Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622001004752
Ethics application status
Approved
Date submitted
28/06/2022
Date registered
18/07/2022
Date last updated
16/06/2023
Date data sharing statement initially provided
18/07/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Prospective Observational Study of Mobile App-Based Patient-Reported Outcomes in Patients Treated with Palbociclib for Advanced Breast Cancer in Australia (PIPPA)
Scientific title
Prospective Observational Study of Mobile App-Based Patient-Reported Outcomes in Patients Treated with Palbociclib for Advanced Breast Cancer in Australia (PIPPA)
Secondary ID [1] 307455 0
A5481171
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Breast Cancer 326836 0
Condition category
Condition code
Cancer 324046 324046 0 0
Breast

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The study is a prospective and multicenter study of patients who have recently initiated on Palbociclib for HR+, HER2– Advanced Breast Cancer in Australia. 149 – 199 patients will be recruited into the study through a “hybrid” approach with 50 – 100 patients recruited through site based channels and 99 patients through virtual channels.

All participants are going to be observed for 12 months, recruitment is expected to take 10 months.

Patients will either be recruited by their treating doctor (a.k.a. site-based cohort) or through social media or other virtual channels via an online screening (a.k.a virtual cohort).

This study is being completed using a mobile phone-based app. Participants will be sent an email with instructions on how to download a mobile phone-based app called ‘ClaimIt’ as well as their unique study number. Their email address will be kept separate from their study information.

Before participating in this study, participants will be asked to give their consent to by clicking on the agree button at the end of the electronic document. Participants will be given the option to send the consent to their email address and can access it at any time on the app.

Participants will be asked to complete questionnaires about their health and how they feel upon commencement of the study and again at month 1, month 3, month 6, month 9 and month 12. At each timepoint, the data collection should take no more than 10 - 15 minutes to complete.

Data will be collected through a set of questionnaires that they would complete through the mobile phone app; these questionnaires will include:
• Baseline questionnaire (at commencement only) – demographics (age, gender, ethnicity, family status), medical history and clinical characteristics, and treatment information.
• SF-12 Health Survey – a measure of quality of life. Completed at commencement, month 1, 3, 6, 9 and 12.
• Cancer Care Ontario Edmonton Symptom Assessment System revised version (ESAS) – a measure of the intensity of symptoms, including pain, tiredness, nausea, depression, anxiety, drowsiness appetite, wellbeing, and shortness of breath. Completed at commencement, month 1, 3, 6, 9 and 12.
• Functional Assessment of Cancer Therapy - Breast (FACT-B) – a measure of quality of life by assessing physical, social, emotional, and functional well-being. Completed at commencement, month 1, 3, 6, 9 and 12.
• Whether participants had any dosing changes or interruptions. Completed at month 1, 3, 6, 9 and 12.
• A brief 5-question- experience survey will be issued at month 12 to gather information regarding overall experience in using the app

Participants will receive reminders by email and app notifications on their smartphone when these tasks would need to be completed. If they miss one of their tasks we will enquire as to the reason they missed the latest study activities to understand potential barriers in using these approaches

This research project has been designed to make sure the researchers interpret the results in a fair and appropriate way and avoids bias from study doctors or participants.

There are no costs associated with participating in this research project, nor will participants be paid.

For the site-based cohort in addition the treating doctor (investigator) will also gather the following information:
• A questionnaire at baseline about demographics (age, gender, ethnicity, family status), medical history and clinical characteristics, and treatment information. Collected at commencement only.
• Whether the participant had any dosing changes or interruptions. Collected at month 1, 3, 6, 9 and 12.

Patients recruited through the site based approach will be identified by participating investigators and subsequently referred to the study platform within seven days of palbociclib initiation for eligibility screening and electronic informed consent form (eICF). Investigators or trained site personnel will then complete an electronic case report form (eCRF) to capture baseline patient demographics, medical history and clinical characteristics, and treatment information at enrollment.
Patients initiated onto palbociclib within the last 90 days will be recruited via virtual channels and will be identified through:
1. Targetted study advertisements placed on social media based on a user’s browing history
2. Partnerships with relevant patient associations (e.g., McGrath Foundation, BCNA) who will share study advertisements to patients through their network
Patients enrolling into the study through virtual channels will be screened to ensure eligibility for the study and subsequently referred to the electronic informed consent form (eICF). Screening for the virtual cohort will be a two stage process where patients following an initial study advertisement for patients with breast cancer will be directed to a study landing page that will assess study eligibility (e.g. treatment received for breast cancer, time since initiation, etc.). Patients meeting the study eligibility criteria will then enter their email address which will be used generate a study identification and invitation email. Patients will then be asked to download the app and complete the eICF
Only patients enrolling in the site-based cohort will be asked to complete a questionnaire at palbociclib initiation. Both patients enrolling through virtual channels and patients referred by a HCP will be asked to enter their date of palbociclib initiation which will be used to generate subsequent questionnaires for PROs collection at 1 month, 3 months, 6 months, 9 months and 12 months post palbociclib initiation.
Treatment related information (i.e. dosing, interruptions) will also be assessed at these timepoints for both cohorts. Qualitative information on study participation will be assessed upon study completion or drop-out.
Intervention code [1] 323945 0
Treatment: Devices
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 331836 0
1. Understand the feasibility of generating clinically meaningful PRO data using app-based approaches in patients with HR+, HER2- Advanced Breast Cancer (ABC) receiving palbociclib in combination aromatase inhibitor or fulvestrant per product label.

Feasibility will be determined based on completeness of all questionnaires and patient-reported outcomes instruments assessed through app analytics of data collected at each timepoint as well as adherence, measured as number data collection points completed out of the six data collection timepoints, throughout the study 12-month follow up. For the site-based cohort consistency with physician reported data will be compared with patient-reported data.

Clinically meaningful PROs were designed to be collected (FACT-B, SF-12 and ESAS) along with clinical experts and breast cancer patients to be assessed in a real-world setting
Timepoint [1] 331836 0
baseline, 1 month, 3 months, 6 months, 9 months and 12 months post-enrolment
Primary outcome [2] 331896 0
2. Understand the feasibility of virtual approaches to recruitment for a PRO study in patients with HR+, HER2- ABC receiving palbociclib in combination with an aromatase inhibitor or fulvestrant per product label.

Feasibility of site-less decentralised recruitment will be assessed through evaluation of social media campaigns, screening rates, enrollment rates of eligible participants, data completeness and time-to-dropout data utilising social media, satisfaction 5-question survey at month 12 and app analytics.
Timepoint [2] 331896 0
baseline, 1 month, 3 months, 6 months, 9 months and 12 months post-enrolment
Secondary outcome [1] 411343 0
Describe quality of life in patients with HR+, HER2– ABC receiving palbociclib in combination with an aromatase inhibitor or fulvestrant as per product label

Utilising change in SF-12 Health Survey score – a measure of quality of life
Timepoint [1] 411343 0
Baseline, Month 1, Month 3, Month 6, Month 9 and Month 12 post-enrolment
Secondary outcome [2] 411523 0
Describe symptoms in patients with HR+, HER2– ABC receiving palbociclib in combination with an aromatase inhibitor or fulvestrant as per product label

Utilising change in Cancer Care Ontario Edmonton Symptom Assessment System revised version (ESAS) – a measure of the intensity of cancer symptoms, including pain, tiredness, nausea, depression, anxiety, drowsiness appetite, well-being and shortness of breath
Timepoint [2] 411523 0
Baseline, Month 1, Month 3, Month 6, Month 9 and Month 12 post-enrolment
Secondary outcome [3] 411524 0
Describe quality of life in patients with HR+, HER2– ABC receiving palbociclib in combination with an aromatase inhibitor or fulvestrant as per product label

Utilising change in the Functional Assessment of Cancer Therapy - Breast (FACT-B) score – a measure of quality of life by assessing physical, social, emotional and functional well-being.
Timepoint [3] 411524 0
Baseline, Month 1, Month 3, Month 6, Month 9 and Month 12 post-enrolment
Secondary outcome [4] 411605 0
Describe dosing administration of palbociclib (e.g., reduction, interruptions, duration) using study specific bespoke questionnaire.
Timepoint [4] 411605 0
Month 1, Month 3, Month 6, Month 9 and Month 12 post-enrolment

Eligibility
Key inclusion criteria
1. Has regular access to the ClaimIt virtual trial platform application via Apple iPhone (compatible with operating system versions 13, 12 and 11) or Android phone (compatible with versions 10, 9, 8) and is willing and able to complete data entry through application
2. Adult women or men (greater or equal to 18 years of age) with diagnosis with advanced or metastatic breast cancer not amenable to resection or radiation therapy with curative intent
3. Evidence of HR+ tumor based on the patient’s most recent tumor biopsy as reported by site-based HCP or self-reported by patient
4. Evidence of an HER2– tumor based on the patient’s most recent tumor biopsy as reported by site-based HCP or self-reported by patient
5. Recently intiated on first or second line treatment with one of the following regimens: (i) palbociclib and an aromatase inhibitor as initial endocrine-based therapy for postmenopausal women with advanced or metastatic disease as per product label or (ii) palbociclib with fulvestrant if the patient has experienced disease progression following endocrine therapy as per product label, where recently initiaited is defined as within the last seven days for the site based cohort and last 90 days for the virtual cohort
6. Evidence of an electronically signed and dated informed consent form indicating that the patient has been informed of all pertinent aspects of the study
7. Able to read and understand English
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Patient has initiated palbociclib more than seven days prior to study or enrolment (site-based cohort)
2. Patient has initiated palbociclib more than 90 days prior to study enrolment (virtual cohort)
3. Patient is initiating adjuvant or neoadjuvant systemic therapy
4. The patient is participating in any interventional clinical trial that includes investigational or marketed products. Patients participating in other investigator-initiated research or non-interventional studies can be included as long as their standard of care is not altered by the study
5. The patient is on active treatment for other malignancies other than Advanced Breast Cancer (ABC) or metastatic breast cancer (mBC)
6. In the judgment of the investigator, the patient’s life expectancy is fewer than 180 days (i.e. six months) at the time of diagnosis of ABC or mBC (site-based cohort)

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis
Analyses will generally be descriptive in nature and will be conducted using SAS statistical software (version 9.3 or higher). All baseline variables will be summarized descriptively through tabular displays of mean, median, ranges and standard deviations of continuous variables and frequency distributions of categorical variables.
The analysis will assess enrollment rates, data completeness and time-to-dropout to understand the feasibility of app-based approaches to data collection. Enrollment rates via virtual approaches and completeness of baseline data collection will be assessed to understand feasibility of virtual approaches to recruitment. Descriptive statistics will be reported on all PRO instruments collected throughout study execution, however no formal statistical tests will be applied.

For the purpose of this study, analyses will generally be descriptive in nature and will be conducted using SAS statistical software (version 9.3 or higher).
All baseline variables will be summarized descriptively through tabular displays of mean, median, ranges and standard deviations of continuous variables and frequency distributions of categorical variables.
Objective 1
In order to understand the feasibility of generating clinically meaningful PRO data using app-based approaches in the study population, the analysis will compare enrollment rates with a range of power calculations derived using different assumptions.

The analysis will also report on the proportion of patients completing questionnaires at each time point, the level of completeness of each questionnaire and the time-to-dropout over the study period.

Finally the analysis will report on the qualitative survey data to help interpret the above analysis and find drivers of study participation (and understand how to drive study participation / continuation in future studies).

Objective 2
In order to understand the feasibility of virtual approaches to recruitment for a PRO study the analysis will compare enrollment rates with a range power calculations derived using different assumptions.

The analysis will report on the level of completeness of all baseline variables that would typically be used as clinicially relevant confounders. A characterisation of the baseline characteristics will be compared to the HCP-reported data for the site-based cohort and the self-reported data for the site-based cohort to identify any clinicially meaningful differences.

Objective 3
To describe PROs for both site-based and virtual cohorts, the different instruments will be assessed longitudinally to understand mean, median, ranges and standard deviations of score and relevant sub-scores. Scores will be compared across site-based and virtual cohorts to understand the difference in patient reported outcomes between the groups, however no formal statistical testing will be applied due to differences in recruitment and inability to control for clinically meaningful confounders.
Objective 4
In order to understand patients access to ABC support services the results to the self-reported question on access to ABC support services will be reported in categorical format.
Objective 5
The analysis will report the proportion of patients experiencing dosing reductions and interruptions at each time point, the analysis will be reported for both site-based and virtual cohorts. Kaplan-Meier curves will be constructed to assess treatment duration for both site-based and virtual cohorts.
Detailed methodology for summary and statistical analyses of data collected in this study will be documented in an SAP, which will be dated, filed, and maintained by the sponsor. The SAP may modify the plans outlined in the protocol; any major modifications of primary endpoint definitions or their analyses would be reflected in a protocol amendment.

Recruitment
Recruitment status
Stopped early
Data analysis
No data analysis planned
Reason for early stopping/withdrawal
Participant recruitment difficulties
Date of first participant enrolment
Anticipated
27/07/2022
Actual
19/09/2022
Date of last participant enrolment
Anticipated
27/05/2023
Actual
5/04/2023
Date of last data collection
Anticipated
27/05/2024
Actual
10/05/2023
Sample size
Target
199
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 22650 0
Epworth Richmond - Richmond
Recruitment hospital [2] 22651 0
Liverpool Hospital - Liverpool
Recruitment hospital [3] 22652 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment postcode(s) [1] 37925 0
3121 - Richmond
Recruitment postcode(s) [2] 37926 0
2170 - Liverpool
Recruitment postcode(s) [3] 37927 0
3168 - Clayton

Funding & Sponsors
Funding source category [1] 311726 0
Commercial sector/Industry
Name [1] 311726 0
Pfizer Australia Pty Ltd
Country [1] 311726 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Pfizer Australia Pty Ltd
Address
Level 15-18 Clarence St, Sydney, NSW 2000
Country
Australia
Secondary sponsor category [1] 313186 0
Commercial sector/Industry
Name [1] 313186 0
IQVIA Solutions Australia Pty Ltd
Address [1] 313186 0
Level 8, 201 Pacific Highway, St Leonards, NSW, 2065,
Country [1] 313186 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 311174 0
Bellberry Limited
Ethics committee address [1] 311174 0
Ethics committee country [1] 311174 0
Australia
Date submitted for ethics approval [1] 311174 0
04/02/2022
Approval date [1] 311174 0
28/06/2022
Ethics approval number [1] 311174 0
2022-02-092

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

Contacts
Principal investigator
Name 120214 0
Dr Richard De Boer
Address 120214 0
Epworth Richmond
89 Bridge Rd, Richmond,
VIC 3121
Country 120214 0
Australia
Phone 120214 0
+61 393 471 547
Fax 120214 0
Email 120214 0
[email protected]
Contact person for public queries
Name 120215 0
Richard De Boer
Address 120215 0
Epworth Richmond
89 Bridge Rd, Richmond,
VIC 3121
Country 120215 0
Australia
Phone 120215 0
+61 393 471 547
Fax 120215 0
Email 120215 0
[email protected]
Contact person for scientific queries
Name 120216 0
Richard De Boer
Address 120216 0
Epworth Richmond
89 Bridge Rd, Richmond,
VIC 3121
Country 120216 0
Australia
Phone 120216 0
+61 393 471 547
Fax 120216 0
Email 120216 0
[email protected]

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.