTN)
Trial acronym
AMR
Linked study record

Health condition
Health condition(s) or problem(s) studied:
severe asthma 309145 0
Condition category
Condition code
Respiratory 308031 308031 0 0
Asthma

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
2
Target follow-up type
Years
Description of intervention(s) / exposure
A 4 year observation of patients treated with Mepolizumab for severe asthma.
Prior to starting treatment with Mepolizumab, patients will complete questionnaires concerning their asthma control and health-related quality of life. Details of medical history, asthma exacerbations and medication use are also recorded. Lung function and blood biomarkers (blood eosinophils, serum IgE) are also reported. Follow up data collection is performed at approximately 4, 7, 12, 18, 24, 36, 48 months after starting treatment. Data collection is aligned with standard clinic visits and assessments. Patients will again complete questionnaires and clinical outcomes will also be reported.

Intervention code [1] 312074 0
Not applicable
Comparator / control treatment
No control group. Observational registry.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 307016 0
Change in asthma control score
(Juniper Asthma Control Questionnaire - ACQ5)
Timepoint [1] 307016 0
Change in asthma control score is assessed at 4(3-5), 7(6-8), 12, 18, and 24, 36 and 48 months post-commencement of treatment. Asthma control is assessed at each time-point for the week preceding.
Primary outcome [2] 307017 0
Number of severe exacerbations
Severe asthma exacerbations identified as those requiring use of systemic corticosteroids prescribed or supervised by a physician, hospitalisation or an emergency department visit requiring systemic corticosteroids. Severe exacerbations are captured via clinical staff report/medical record review.
Timepoint [2] 307017 0
The number of severe exacerbations that have occurred since the previous visit are assessed at 4(3-5), 7(6-8), 12, 18, 24, 36 and 48 months post-commencement of treatment.
Secondary outcome [1] 350363 0
Quality of life
(Juniper Asthma Quality of Life Questionnaire)
Timepoint [1] 350363 0
Quality of life is assessed at 4(3-5), 7(6-8), 12, 18 and 24 months post-commencement of treatment. The AQLQ is completed at each time-point for the 2 weeks preceding.
Secondary outcome [2] 350364 0
Blood eosinophil levels
Timepoint [2] 350364 0
Blood eosinophil level is measured at 4(3-5) months, 12 months and 24, 36 and 48 months after commencement of treatment.
Secondary outcome [3] 350365 0
Lung function (FEV1, FVC) is assessed using standard spirometers in the clinic or pulmonary function laboratory.
Timepoint [3] 350365 0
Spirometry is performed to determine lung function at 4(3-5), 7(6-8), 12, 18, 24, 36 and 48 months post-commencement of treatment.
Secondary outcome [4] 350366 0
Health care resource utilisation is assessed via patient interview and medical record review.
Timepoint [4] 350366 0
Health care resource utilisation is assessed at 4(3-5), 7(6-8), 12, 18, 24, 36 and 48 months post-commencement of treatment. Health care resource utilisation since the previous assessment is assessed.
Secondary outcome [5] 350367 0
Drug-related adverse events will be assessed by clinical observation/patient report.
Acute and delayed systemic reactions, including hypersensitivity reactions (e.g. anaphylaxis, urticaria, angioedema, rash, bronchospasm, hypotension), have occurred following administration of mepolizumab. These reactions generally occur within hours of administration, but in some instances had a delayed onset (i.e., days).
Timepoint [5] 350367 0
during the week following administration of mepolizumab and during the 4-year followup
Secondary outcome [6] 441132 0
OCS use associated complications/effects are assessed via patient interview/questionnaire and medical record review.
Timepoint [6] 441132 0
OCS use associated complications/effects are collected at 4(3-5), 7(6-8), 12, 18, 24, 36 and 48 months post-commencement of treatment.

Eligibility
Key inclusion criteria
1. Able to provide informed written consent
2. Age greater than or equal to 12 years
3. Asthma diagnosis confirmed by doctor or hospital and documented for at least 1 year
4. Confirmed variable airflow obstruction (1 or more of the following) OR as required by current PBS criteria :
a. FEV1 reversibility greater than or equal to 12% and greater than or equal to 200mL at baseline within 30 minutes after administration of salbutamol (200-400micrograms), OR
b. Airway hyper-responsiveness (AHR) defined as >20% decline in FEV1 during a direct bronchial provocation test or >15% decline during an indirect test, OR
c. Peak Expiratory Flow (PEF) variability of >15% between the 2 highest and 2 lowest peak expiratory flow rates during 14 days
5. Optimised asthma management skills (formal assessment of and adherence to correct inhaler technique, documented in medical records)

6. Eligible to commence mepolizumab via the Pharmaceutical Benefit Scheme (PBS)-subsidised criteria OR outside of the PBS restrictions
7. Optimised asthma therapy (unless contraindicated or not tolerated):
a. greater than or equal to 12 months maximal inhaled therapy with adherence and correct technique documented, including:
i. High dose inhaled corticosteroid (ICS)
ii. Long-acting beta-2 agonist (LABA) therapy
AND
b. Treatment with oral corticosteroids (OCS)
8. Uncontrolled asthma, as defined by:
a. Asthma Control Questionnaire (ACQ-5) score AND
b. One of the following experienced in the previous year:
i. greater than or equal to 1 admission to hospital for a severe asthma exacerbation, OR
ii. greater than or equal to 1 severe asthma exacerbation requiring documented use of systemic corticosteroids (OCS initiated or increased for at least 3 days, or parenteral corticosteroids) prescribed/supervised by a physician.
9. Eosinophilic asthma as determined from peripheral blood eosinophil count (as according to the current PBS criteria).
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. People highly dependent on medical care
2. Cognitive impairment preventing completion of data collection forms

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
Summary statistics will be produced for the key variables contained within the database, such as asthma characteristics, and asthma treatment for baseline data. Mepolizumab treatment cycles will be described and clinical response variables summarised. Relevant comparisons will be conducted between defined sub-groups and based on subject characteristic variables [e.g. age, sex, treatment intensity, asthma duration, etc]. Subject to data availability, data linkage analysis may be conducted for selected outcome variables.
A target minimum sample size is 100 participants which will give sufficient power to allow precision around point estimates. The likely sample size will be between 200 and 400 participants over the study enrolment period.
Demographic and participant characteristics will be summarised using measures of central tendency (mean, median) and appropriate variance estimates. Proportions of responders, and participants developing adverse events will be reported with 95% confidence intervals. Subgroup analyses will be performed with participants categorized by response; comorbidity, phenotypic characteristics.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
19/01/2017
Date of last participant enrolment
Anticipated
30/06/2019
Actual
3/04/2024
Date of last data collection
Anticipated
30/04/2021
Actual
30/04/2024
Sample size
Target
500
Accrual to date
Final
426
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 11594 0
John Hunter Hospital - New Lambton
Recruitment hospital [2] 11595 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 11596 0
Concord Repatriation Hospital - Concord
Recruitment hospital [4] 11597 0
Campbelltown Hospital - Campbelltown
Recruitment hospital [5] 11598 0
The Royal Adelaide Hospital - Adelaide
Recruitment hospital [6] 11599 0
Austin Health - Austin Hospital - Heidelberg
Recruitment hospital [7] 11600 0
The Alfred - Prahran
Recruitment hospital [8] 11601 0
Frankston Hospital - Frankston
Recruitment hospital [9] 11602 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [10] 11603 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [11] 11604 0
Westmead Hospital - Westmead
Recruitment hospital [12] 11605 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [13] 11606 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [14] 11608 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [15] 11609 0
Gold Coast University Hospital - Southport
Recruitment hospital [16] 11610 0
St Vincent's Hospital (Melbourne) Ltd - Fitzroy
Recruitment hospital [17] 11611 0
Royal Hobart Hospital - Hobart
Recruitment hospital [18] 11612 0
Box Hill Hospital - Box Hill
Recruitment hospital [19] 27281 0
Liverpool Hospital - Liverpool
Recruitment hospital [20] 27282 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [21] 27283 0
Institute for Respiratory Health - Nedlands
Recruitment postcode(s) [1] 23637 0
2305 - New Lambton
Recruitment postcode(s) [2] 23638 0
2050 - Camperdown
Recruitment postcode(s) [3] 23639 0
2139 - Concord
Recruitment postcode(s) [4] 23640 0
2560 - Campbelltown
Recruitment postcode(s) [5] 23641 0
5000 - Adelaide
Recruitment postcode(s) [6] 23642 0
3084 - Heidelberg
Recruitment postcode(s) [7] 23643 0
3004 - Prahran
Recruitment postcode(s) [8] 23644 0
3199 - Frankston
Recruitment postcode(s) [9] 23645 0
3168 - Clayton
Recruitment postcode(s) [10] 23646 0
4102 - Woolloongabba
Recruitment postcode(s) [11] 23647 0
2145 - Westmead
Recruitment postcode(s) [12] 23648 0
5042 - Bedford Park
Recruitment postcode(s) [13] 23649 0
6150 - Murdoch
Recruitment postcode(s) [14] 23651 0
4101 - South Brisbane
Recruitment postcode(s) [15] 23652 0
4215 - Southport
Recruitment postcode(s) [16] 23653 0
3065 - Fitzroy
Recruitment postcode(s) [17] 23654 0
7000 - Hobart
Recruitment postcode(s) [18] 23655 0
3128 - Box Hill
Recruitment postcode(s) [19] 43369 0
2170 - Liverpool
Recruitment postcode(s) [20] 43370 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 300338 0
Commercial sector/Industry
Name [1] 300338 0
GlaxoSmithKline Australia Pty Ltd
Country [1] 300338 0
Australia
Primary sponsor type
Government body
Name
Hunter New England Local Health District
Address
Lookout Road,
New Lambton Heights NSW 2305
Country
Australia
Secondary sponsor category [1] 299777 0
None
Name [1] 299777 0
Address [1] 299777 0
Country [1] 299777 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 301150 0
Hunter New England Human Research Ethics Committee
Ethics committee address [1] 301150 0
Ethics committee country [1] 301150 0
Australia
Date submitted for ethics approval [1] 301150 0
29/09/2016
Approval date [1] 301150 0
07/11/2016
Ethics approval number [1] 301150 0
2019/ETH00986
Ethics committee name [2] 301153 0
Tasmanian Health and Medical Human Research Ethics Committee
Ethics committee address [2] 301153 0
Ethics committee country [2] 301153 0
Australia
Date submitted for ethics approval [2] 301153 0
09/11/2017
Approval date [2] 301153 0
18/12/2017
Ethics approval number [2] 301153 0
H0016979
Ethics committee name [3] 301154 0
South Metropolitan Health Service Human Research Ethics Committee
Ethics committee address [3] 301154 0
Ethics committee country [3] 301154 0
Australia
Date submitted for ethics approval [3] 301154 0
Approval date [3] 301154 0
31/08/2017
Ethics approval number [3] 301154 0
2017-025
Ethics committee name [4] 301155 0
Mater Health Services Human Reserach Ethics Committee
Ethics committee address [4] 301155 0
Ethics committee country [4] 301155 0
Australia
Date submitted for ethics approval [4] 301155 0
Approval date [4] 301155 0
03/01/2017
Ethics approval number [4] 301155 0
HREC/16/MHS/100 Australian Mepolizumab Registry

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 86022 0
Prof Peter Gibson
Address 86022 0
Asthma and Breathing Research Program, Level 2 West Wing, Hunter Medical Research Institute Building Lot 1 Kookaburra Circuit New Lambton Heights, NSW 2305
Country 86022 0
Australia
Phone 86022 0
+61 2 4042 0099
Fax 86022 0
Email 86022 0
peter.gibson@health.nsw.gov.au
Contact person for public queries
Name 86023 0
Erin Harvey
Address 86023 0
Asthma and Breathing Research Program, Level 2 West Wing, Hunter Medical Research Institute Building Lot 1 Kookaburra Circuit New Lambton Heights, NSW 2305
Country 86023 0
Australia
Phone 86023 0
+61 2 4042 0099
Fax 86023 0
Email 86023 0
erin.harvey@health.nsw.gov.au
Contact person for scientific queries
Name 86024 0
Peter Gibson
Address 86024 0
Asthma and Breathing Research Program, Level 2 West Wing, Hunter Medical Research Institute Building Lot 1 Kookaburra Circuit New Lambton Heights, NSW 2305
Country 86024 0
Australia
Phone 86024 0
+61 2 4042 0099
Fax 86024 0
Email 86024 0
peter.gibson@health.nsw.gov.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseMepolizumab effectiveness and identification of super-responders in severe asthma.2020https://dx.doi.org/10.1183/13993003.02420-2019
N.B. These documents automatically identified may not have been verified by the study sponsor.