A Randomised, Crossover Study of Self-monitoring of Symptoms and Spirometry Via the patientMpower Platform in Patients With Idiopathic Pulmonary Fibrosis
Phase: NA
Conditions: Idiopathic Pulmonary Fibrosis
Sponsor: patientMpower Ltd.

 
Confidential ©patientMpower 
 
A pilot-scale observational study of self-monitoring of 
symptoms and spirometry via the patientMpower 
platform in patients with idiopathic pulmonary 
fibrosis. 
 
IPF patientMpower 02 
 
Draft protocol date: 12 July 2017 
 
Number of pages: 31 
 
Version 2.0 
 
Authors: 
 
Colin Edwards PhD, patientMpower, Dublin, Ireland. 
 
Eamonn Costello, patientMpower, Dublin, Ireland. 
 
Prof. Anthony O’Regan, Galway University Hospital, Galway, Ireland. 
 
NCT 03104322 
 
 
 

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Table of Contents 
PROTOCOL SUMMARY .................................................................................... 4 
FLOW CHART .................................................................................................... 6 
List of Tables ..................................................................................................... 8 
List of Figures ................................................................................................... 9 
Figure 3.2: 1:  Diagram of study design ................................................................... 9 
List of Abbreviations ...................................................................................... 10 
1.  
Introduction .......................................................................................... 11 
1.1  
Medical background .................................................................................. 11 
1.2  
Product profile ........................................................................................... 11 
2. 
Rationale, objectives and benefit-risk assessment ............................ 12 
2.1  
Rationale for performing the trial ............................................................ 12 
2.2  
Trial objectives ........................................................................................... 13 
2.3  
Benefit-risk assessment ........................................................................... 13 
3.  
Description of design and trial population ...................................... 13 
3.1  
Overall design and plan ............................................................................ 13 
3.1.1  
Administrative structure of trial ................................................................. 13 
3.2  
Discussion of trial design, including choice of control group ............. 14 
Figure 3.2: 1  Diagram of study design .................................................................. 14 
3.3  
Selection of trial population ..................................................................... 15 
3.3.1  
Main diagnosis for study entry .................................................................. 15 
3.3.2  
Inclusion criteria .......................................................................................... 15 
3.3.3  
Exclusion criteria ......................................................................................... 15 
3.3.4  
Removal of patients from study or assessments ................................... 16 
4.  
Treatments ............................................................................................ 16 
4.1  
Study intervention ..................................................................................... 16 
4.1.1  
Identity of study intervention ..................................................................... 16 
4.1.2  
Method of assigning patients to study intervention ............................... 17 
4.1.3  
Blinding and procedures for unblinding ................................................... 17 
4.2  
Concomitant therapy, restrictions and rescue treatment ..................... 17 
4.2.1  
Rescue medication, emergency procedures and additional treatment
 
17 
4.2.2  
Restrictions .................................................................................................. 17 
4.3  
Treatment compliance ............................................................................... 18 
5  
Variables and their assessment ............................................................ 18 
5.1  
Efficacy ....................................................................................................... 18 
5.1.1  
Efficacy endpoints ...................................................................................... 18 
5.1.2  
Assessment of efficacy .............................................................................. 21 
5.2  
Safety........................................................................................................... 21 
5.2.1  
Safety endpoints ......................................................................................... 21 
5.3.  
Other variables ........................................................................................... 22 
5.3.2  
Other assessments .................................................................................... 22 
5.4  
Appropriateness of measurements ......................................................... 22 
6 
Investigational plan ................................................................................. 23 
6.1  
Visit schedule ............................................................................................. 23 

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6.2  
Details of trial procedures at selected visits .......................................... 23 
6.2.1  
Baseline clinic visit ...................................................................................... 23 
6.2.2  
Treatment and observation period ........................................................... 24 
6.2.3 
8-week clinic visit ............................................................................................ 25 
6.2.4  
End of Study clinic visit ( at least 16 weeks) .......................................... 25 
7.  
Statistical methods and determination of sample size .................. 26 
7.1  
Statistical design and model .................................................................... 26 
7.2  
Null and alternative hypotheses .............................................................. 26 
7.3  
Planned analyses ....................................................................................... 26 
7.3.1  
Primary analyses ........................................................................................ 26 
7.3.2  
Secondary analyses ................................................................................... 26 
7.3.3  
Safety analyses ........................................................................................... 26 
7.3.4 
 Interim analyses ............................................................................................. 26 
7.3.5  
Health economic analyses ........................................................................ 27 
7.4  
Handling of missing data .......................................................................... 27 
7.5.  
Randomisation ........................................................................................... 27 
7.6  
Determination of sample size ................................................................... 27 
8.  
Informed consent, data protection and trial records ...................... 27 
8.1  
Study approval, patient information and informed consent ................ 27 
8.2  
Data quality assurance .............................................................................. 27 
8.3  
Records ....................................................................................................... 27 
8.3.1  
Source documents ...................................................................................... 27 
8.3.2  
Direct access to source data and documents ........................................ 28 
8.3.3  
Storage of records ...................................................................................... 28 
8.4  
Statement of confidentiality ..................................................................... 28 
8.5  
Completion of trial ..................................................................................... 29 
8.6  
Protocol violations..................................................................................... 29 
8.7  
Compensation available to the patient in the event of trial-related 
injury 29 
9 
References ............................................................................................... 29 
10  
Appendices ........................................................................................... 31 
10.1  
modified Medical Research Council Dyspnoea Scale ........................... 31 
10.2 
Instructions for performing spirometry at home ................................... 31 
11  
Summary of clinical trial protocol modifications ............................ 31 
 
 
 

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PROTOCOL SUMMARY 
 
PRODUCT 
patientMpower platform 
CLINICALTRIALS.GOV IDENTIFIER NCT 03104322 
PROTOCOL TITLE 
A pilot-scale observational study of 
self-monitoring of symptoms and 
spirometry via the patientMpower 
platform in patients with idiopathic 
pulmonary fibrosis 
CO-ORDINATING INVESTIGATOR 
Prof. Anthony O’Regan, Galway 
University Hospital 
NUMBER OF TRIAL SITES 
One 
CLINICAL PHASE 
Not applicable 
STUDY OBJECTIVES 
Determine the  
• acceptability of the 
patientMpower platform from 
patient and healthcare 
professional perspective  
• the impact of active 
engagement and self-
monitoring using 
patientMpower on Patient 
Reported Outcome Measures 
(PROMs) in IPF 
• impact of patientMpower 
platform on medication 
compliance. 
• correlation between patient-
reported measures (PROM, 
spirometry) and clinical 
measures and outcomes 
METHODOLOGY 
Single-centre, prospective, open-
label, usual care controlled, open, 
fixed-order, two-period crossover 
observational study  
NUMBER OF SUBJECTS 
Total randomised: 8 
Each treatment  
patientMpower platform followed by 
usual care observation: 8 
 
DIAGNOSIS  
Confirmed diagnosis of IPF 
MAIN CRITERIA FOR INCLUSION 
Age ≥40 years, daily access to 
smartphone or tablet device, written 
informed consent 
TEST PRODUCT 
patientMpower platform (via 
smartphone or tablet device) 
COMPARATOR PRODUCT 
Usual care 
DURATION OF OBSERVATION 
Sixteen weeks (two periods of eight 
weeks) 

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END OF STUDY DEFINITION 
Sixteen weeks 
PRIMARY ENDPOINTS 
Acceptability of the patientMpower 
platform from patient and healthcare 
professional perspective. 
SECONDARY ENDPOINTS 
Impact of active engagement and 
self-monitoring using the 
patientMpower platform on PROMs 
in IPF 
Impact of using the patientMpower 
platform on exercise performance 
(e.g. walking distance) 
Effect of the patientMpower platform 
on medication compliance 
Correlation between patient-reported 
measures (PROMs, spirometry) and 
clinical measures and outcomes 
INTERIM ANALYSIS 
None planned 
STATISTICAL METHODS 
Descriptive statistics tables will be 
prepared 
 
 
 
 

 
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FLOW CHART 
 
 
Baseline 
(clinic visit) 
Daily (patient 
-reported) 
2 days 
(telephone 
call) 
14 days 
(telephone 
call) 
8 weeks 
(clinic visit) 
End of study 
12-16 weeks 
(clinic visit) 
 
Period 1: patientMpower platform (~8 weeks) + Period 2: usual care (~8 weeks) 
 
Period 1 
Period 2 
Informed consent and randomisation 
X 
 
 
 
 
 
Spirometry, dyspnoea1, 6-minute walk 
distance 
X 
 
 
 
X 
X 
Demographic data (include IPF & medicines 
history) 
X 
 
 
 
 
 
Instruction on patientMpower platform, 
home spirometry and start using platform 
X 
 
 
 
 
 
Patient training & encouragement 
X 
 
X 
X 
 
 
2Record compliance/changes (IPF 
medicine) 
X 
X 
X 
X 
X 
X 
2Patient-measured FVC 
X 
X 
X 
X 
X 
 
Record impact of IPF (PROM)3 
X 
X 
X 
X 
X 
X 
4Patient-reported outcomes (e.g. oxygen 
consumption, dyspnoea, pulse oximetry, 
cough, vital signs) 
 
X 
 
 
 
 
Stop use of patientMpower platform 
 
 
 
 
X 
 
Utility & acceptability of platform5 
 
 
 
 
X 
 
Clinic-reported outcomes (e.g. 
exacerbations) 
 
 
 
 
 
X 
End of study 
 
 
 
 
 
X 

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1 Modified Borg scale used to assess dyspnoea in clinic. 
2 Reported by patient on patientMpower platform every day during Period 1 (up to 8 weeks). Assessed by clinic at end of  Period 1 
and Period 2. 
3 Impact of IPF on daily life (patient reported outcome measure) to be assessed at clinic at baseline, at the end of Period 1 and and 
at the end of Period 2 (i.e. end of  study) and recorded on the patientMpower platform. Impact of IPF on daily life (patient reported 
outcome measure) to be reported by the patients on patientMpower platform at start of Period 1 and then every week during Period 
1. 
4 Reported by patient as often as possible, ideally each day 
5Patient and healthcare professional perspective. If patient is withdrawn prematurely, try to capture patient perspective of utility and 
acceptability of platform at time of withdrawal. 
 

 
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List of Tables 
 
None 
 
 

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List of Figures 
 
Figure 3.2: 1:  
Diagram of study design 
 
 
 
 
 
 
 

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List of Abbreviations 
 
ATS  
American Thoracic Society 
DLCO  
Diffusing Capacity of the Lung for Carbon Monoxide 
ERS  
European Respiratory Society 
FEV1  
Forced Expiratory Volume in 1 second 
FVC  
Forced Vital Capacity 
IP 
 
Internet Protocol 
IPF 
 
Idiopathic pulmonary fibrosis 
JRS 
 
Japanese Respiratory Society 
K-BILD 
King’s Brief Interstitial Lung Disease Questionnaire 
mMRC 
modified Medical Research Council 
PEF  
Peak Expiratory Flow 
PROM 
 
patient reported outcome measure 
SSH  
Secure Shell 
SSL 
 
Secure Sockets Layer 
TLS 
 
Transport Layer Security 
 
 

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1.  
Introduction 
1.1  
Medical background 
 
Idiopathic pulmonary fibrosis (IPF) is an irreversible lung disease leading to 
progressive dyspnoea and a deterioration of pulmonary function reflected by a 
progressive decline of forced vital capacity (FVC) and diffusing capacity of the 
lung for carbon monoxide (DLCO). Progression of the disease results in a 
serious limitation of physical activities and has a major impact on the patient’s 
quality of life [1].  
 
Digital medicine platforms which directly record patient experiences (e.g. 
symptoms, impact on daily life, medication compliance) and measurements 
(e.g. blood pressure, blood sugar levels) have been developed for chronic 
medical conditions and may be valuable in helping the patients to manage 
their condition. To date, no digital platform has been specifically developed for 
patients with IPF. 
 
At the time of writing, there are few simple disease-specific instruments to 
assess health-related quality of life in patients with IPF. A modified version of 
the St. George’s Respiratory Questionnaire has been developed for IPF [2]. 
Another group has developed and tested the King’s Brief Interstitial Lung 
Disease Questionnaire (K-BILD) in an interstitial lung disease population [3,4]. 
The Brompton Hospital, London has recently developed a patient reported 
outcome measure (PROM) specifically to capture the impact of IPF on the 
patients’ daily life and this is currently being evaluated [5]. This PROM can be 
used on a very regular basis (e.g. every week) and could provide information 
on longitudinal trends on the impact of IPF on daily life. It is possible that 
patient-reported information may be more useful than clinic-reported 
assessments in characterizing the impact of IPF on quality of life.  
 
1.2  
Product profile 
 
The patientMpower platform is a digital medicine platform which has been 
developed for and is used in providing support to patients with breast cancer, 
prostate cancer and post-renal transplant. The platform is downloaded as an 
app to a mobile phone or tablet device and patients can record various 
parameters (e.g. blood pressure, symptoms, medication compliance, impact 
of their medical condition on daily life) on a regular basis (sometimes daily). 
The patient has a permanent health diary of their self-reported measurements 
available to them on their mobile device. The platform includes a health 
journal which allows patients to record symptoms at the time they occur. This 
is helpful for the patient in monitoring their health and in preparing them for 
appointments with their healthcare professionals, particularly if there is a long 
interval between clinic visits. If appropriate, certain clinic-derived 
measurements (e.g. therapeutic drug levels, spirometry data) can be shared 
with the patient via the platform.  
 

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A version of the patientMpower platform has been developed for renal 
transplant recipients and this is now offered as standard care at the national 
renal transplantation centre in Ireland. It has been positively received by 
patients and healthcare professionals and studies of its use are ongoing. 
However, it is important to recognise that patient demographic factors vary by 
medical condition and this can have an impact on the utility and acceptability 
of digital health platforms in the patient user group. 
 
This version of the patientMpower platform has specifically been developed to 
capture parameters which are relevant for patients with IPF. Examples of 
measures which can be recorded by the patient include dyspnoea [modified 
Medical Research Council (mMRC) Dyspnoea Scale], activity (steps/day), 
vital signs, temperature, oxygen use, type of activity linked to maximum 
dyspnoea, medication compliance and cough. In addition, patients can report 
measures linked to the recently developed PROM specifically developed to 
capture the impact of IPF on patients’ daily life [5].  
 
A recent study in patients with IPF has suggested that daily home monitoring 
of FVC by patients is clinically informative and daily FVC may be of value as a 
primary endpoint in short proof-of-concept studies [6]. The patientMpower 
platform can link to home spirometry devices to allow longitudinal collection 
and upload of long-term patient-measured FVC data to the platform. The 
Spirobank Smart spirometer (Medical International Research, Via del 
Maggiolino 125, 00155 Roma, Italy. www.spirometry.com) is one example of a 
home spirometer which can collect and wirelessly upload FVC and other 
spirometry data to the patientMpower platform. 
 
This study will capture longitudinal data on home measurement (by the 
patient) of FVC and patient-reported outcomes within the same cohort of 
patients. This will enable assessment of the degree of correlation between the 
two types of measurement.  
 
2. 
Rationale, objectives and benefit-risk assessment 
2.1  
Rationale for performing the trial 
 
There are few data on longitudinal trends in patient reported outcomes and 
patient-measured FVC in IPF. It is possible that these data may be predictive 
of important health outcomes (e.g. exacerbations). The patientMpower 
platform provides a tool to collect and share this type of information between 
patients and their healthcare professionals. 
 
In addition, as no specific digital patient support platform for patients with IPF 
is available and validated, there is sufficient justification in testing the 
effectiveness and acceptability of the patientMpower platform in a controlled 
observational setting. 
 

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2.2  
Trial objectives 
 
The objectives of this observational study of patientMpower in IPF are: 
• Acceptability of the patientMpower platform from patient and healthcare 
professional perspective  
• Impact of active engagement and self-monitoring using patientMpower 
on PROMs in IPF. 
• Impact of patientMpower platform on medication compliance.  
• Correlation between patient-reported measures (PROMs, spirometry) 
and clinical measures and outcomes. 
 
2.3  
Benefit-risk assessment 
 
The patientMpower platform has been evaluated in other clinical settings (e.g. 
prostate cancer, post renal transplant). The renal transplant version of the 
patientMpower platform is now offered as standard care at the national renal 
transplantation centre in Ireland. It has been positively received by patients 
and healthcare professionals and studies of its use are ongoing. 
 
It is not expected that the study or patientMpower platform will create any 
additional risks for IPF patients. 
3.  
Description of design and trial population 
3.1  
Overall design and plan 
 
This is an open-label, prospective, usual care controlled, open, fixed-order 
two-period crossover observational study of a population of IPF patients. 
Patients who enter the study will be trained in correct use of the 
patientMpower platform and the Spirobank Smart home spirometer and 
encouraged to use them on a daily basis. The study will not make any other 
changes to the therapeutic interventions offered to the patients. 
3.1.1  Administrative structure of trial 
 
This is a single centre study at a tertiary centre in Ireland with expertise in the 
treatment of IPF. 
 
The study is sponsored by an unrestricted research grant from the Quality 
Innovation Corridor Digital Programme of eHealth Ireland. The patientMpower 
platform was developed and is owned by patientMpower, Dublin 8, Ireland. 
The Spirobank Smart home spirometer was developed by Medical 
International Research, Via del Maggiolino 125, 00155 Roma, Italy. The 
protocol was designed by patientMpower Ltd. and Prof. Anthony O’Regan, 
Galway University Hospital, the principal investigator. 
 
 

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3.2  
Discussion of trial design, including choice of control group 
 
 
This is an open-label, prospective, usual care controlled, open, fixed-order 
two-period crossover observational study of a population of IPF patients. 
 
The treatment sequence is period 1: patientMpower platform and usual care 
followed by period 2: usual care alone. The design is illustrated in Figure 3.2: 
1 below: 
 
Figure 3.2: 1  
Diagram of study design 
 
 
The control treatment is usual care alone. The active treatment is 
patientMpower platform and usual care. 
 
This design allows comparison of the effect of the patientMpower platform 
with usual care within the same patient.  
 
Each treatment period is eight weeks. The total observation time is 
approximately sixteen weeks (i.e. two treatment periods of eight weeks). In 
routine practice, patients with IPF are seen at the clinic at intervals of 
approximately sixteen weeks (similar to the duration of the study). 
 
It is not anticipated that there are any carryover effects ofthe patientMpower 
platform so there is no washout between treatment periods 1 and 2. 
 
Patients enrolled in the study will capture information on relevant health 
outcomes related to IPF on a regular basis (daily for some parameters) using 
the patientMpower platform. Patients will also measure FVC at home once per 
day using the Spirobank Smart spirometer (ideally at approximately the same 
time each day). The patient measured FVC and other spirometry data will be 
captured automatically by the patientMpower platform. These data will form 
the patient-reported outcome database. 
 
Clinical assessments (e.g. FVC, 6-minute walking distance, impact of IPF on 
daily life) in the same cohort of patients will be assessed at the beginning and 
end of the study. 

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This design allows comparison of patient-reported data and their longitudinal 
trends with clinic reported data in a cohort of patients with IPF. The correlation 
between the two types of data and the value of patient measurements in 
predicting health outcomes can be assessed and quantified.   
 
This design will also allow evaluation of the utility and acceptability of the 
patientMpower in patients with IPF. This will be assessed from both the 
patient and healthcare professional perspectives. 
 
The patientMpower platform will include key questions derived from a PROM 
developed specifically for patients with IPF [5] and will help to evaluate the 
utility of an electronic health diary in capturing PROM data in IPF patients. 
 
3.3  
Selection of trial population 
 
3.3.1  Main diagnosis for study entry 
 
Idiopathic pulmonary fibrosis 
3.3.2  Inclusion criteria 
 
Aged at least 40 years 
 
Confirmed diagnosis of IPF [according to American Thoracic Society (ATS), 
European Respiratory Society (ERS) or Japanese Respiratory Society (JRS) 
criteria] [7]. 
 
Has daily unrestricted access to a suitable smartphone or tablet device at 
home.  
 
Demonstrate understanding of the protocol requirements, and correct use of 
the Spirobank Smart spirometer and the patientMpower platform. 
 
Able and willing to perform spirometry every day at home. 
 
Willing to give written informed consent. 
 
3.3.3  Exclusion criteria 
 
Significant confusion or any concomitant medical condition which would limit 
the ability of the patient to record symptoms or use a home spirometer on a 
regular basis. 
 
New prescription of antifibrotic therapy for IPF (e.g. pirfenidone, nintedanib) 
within the four weeks before the baseline visit. 
 

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Recent exacerbation of IPF or other clinically significant change in the 
patient’s medical condition in the four weeks before the baseline visit. 
3.3.4  Removal of patients from study or assessments 
 
3.3.4.1  
Removal of individual patients 
 
Patients are free to withdraw from the study at any time without any impact on 
their ongoing medical care. 
 
The investigator may withdraw a patient from the study at any time if they 
believe that further participation in the study is not in the best interests of the 
patient. 
 
3.3.4.2  
Discontinuation of the study by the sponsor. 
 
The study sponsor may terminate the study early if recruitment is significantly 
behind schedule or if for any other reason, it is unlikely that the study can be 
completed. 
 
4.  
Treatments 
 
All patients will continue to receive all usual care throughout the study as 
prescribed by their healthcare professionals.  
 
4.1  
Study intervention 
 
4.1.1  Identity of study intervention 
 
The study intervention is a digital medicine platform, patientMpower. This 
version has been developed specifically for patients with IPF. The platform is 
an electronic application downloaded to the patient’s mobile phone or tablet 
device. The application is designed to allow the patient to report various 
parameters relevant to IPF and record these on a regular basis, ideally daily. 
The information recorded by the patient will be stored in a secure cloud 
system and will be available to the patient through their phone or mobile 
device at all times. No personal health data are stored on the phone or mobile 
device itself. 
 
Patients will be asked to report measurements on the patientMpower platform 
each day. Patient-reported measures (at a minimum) will include FVC (one 
reading once/day), dyspnoea, activity (steps/day), distance walked per day 
and compliance with IPF medication. In addition, the impact of IPF on daily life 
(as response to PROM questions) should be reported every week.  
 

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Additional patient-reported measures which can be reported on the 
patientMpower platform include vital signs (e.g. heart rate), temperature, 
pulse oximetry, cough severity, activity causing worst dyspnoea and oxygen 
consumption. These are optional measurements and will only be recorded 
where practical for the patients. 
 
4.1.2  Method of assigning patients to study intervention 
 
All patients who give informed consent will be allocated to the treatment 
sequence described in section 3.2 above. 
 
Patients will be given training on the correct use of the patientMpower 
platform and Spirobank Smart home spirometer at the start of the 
patientMpower platform + usual care period and will be encouraged to use 
them on a daily basis. 
 
4.1.3  Blinding and procedures for unblinding 
 
The study is open-label.  
 
4.2  
Concomitant therapy, restrictions and rescue treatment 
4.2.1  Rescue medication, emergency procedures and additional 
treatment 
 
4.2.1.1  
Management of acute exacerbations 
 
Any exacerbations of the patient’s IPF or any other underlying medical 
condition(s) should be treated according to standard procedures. 
4.2.1.2  
Management of other adverse events 
 
Any other adverse events should be treated according to standard 
procedures. 
4.2.2  Restrictions 
4.2.2.1  
Restrictions on concomitant treatment 
 
There are no restrictions on concomitant treatment. All concomitant 
treatments as prescribed by the patient’s healthcare professionals are 
allowed. Patients will continue to take all medicines as prescribed by their 
healthcare professionals. 
 
 

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4.2.2.2  
Restrictions on diet and life-style 
 
There are no restrictions on diet or life-style. Patients will continue to follow all 
instructions on diet, exercise and lifestyle as directed by their healthcare 
professionals. 
 
4.3  
Treatment compliance 
 
Patients will use the patientMpower platform to record daily compliance with 
medications prescribed for treatment of their IPF. 
 
5  
Variables and their assessment 
 
5.1  
Efficacy 
 
The objectives of this observational study of patientMpower in IPF are to 
determine the: 
• Acceptability of the patientMpower platform from patient and healthcare 
professional perspective.  
• Impact of active engagement and self-monitoring using patientMpower 
on PROMs in IPF. 
• Impact of patientMpower platform on medication compliance.  
• Correlation between patient-reported measures (PROMs, spirometry) 
and clinical measures and outcomes. 
 
5.1.1  Efficacy endpoints 
 
5.1.1.1  
Primary endpoint 
 
The primary endpoint is the acceptability of the patientMpower platform from 
patient and healthcare professional perspective.  
 
The primary endpoint variables (from the patient perspective) will be assessed 
by their response to these questions:  
 
• the instructions given in using the patientMpower platform were clearly 
understandable (strongly agree/agree/disagree/strongly disagree) 
• using the patientMpower platform helped me to take the correct dose of 
my medicines for lung fibrosis every day (strongly 
agree/agree/disagree/strongly disagree) 
• using the patientMpower platform helped me to take my medicines for 
lung fibrosis at the correct time every day (strongly 
agree/agree/disagree/strongly disagree) 

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• using the patientMpower platform helped me to reach my personal 
exercise goal every day (strongly agree/agree/disagree/strongly 
disagree) 
• using the patientMpower platform helped me to walk further (or 
exercise more) compared with before (strongly 
agree/agree/disagree/strongly disagree) 
• using the patientMpower platform gave me more confidence/a greater 
sense of control in managing my lung health (strongly 
agree/agree/disagree/strongly disagree) 
• I found it useful to be able to record the impact of lung fibrosis on my 
daily life (strongly agree/agree/disagree/strongly disagree) 
• using the patientMpower platform encouraged me to look at the 
informational videos on the platform (strongly 
agree/agree/disagree/strongly disagree) 
• my preference for using the patientMpower platform versus not using it 
(yes, no preference, no) 
• my difficulty rating in using the patientMpower platform (very easy, 
easy, difficult, very difficult) 
• what was the effect of using the patientMpower platform on the impact 
of lung fibrosis on my well-being and daily life (positive, negative, 
optional open text field for participant to give opinion) 
• I found it tiring or irritating to use the patientMpower platform (strongly 
agree/agree/disagree/strongly disagree) 
• do I want to continue using the patientMpower platform after the end of 
the study (yes, no) 
• I would recommend other people with my condition to use the 
patientMpower platform (yes/no) 
• what other measurements, reminders or information would be useful to 
have on the patientMpower platform? (optional open text for patient to 
give opinion) 
• describe the benefits and/or disadvantages of using the patientMpower 
platform (optional open text for patient to give opinion) 
• any other comments on the patientMpower platform (optional open text 
field for participant to give opinion) 
 
The primary endpoint variable (from the healthcare professional perspective) 
will be assessed by their response to these questions:  
 
• preference for using the patientMpower platform versus not using it 
(yes, no preference, no) 
• difficulty rating in using the patientMpower platform (very easy, easy, 
difficult, very difficult) 
• did using the patientMpower platform help me to help the patient 
manage their IPF better? (yes, no) 
• did using the patientMpower platform help the patient to take their IPF 
medicines at the correct dose every day (yes, no) 
• do I believe the patient should continue using the patientMpower 
platform after the end of the study (yes, no) 

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• what other measurements, reminders or information would be useful to 
have on the patientMpower platform? (Open text for healthcare 
professional to give opinion) 
• describe the benefits and/or disadvantages of using the patientMpower 
platform (Open text for healthcare professional to give opinion) 
 
5.1.1.2  
Secondary endpoints 
 
The secondary endpoints include 
• the impact of active engagement and self-monitoring using the 
patientMpower platform on PROMs in IPF 
• the impact of using the patientMpower platform on exercise 
performance (e.g. walking distance) 
• the effect of the patientMpower platform on medication compliance 
• the correlation between patient-reported measures (PROMs, 
spirometry) and clinical measures and outcomes. 
 
The secondary endpoint variables (reported by patients) are: 
 
• FVC (recorded once per day) 
• maximum level of dyspnoea each day (ideally linked to description of 
activity causing maximum dyspnoea) 
• activity (number of steps/day) 
• distance walked per day 
• compliance with medicines prescribed for treatment of IPF 
• addition of any new prescribed medicines for treatment of IPF 
• impact of their medical condition on their daily life (once per week; 
questions to be derived from PROM) 
 
Additional secondary endpoint variables which can be recorded (if 
measurement devices available to the patient) include:  
• duration of walking per day 
• number of episodes of walking per day 
• cough (worst severity each day) 
• heart rate (if patient has access to wearable fitness device) 
• blood pressure (if patient has access to measurement device) 
• temperature (if patient has access to measurement device) 
• body weight (once/week) 
• oxygen saturation at rest (if patient has access to pulse oximetry 
device and wishes to record saturation) 
• oxygen consumption (cylinders/month) 
 
Patients can also record symptoms using the health journal entry pages on 
the patientMpower platform at any time.  
 
Patients will assess dyspnoea with the mMRC scale [8] which may be a useful 
prognostic indicator in IPF [9]. 
 

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Clinic-derived measurements (for example): 
• FVC  
• DLCO (if measured) 
• 6-minute walking distance  
• Dyspnoea (e.g. modified Borg scale) [10] 
• Impact of the patient’s medical condition on their daily life (using the 
same PROM questions as for the patient) 
Health outcomes (for example): 
• Medication adherence 
• Change in IPF medication (dose change or new medicine prescribed) 
• Oxygen usage 
• Exacerbations of IPF 
• Hospitalisations due to IPF 
 
5.1.2  Assessment of efficacy 
 
The primary efficacy endpoint data will be assessed by analysis of changes in 
the patient-reported FVC and health parameters, clinic-derived parameters 
and health outcomes over time (described above in 5.1.1.1). 
 
The impact of IPF on daily life (i.e. PROM) at the end of each treatment period 
will be compared with the start of each treatment period. In addition the end of 
period PROM will be compared between treatment periods. 
 
The longitudinal trend of patient-measured FVC will be compared with the 
FVC values observed in the clinic at the beginning and end of each treatment 
period. 
 
The secondary efficacy endpoints will be assessed by analysis of the 
responses to the patient and healthcare professional questionnaires 
(described above in 5.1.1.2).  
 
5.2  
Safety 
 
It is not anticipated that any safety issues will arise from use of the 
patientMpower platform or the Spirobank Smart spirometer. 
 
Any adverse events observed with medical treatments should be reported to 
the manufacturers of those treatments. 
 
5.2.1  Safety endpoints 
 
None. 
 
 
 

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5.3.  Other variables 
 
Demographic data (date of birth, gender, date of IPF diagnosis, other 
respiratory conditions, medication prescribed for IPF). 
 
Type of smartphone or tablet device (i.e. iPad or Android tablet, iPhone or 
Android phone) 
 
Engagement of patients with the patientMpower platform will be assessed by 
analysis of the numbers of 
• patients asked to take part in the study 
• patients who give informed consent to take part in the study. 
• consented patients who download the platform application to their 
smartphone or tablet device 
• patients who use the platform at least once after downloading 
• patients who use the platform more than once 
• frequency of use by each patient 
• date intervals between informed consent, download, first use 
• date intervals between first and subsequent uses 
• frequency of recording FVC at home 
• frequency of recording other measures at home 
5.3.2  Other assessments 
 
Data on the air quality and weather conditions at the patients’ geographical 
locations over the duration of the study will be captured. This may be 
analysed retrospectively to see if there is any correlation between air quality 
and impact of IPF on patient quality of life, respiratory measurements or 
exacerbations. 
 
Data on the ultraviolet index at the patients’ geographical locations over the 
duration of the study will be captured. This may be analysed retrospectively to 
see if there is any correlation between possible ultraviolet exposure and 
impact of IPF on patient quality of life or possible side-effects of IPF 
treatment. 
 
5.4  
Appropriateness of measurements 
 
The primary endpoint parameters are measurements which are affected by 
the patient’s IPF and respiratory health.  
 
The questionnaires used to assess usefulness and patient acceptability have 
been used to assess these parameters for other patient support platforms and 
have been shown to be robust. 
 
 
 

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6 
Investigational plan 
 
Patients who participate in the study will beallocated to the treatment 
sequence. Patients will follow their usual care programme throughout the 
study.  
 
During the patientMpower treatment period, patients will be encouraged to 
use the patientMpower platform and Spirobank Smart spirometer every day to 
record parameters relevant to their health status (described in section 5.1.1.2 
above). 
 
6.1  
Visit schedule 
 
The total observation period will be approximately sixteen (16) weeks. 
 
6.2  
Details of trial procedures at selected visits 
 
The trial procedures at each visit are summarized in the flow charts on page 
6. 
 
6.2.1  Baseline clinic visit 
 
Patients visiting the IPF clinic for diagnosis or routine care will be offered the 
opportunity to participate in the study. 
 
The study will be explained to the patient and written informed consent 
obtained before any study-specific procedures. On receipt of written informed 
consent, a unique identification number will be assigned to the patient.  
 
The patient will be allocated to the treatment sequence:  
period 1: patientMpower platform and usual care followed by period 2: usual 
care alone. 
 
The patient and the investigator site will know the treatment sequence 
assigned to the patient.  
 
Demographic data, medical history and concomitant therapy for IPF will be 
recorded. 
 
The following parameters will be assessed and recorded in the clinic records: 
• spirometry (to include FVC) 
• DLCO (if measured) 
• patient’s exercise ability (e.g. 6-minute walking distance or other 
standard measure) 
• dyspnoea (e.g. modified Borg scale) 
• impact of IPF on daily life (using the same PROM as in the 
patientMpower platform) 

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6.2.2  Treatment and observation period 
 
Period 1: patientMpower platform and usual care 
Period 2: usual care alone 
 
At the baseline visit, the patientMpower platform will be downloaded to the 
patient’s mobile phone and they will be trained in correct use of the platform. 
A tablet device will be supplied to the patient if they do not have access to a 
suitable smart device at home.  
 
The patient will be instructed in the correct use of the Spirobank Smart home 
spirometer and uploading of data to the patientMpower platform. The patient’s 
understanding of the patientMpower platform, Spirobank Smart home 
spirometer and the study procedures should be checked before they leave the 
clinic. 
 
Patients will be encouraged to record the parameters described in section 
5.1.1.1 on a regular basis.  
 
The following should be recorded by the patient each day: 
• FVC (one spirometry maneuver each day ideally at approximately the 
same time each day) 
• compliance with medication prescribed for IPF 
• any changes to medication taken for IPF 
• activity (number of steps per day) 
• distance walked per day 
• maximum level of dyspnoea 
 
Impact of IPF on daily life (PROM) should be recorded by the patients once 
per week. 
 
Other parameters should be recorded by the patient (if suitable measurement 
devices are available to the patient and where practical for the patient) 
frequently. Examples include duration of walking, number of episodes of 
walking, cough, heart rate, blood pressure, temperature, body weight, oxygen 
saturation and oxygen usage (cylinders/month). 
 
Two (2) days after starting period 1, the patient will be contacted by telephone 
to check the following 
• patient understanding of the study 
• they have started to record medication compliance, activity, dyspnoea 
and FVC every day 
• patient is recording symptoms and other parameters on a regular basis 
(ideally daily) 
 
Appropriate advice will be given to the patient if needed. 
 

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Fourteen (14) days after starting period 1, the patient will be contacted by 
telephone to check the following 
• patient understanding of the study 
• patient is continuing to record medication compliance, activity, 
dyspnoea and FVC every day 
• patient is recording impact of IPF on daily life (PROM) every week  
• patient is continuing to record symptoms and other parameters on a 
regular basis (ideally daily) 
 
Appropriate advice will be given to the patient if needed. 
 
The patient will continue using the patientMpower platform until the clinic visit 
at eight (8) weeks after starting period 1. 
 
6.2.3 8-week clinic visit 
 
At this visit, the following parameters will be assessed and recorded in the 
clinic records: 
• spirometry (to include FVC) 
• patient’s exercise ability (e.g. 6-minute walking distance or other 
standard measure) 
• dyspnoea (e.g. modified Borg scale) 
• impact of IPF on daily life (using the same PROM as in the 
patientMpower platform) 
• medication adherence 
• changes in or addition of any new medicines for the treatment of IPF 
• exacerbations of IPF since baseline 
• hospitalisations due to IPF since baseline 
 
The patient and healthcare professional opinion on the usefulness and 
acceptability of the patientMpower platform will be assessed and recorded. 
 
The patient will stop using the patientMpower platform and will crossover to 
the alternative treatment period (usual care only for a further eight weeks). 
There will be no further interventions until the End of Study visit (at least 
sixteen weeks after the baseline visit). 
 
6.2.4  End of Study clinic visit ( at least 16 weeks) 
 
The following parameters will be assessed and recorded in the clinic records: 
• spirometry (to include FVC) 
• DLCO (if measured) 
• patient’s exercise ability (e.g. 6-minute walking distance or other 
standard measure) 
• dyspnoea (e.g. modified Borg scale) 
• impact of IPF on daily life (using the same PROM as in the 
patientMpower platform)  
• medication adherence 

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• changes in or addition of any new medicines for the treatment of IPF 
• exacerbations of IPF since previous visit 
• hospitalisations due to IPF since previous visit 
 
7.  
Statistical methods and determination of sample 
size 
 
This a pilot study to assess the feasibility of patients with IPF using the 
patientMpower platform including home-based FVC to monitor their health. 
 
7.1  
Statistical design and model 
 
Single-centre, prospective, open-label, usual care controlled, open, fixed-
order, two-period crossover observational study 
7.2  
Null and alternative hypotheses 
 
Not relevant. 
 
7.3  
Planned analyses 
 
Results will be collected and summarized for descriptive statistical display. 
 
7.3.1  Primary analyses 
 
The primary efficacy endpoints will be assessed by analysis of the responses 
to the patient and healthcare professional questionnaires (described above in 
5.1.1.1).  
 
7.3.2  Secondary analyses 
 
The secondary efficacy endpoints in the two treatment periods will be 
compared.  
 
7.3.3  Safety analyses 
 
Not relevant. 
 
7.3.4  Interim analyses 
 
None planned. 
 

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7.3.5  Health economic analyses 
 
Health outcomes (e.g. exacerbations of IPF) will be recorded. 
 
7.4  
Handling of missing data 
 
No imputations of missing data will be made. 
 
7.5.  Randomisation 
 
Not relevant. 
 
7.6  
Determination of sample size 
 
This is pilot study. The sample size of 8 subjects has been chosen arbitrarily.  
 
8.  
Informed consent, data protection and trial 
records 
 
8.1  
Study approval, patient information and informed consent 
 
The study will be approved by the relevant ethics committee(s) for the 
participating centre. 
 
The study will be discussed with each patient and they will be provided with a 
written document describing the study conditions and procedures. 
 
All patients will give written informed consent before participation. 
 
8.2  
Data quality assurance 
 
All endpoint data will be stored on a central database for analysis. The data 
as reported by the patients will not be queried before descriptive statistical 
analysis tables are prepared. 
 
8.3  
Records 
 
8.3.1  Source documents 
 
The original electronic data will be the source document.  
 

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8.3.2  Direct access to source data and documents 
 
Source data verification will not be performed. 
 
8.3.3  Storage of records 
 
Medical data relating to patient care will be stored in the medical records 
according to the usual procedures of the investigator site. 
 
The endpoint data collected by the participants and recorded on the 
patientMpower platform will be stored indefinitely in a secure cloud database 
managed by patientMpower Ltd. These data will be available to patients 
indefinitely to aid them in self-management of their medical condition. 
Participants can request deletion of their data from the patientMpower cloud 
database at any time. 
 
The information on the patientMpower Ltd database for this study may be 
analysed again as part of other scientific studies in the future. 
8.4  
Statement of confidentiality 
 
Participants will only be identified by a unique identification number and data 
will be anonymised on the trial database. The identity of the participants will 
be known to patientMpower Ltd. All data will be treated as confidential. Each 
patient’s data will linked to their unique identification number on the trial 
database.  
 
The patientMpower platform is designed with stringent security protocols. The 
solution is hosted in Google Compute Engine.  The security protocols used 
include: 
• platform uses a PostgreSQL database (https://www.postgresql.org/) 
which is backed up nightly 
• platform is patched regularly to ensure it is maintained against security 
vulnerabilities 
• only certain Internet Protocol (IP) addresses can login to the cloud 
infrastructure using Secure Shell (SSH) with IP whitelists and 
public/private key access only 
• built-in firewalls 
• encrypted data storage  
• patientMpower staff access to the PostgreSQL database, and content 
system is restricted and monitored 
• a unique username and password for each user.  
• audit and accounting of all access to the system is recorded. In the 
event of any staff looking at data without proper authorisation, there is 
an audit trail of what data was viewed 
• data transfer between the patient mobile device and cloud server is 
sent securely via Transport Layer Security (TLS) and the platform’s 
cloud infrastructure uses an Extended Validation Secure Sockets Layer 
(SSL) Certificate issued by Digicert (https://www.digicert.com) 

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• data on the server is encrypted, only authenticated users can access 
the server 
 
8.5  
Completion of trial 
 
The trial will be complete when 8 patients have completed the 16-week 
observation period. 
 
If it appears to be unlikely that the target number of patients can be achieved 
(e.g. because of slow recruitment) a lower target will be set (after discussion 
and agreement with investigator sites). 
 
8.6  
Protocol violations 
 
All data will be analysed on an intention-to-treat basis without regard to 
protocol violations. 
 
8.7  
Compensation available to the patient in the event of trial-
related injury 
 
It is not anticipated that any trial-related injury will occur. 
 
9 
References 
 
1. Raghu G, Collard HR, Egan JJ, et al. An official ATS/ERS/JRS/ALAT 
statement — idiopathic pulmonary fibrosis: evidence based guidelines 
for diagnosis and management. Am J Respir Crit Care Med 2011; 183: 
788-824. 
2. Yorke J, Jones PW, Swigris JJ. Development and validity testing of an 
IPF-specific version of the St. Goerge’s Respiratory Questionnaire. 
Thorax 2010; 65: 921-6. 
3. Patel AS, Siegert RJ, Brignall K, Gordon P, Steer S, Desai SR et al. 
The development and validation of the King’s Brief Interstitial Lung 
Disease (K-BILD) health status questionnaire. Thorax 2012. Doi 
10.1136/thoraxjnl-2012-201581. 
4. Patel AS, Siegert RJ, Keir GJ, Bajwah S, Barker RD, Maher TM et al. 
The minimal important difference of the King’s Brief Interstitial Lung 
Disease (K-BILD) and forced vital capacity in interstitial lung disease. 
Respir. Med. 2013; 107: 1438-43. 
5. Russell A-M, Jones G, Sabella R, Cassidy N, Burge G, Lines S et al. 
Health status and impact of living with idiopathic pulmonary fibrosis 
(IPF): UK and Ireland Delphi survey. Eur Resp J 2016 (in press). 
6. Russell A-M, Adamali H, Molyneaux PL, Lukey PT, Marshall RP, 
Renzoni EA, Wells AU, Maher TM. Daily home spirometry: an effective 

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tool for detecting progression in idiopathic pulmonary fibrosis. Am J 
Respir Crit Care Med 2016. 10.1164/rccm.201511-2152OC.   
7. Raghu G, Collard HR, Egan JJ, Martinez FJ, Behr J, Brown KK et al. 
An official ATS/ERS/ JRS/ALAT statement. Idiopathic pulmonary 
fibrosis: evidence-based guidelines for diagnosis and management. 
Am J Respir Crit Care Med 2011;183:788–824. 
8. Stenton C. The MRC breathlessness scale. Occupational Med. 2008; 
58: 226-7. 
9. Nishiyama O, Taniguchi H, Kondoh Y, Kimura T, Kato K, Kataoka K et 
al. A simple assessment of dyspnoea as a prognostic indicator in 
idiopathic pulmonary fibrosis. Eur. Respir. J. 2010; 36: 1067-72. 
10. Borg GA Psychophysical bases of perceived exertion. Med Sci Sports 
Exerc. 1982; 14(5):377-81 
 
 

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10  Appendices 
 
10.1  modified Medical Research Council Dyspnoea Scale 
 
Grade 
Description of breathlessness 
0 
I only get breathless with strenuous exercise 
1 
I only get short of breath when hurrying on level ground or 
walking up a sight hill 
2 
On level ground, I walk slower than people of the same age 
because of breathlessness or have to stop for breath when 
walking at my own pace 
3 
I stop for breath after walking about 100 yards or after a few 
minutes on level ground 
4 
I am too breathless to leave the house or I am breathless when 
dressing 
None. 
 
10.2 Instructions for performing spirometry at home 
 
Patients will perform one spirometry reading at home once per day (ideally at 
12:00 noon + 1:00 hour).  
 
Each patient will be supplied with a Spirobank Smart home spirometer 
(Medical International Research, Via del Maggiolino 125, 00155 Roma, Italy. 
www.spirometry.com). This spirometer will automatically upload Peak 
Expiratory Flow (PEF), Forced Expiratory Volume in 1 second (FEV1), FVC 
and FEV1/FVC data directly to the patientMpower platform. 
 
11  Summary of clinical trial protocol modifications 
 
The protocol was revised to Version 2 on 12th July 2017. The design was 
changed from a randomized, two-period crossover design to a fixed-order 
two-period crossover design. This change was made due to operational 
difficulties in implementing the original design. There were no changes to the 
patient selection criteria or study measurements.