Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623000692639
Ethics application status
Approved
Date submitted
6/06/2023
Date registered
28/06/2023
Date last updated
28/06/2023
Date data sharing statement initially provided
28/06/2023
Type of registration
Prospectively registered

Titles & IDs
Public title
Australian Mepolizumab Registry for Chronic Rhinosinusitis with Nasal Polyps (AMR-CRSwNP)
Scientific title
Australian Mepolizumab Registry for Chronic Rhinosinusitis with Nasal Polyps
Secondary ID [1] 309683 0
protocol 219768
Universal Trial Number (UTN)
Trial acronym
AMR-CRSwNP
Linked study record

Health condition
Health condition(s) or problem(s) studied:
chronic rhinosinusitis with nasal polyps 330058 0
Condition category
Condition code
Inflammatory and Immune System 326960 326960 0 0
Other inflammatory or immune system disorders
Respiratory 327273 327273 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
12
Target follow-up type
Months
Description of intervention(s) / exposure
Observational post-marketing surveillance registry of adults receiving mepolizumab treatment for severe eosinophilic chronic rhinosinusitis with nasal polyps.

Participants will participate in 2 structured computer-assisted telephone interviews with a member of the research team, and complete post-interview questionnaires. These are completed at baseline (prior to commencement of mepolizumab treatment) and again 12 months after mepolizumab commencement. Completion of each interview and the questionnaires will take approximately 2 hours. Data from the participants' medical records are also collected for the baseline and 12 month followup assessments (including nasal polyp scores, sinus surgery details).

The participants' response to mepolizumab treatment will be observed (rhinosinusitis/nasal polyps symptoms, requirement for sinus surgery, health-related quality of life, requirement for systemic corticosteroid treatment, adverse drug-related effects, asthma symptom control for participants with asthma diagnosis)
Intervention code [1] 326128 0
Diagnosis / Prognosis
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334794 0
Change from baseline in nasal obstruction visual analogue scale (VAS)

Participant indicates severity of symptoms. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [1] 334794 0
Baseline and 12 months after commencement of mepolizumab
Primary outcome [2] 334795 0
Change from baseline in overall symptom visual analogue scale (VAS)

Participant indicates Overall Symptom severity on an Overall Symptoms VAS. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [2] 334795 0
Baseline and 12 months after commencement of mepolizumab
Primary outcome [3] 334796 0
Change from baseline in bilateral endoscopic nasal polyp score

Each nostril is scored on a scale of 0 to 4, with the total score being the sum of left and right nostrils (range: 0-8). Higher score indicates worse status.
Timepoint [3] 334796 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [1] 422115 0
Number of systemic corticosteroid treatment courses

Collected directly from participant during participant interview.
Timepoint [1] 422115 0
12 months after commencement of mepolizumab
Secondary outcome [2] 422116 0
Change from baseline in 22-item Sino-Nasal Outcome Test (SNOT-22)

Disease-specific health-related quality of life questionnaire. Participants grade the severity of their symptoms over the previous 2 weeks (0 = No Problem, 1, = Very Mild Problem, 2 = Mild or slight Problem, 3 = Moderate Problem, 4 = Severe Problem, 5 = Problem as bad as it can be). Total score range is 0-110, where higher score is worse.
Timepoint [2] 422116 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [3] 422117 0
Number of sinus surgeries after commencement of mepolizumab
Timepoint [3] 422117 0
12 months after commencement of mepolizumab

Data collected via review of medical records.
Secondary outcome [4] 422233 0
Change from baseline in 5-item Juniper Asthma Control Questionnaire (ACQ-5) score

Participants with asthma only. Assessment of participant's asthma control during the week prior to questionnaire completion. Participant completes questionnaire at baseline assessment (prior to mepolizumab commencement).
Timepoint [4] 422233 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [5] 422234 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] 422234 0
Ongoing during 12 month study period
Secondary outcome [6] 422274 0
Change from baseline in EuroQOL-5Dimension-5Level (EQ-5D-5L) profile

Questionnaire for self-reported health status. Participants rate their health on the day across domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression).
Timepoint [6] 422274 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [7] 423265 0
Change from baseline in nasal discharge visual analogue scale (VAS)

Participant indicates severity of symptoms. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [7] 423265 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [8] 423266 0
Change from baseline in mucus in the throat visual analogue scale (VAS)

Participant indicates severity of symptoms. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [8] 423266 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [9] 423267 0
Change from baseline in loss of smell visual analogue scale (VAS)

Participant indicates severity of symptoms. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [9] 423267 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [10] 423268 0
Change from baseline in facial pain visual analogue scale (VAS)

Participant indicates severity of symptoms. VAS from 0 to 10 (10 = worst outcome), participant identifies point on line.
Timepoint [10] 423268 0
Baseline and 12 months after commencement of mepolizumab
Secondary outcome [11] 423310 0
Time to first sinus surgery after commencement of mepolizumab
Timepoint [11] 423310 0
12 months after commencement of mepolizumab

Data collected via review of medical records.
Secondary outcome [12] 423311 0
Change from baseline in EuroQOL-5Dimension-5Level (EQ-5D-5L) VAS

Questionnaire for self-reported health status. Participants rate overall assessment of their health via visual analogue score (VAS). VAS from 0 to 100 (0 = worst health imaginable).
Timepoint [12] 423311 0
Baseline and 12 months after commencement of mepolizumab.

Eligibility
Key inclusion criteria
Participants will have a confirmed diagnosis of severe CRSwNP with eosinophilic inflammation. They will be eligible for assessment for commencement of Pharmaceutical Benefits Scheme (PBS)-subsidised mepolizumab per below criteria #3-7 (OR eligible to commence mepolizumab for CRSwNP outside of the PBS restrictions)

1. Able to provide informed consent
2. Age greater than or equal to 18 years
3. Diagnosis of CRSwNP confirmed by direct nasal examination (OR diagnosis from at least 2 physicians/ENTs experienced in management of CRSwNP)
4. Peripheral blood eosinophil (PBE) count of greater than or equal to 300 cells/µL
5. Adherence to intranasal corticosteroid therapy for at least 2 months (unless contraindicated or not tolerated)
6. Previous nasal polyps surgery; OR details of surgical exception
7. At least 2 of the following:
-Baseline bilateral nasal polyp (NP) score of greater than or equal to 5 (out of maximum score of 8, with minimum score of 2 in each nasal cavity)
-Baseline nasal obstruction visual analogue score (VAS) of > 5 (out of 10)
-Baseline overall symptom visual analogue score (VAS) of > 7 (out of 10)
8. Able to access a telephone and/or computer with internet
Minimum age
18 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 data collection

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 registry, such as CRS/NP characteristics, and treatment for baseline data. Mepolizumab treatment clinical response variables will be summarised (eg. nasal obstruction VAS, overall symptoms VAS, requirement for surgery, bilateral endoscopic NP score).

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 categorised by response; comorbidity, phenotypic characteristics. Analysis of normally distributed data will be performed using the unpaired Student’s t test for comparison between two groups. Analysis of non-parametric data will be performed using the Wilcoxon rank sum test for comparison between two groups or the Kruskal-Wallis test with post-hoc test applied to correct for multiple comparisons. Chi2 or Fisher’s exact test will be used to analyse categorical data.


Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/07/2023
Actual
Date of last participant enrolment
Anticipated
31/12/2024
Actual
Date of last data collection
Anticipated
31/12/2025
Actual
Sample size
Target
140
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 24760 0
John Hunter Hospital - New Lambton
Recruitment hospital [2] 24761 0
Concord Repatriation Hospital - Concord
Recruitment hospital [3] 24762 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [4] 24763 0
St George Hospital - Kogarah
Recruitment hospital [5] 24765 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [6] 24766 0
Royal Brisbane & Womens Hospital - Herston
Recruitment hospital [7] 24767 0
The Queen Elizabeth Hospital - Woodville
Recruitment hospital [8] 24768 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [9] 24771 0
St Vincent's Private Hospital (Darlinghurst) - Darlinghurst
Recruitment hospital [10] 24772 0
The Royal Victorian Eye and Ear Hospital - East Melbourne
Recruitment hospital [11] 24868 0
Sydney ENT Clinic - Darlinghurst
Recruitment hospital [12] 24875 0
The ENT Centre - Hornsby
Recruitment hospital [13] 24876 0
Sydney Sinus and Allergy Centre - Edgecliff
Recruitment hospital [14] 24877 0
Sydney Centre for Ear Nose and Throat - East Frenchs Forest
Recruitment hospital [15] 24878 0
Perth ENT Centre - Subiaco
Recruitment hospital [16] 24879 0
St Vincent's Clinic - Darlinghurst
Recruitment postcode(s) [1] 40402 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 40527 0
2027 - Edgecliff
Recruitment postcode(s) [3] 40394 0
2050 - Camperdown
Recruitment postcode(s) [4] 40526 0
2077 - Hornsby
Recruitment postcode(s) [5] 40528 0
2086 - East Frenchs Forest
Recruitment postcode(s) [6] 40393 0
2139 - Concord
Recruitment postcode(s) [7] 40395 0
2217 - Kogarah
Recruitment postcode(s) [8] 40392 0
2305 - New Lambton
Recruitment postcode(s) [9] 40403 0
3002 - East Melbourne
Recruitment postcode(s) [10] 40400 0
3050 - Parkville
Recruitment postcode(s) [11] 40398 0
4029 - Herston
Recruitment postcode(s) [12] 40399 0
5011 - Woodville
Recruitment postcode(s) [13] 40529 0
6008 - Subiaco
Recruitment postcode(s) [14] 40397 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 313877 0
Commercial sector/Industry
Name [1] 313877 0
GlaxoSmithKline
Country [1] 313877 0
United Kingdom
Primary sponsor type
University
Name
The University of Newcastle
Address
The University of Newcastle
University Drive, Callaghan, New South Wales 2308, Australia
Country
Australia
Secondary sponsor category [1] 315784 0
None
Name [1] 315784 0
Address [1] 315784 0
Country [1] 315784 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 313024 0
Hunter New England Human Research Ethics Committee
Ethics committee address [1] 313024 0
HNE Research Office, Hunter New England Local Health District,
Level 3, POD, Hunter Medical Research Institute,
Lot 1 Kookaburra Circuit, New Lambton Heights NSW 2305,
Australia.
Ethics committee country [1] 313024 0
Australia
Date submitted for ethics approval [1] 313024 0
28/04/2023
Approval date [1] 313024 0
18/05/2023
Ethics approval number [1] 313024 0
2023/ETH00846

Summary
Brief summary
The Australian Mepolizumab Registry for Chronic Rhinosinusitis with Nasal Polyps (AMR-CRSwNP) will be an electronic registry of well characterised patients with severe eosinophilic CRSwNP who receive mepolizumb treatment. It will be a national, multi-centre, observational post-marketing surveillance registry. Patients will receive mepolizumab via the Australian Commonwealth Government Pharmaceutical Benefits Scheme (PBS) or outside of the PBS restrictions.
Patients will be identified for participation across multiple clinics in Australia. The central registry team will manage participant recruitment and data collection via a central telehealth model. Participants will be characterised at baseline (before starting mepolizumab) and have 12 months follow-up.
The registry will provide insight into CRSwNP patient characteristics , report on mepolizumab use (effectiveness, safety) and be a databank for future research.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 126722 0
Prof Peter Gibson
Address 126722 0
Department of Respiratory & Sleep Medicine, John Hunter Hospital,
Asthma and Breathing Research Program,
Level 2 West Wing, Hunter Medical Research Institute, New Lambton Heights, NSW
C/- John Hunter Hospital
Locked Bag 1, HRMC NSW 2310
Country 126722 0
Australia
Phone 126722 0
+61 2 4042 0143
Fax 126722 0
+61 2 4042 0046
Email 126722 0
peter.gibson@health.nsw.gov.au
Contact person for public queries
Name 126723 0
Prof Peter Gibson
Address 126723 0
Department of Respiratory & Sleep Medicine, John Hunter Hospital,
Asthma and Breathing Research Program,
Level 2 West Wing, Hunter Medical Research Institute, New Lambton Heights, NSW
C/- John Hunter Hospital
Locked Bag 1, HRMC NSW 2310
Country 126723 0
Australia
Phone 126723 0
+61 2 4042 0143
Fax 126723 0
+61 2 4042 0046
Email 126723 0
peter.gibson@health.nsw.gov.au
Contact person for scientific queries
Name 126724 0
Prof Peter Gibson
Address 126724 0
Department of Respiratory & Sleep Medicine, John Hunter Hospital,
Asthma and Breathing Research Program,
Level 2 West Wing, Hunter Medical Research Institute, New Lambton Heights, NSW
C/- John Hunter Hospital
Locked Bag 1, HRMC NSW 2310
Country 126724 0
Australia
Phone 126724 0
+61 2 4042 0143
Fax 126724 0
+61 2 4042 0046
Email 126724 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
No additional documents have been identified.