An Observational Study of Self-monitoring of Spirometry and Symptoms Via the patientMpower App in Patients With Idiopathic Pulmonary Fibrosis
Conditions: Idiopathic Pulmonary Fibrosis
Sponsor: patientMpower Ltd.

 
Confidential ©patientMpower 
 
An observational study of self-monitoring of 
spirometry and symptoms via the patientMpower app 
in patients with idiopathic pulmonary fibrosis. 
 
IPF patientMpower 03 
 
Protocol date: 11 January 2018 
 
Number of pages: 37 
 
Version 1.0 
 
Authors: 
 
Colin Edwards PhD, patientMpower, Dublin, Ireland. 
 
Eamonn Costello, patientMpower, Dublin, Ireland. 
 
Dr. Katherine O’Reilly, Mater Misericordiae University Hospital, Eccles Street, 
Dublin 7, Ireland (Principal investigator) 
 
NCT 03744598 
 
 
 

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Table of Contents 
PROTOCOL SUMMARY 
4 
FLOW CHARTS 
6 
Flow chart 1: 
6 
Flow chart 2  
7 
List of Tables 
9 
List of Figures 
10 
Figure 1:  
10 
List of Abbreviations 
11 
1.  
12 
1.1  
12 
1.2  
12 
2. 
14 
2.1  
14 
2.2  
15 
2.3  
15 
3.  
15 
3.1  
15 
3.1.1  
16 
3.2  
16 
3.3  
17 
3.3.1  
17 
3.3.2  
17 
3.3.3  
18 
3.3.4  
18 
4.1  
18 
4.1.1  
19 
4.1.2  
19 
4.1.3  
19 
4.2  
19 
4.2.1  
19 
4.2.2  
20 
4.3  
20 
5  
20 
5.1  
20 
5.1.1  
20 
5.1.2  
23 
5.2  
24 
5.3.  
24 
5.3.2  
24 
5.4  
25 
6 
25 
6.1  
25 
6.2  
25 

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6.2.1  
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6.2.2  
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6.2.3  
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6.2.4  
28 
7.  
28 
7.1  
28 
7.2  
28 
7.3  
29 
7.3.1  
29 
7.3.2  
29 
7.3.3  
29 
7.3.4  
29 
7.3.5  
29 
7.4  
30 
7.5.  
30 
7.6  
30 
8.  
31 
8.1  
31 
8.2  
31 
8.3  
31 
8.3.1  
31 
8.3.2  
31 
8.3.3  
31 
8.4 
32 
Figure 8.4: 1 Screenshot of journal entry page: 
30 
8.5  
34 
8.6  
34 
8.7  
35 
9 
35 
10  
37 
10.1  
37 
10.2 
37 
10.3 
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10.3.1 
37 
10.3.2 
38 
10.3.3 
38 
11  
40 
 
 
 

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PROTOCOL SUMMARY 
 
PRODUCT 
patientMpower app 
WWW.CLINICALTRIALS.GOV 
IDENTIFIER 
NCT 00000000 
PROTOCOL TITLE 
An observational study of self-
monitoring of spirometry and 
symptoms via the patientMpower 
app in patients with idiopathic 
pulmonary fibrosis 
CO-ORDINATING INVESTIGATOR 
Dr. Katherine O’Reilly, Mater 
Misericordiae University Hospital, 
Eccles Street, Dublin 7, Ireland 
(Principal investigator) 
NUMBER OF STUDY SITES 
One 
CLINICAL PHASE 
Not applicable 
STUDY OBJECTIVES 
Characterise longitudinal trends of 
patient-reported Forced Vital 
Capacity (FVC) and outcome 
measures in patients with idiopathic 
pulmonary fibrosis (IPF). 
Determine the correlation between 
patient-reported FVC and outcome 
measures with clinic-observed 
measurements. 
Assess if longitudinal trends in 
patient-reported FVC predict 
clinical outcomes. 
Characterise the acceptability and 
utility of the patientMpower app 
from patient and healthcare 
professional perspectives. 
METHODOLOGY 
Multi-center, prospective, single-
arm, open design 
NUMBER OF SUBJECTS 
Total randomised: up to 25 
Each treatment:  
patientMpower app: 25 
DIAGNOSIS  
Confirmed diagnosis of IPF 
MAIN CRITERIA FOR INCLUSION 
Age ≥18 years, daily access to 
smartphone or tablet device, email 
address, demonstrated 
understanding of use of 
patientMpower app and home 
spirometry, written informed consent 
 
 
 

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TEST PRODUCT 
patientMpower app (via patient’s 
smartphone or tablet device) 
DURATION OF OBSERVATION 
Sixteen (± eight) weeks 
END OF STUDY DEFINITION 
Sixteen (± eight) weeks 
PRIMARY ENDPOINTS 
Trend of patient-measured FVC over 
time. 
Degree of correlation between 
patient-measured FVC with clinic-
observed measures.  
SECONDARY ENDPOINTS 
Trends of patient-reported 
outcomes over time. 
Degree of correlation between 
patient-reported outcomes with 
clinic-observed measures.  
Patient and healthcare professional 
perspectives on preference for and 
difficulty using the app and 
helpfulness in managing IPF. 
Impact of app on patient self-
management of IPF and daily living.  
INTERIM ANALYSIS 
None planned 
STATISTICAL METHODS 
Descriptive statistics tables will be 
prepared 
 
 

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FLOW CHARTS 
 
Flow chart 1: 
Patient recruitment process 
 
Step 
Day 
Action 
1 
0 
(Baseline) 
At a usual care visit to study centre, research team 
discusses study with patient (face-to-face), confirms 
patient’s understanding of the study processes and 
willingness to participate. 
Obtains written informed consentA. 
Study centre conducts usual clinical assessments. 
Study starts. 
 
2 
0 
(Baseline) 
Study centre advises patientMpower Ltd. of email 
address of patients who have given consent to 
participate. 
 
3 
0 -2 
patientMpower sends information pack (electronic; 
includes instructions on installation of app) and 
home spirometer to patientB. 
 
4 
0 - 2 
Patient starts to use patientMpower app and home 
spirometry as soon as possible after installation of 
app and pairing of spirometer. 
 
 
A Includes explicit consent by patient for patientMpower to know their identity 
and email address and collect, store and analyse data reported by the 
patient on the patientMpower app. 
B On request by the patient, patientMpower Ltd can contact patient to offer 
help with installation of patientMpower application and pairing of spirometer 
(so that application and spirometer are installed and operating correctly). 
 
 
 

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Flow chart 2  
Assessments during observation period 
 
 
Baseline 
(clinic visit) 
Install app & 
pair 
spirometer 
*Daily (patient 
-reported) 
16 (±8) 
weeks End 
study 
 (clinic visit)1 
 
day 0 
day 0-2  
daily 
week 16 
(approx.) 
Informed consent 
X 
 
 
 
Start of study 
X 
 
 
 
Spirometry, assess 
dyspnoea 
X 
 
 
X 
Demographic data (include 
IPF history & medicines 
history3) 
X 
 
 
 
Patient training & 
encouragement 
X 
 
 
 
Installation of 
patientMpower app & pair 
spirometer 
 
X 
 
 
Record compliance or 
changes (IPF medicine)2 
X 
 
X 
X3 
Patient-measured FVC2 
X 
 
X 
X 
Record impact of IPF 
(PROM)4,5 
X4 
 
X5 
X4 
Patient outcomes (e.g. 
oxygen consumption, 
dyspnoea, pulse oximetry, 
symptoms, vital signs)6 
 
 
X 
 
Utility & acceptability of 
app7 
 
 
 
X 
Clinic-reported outcomes 
(e.g. exacerbations) 
 
 
 
X 
End of study 
 
 
 
X 
 
1 End of study visit will be date of usual scheduled clinic visit. 
 
2 Reported by patient on patientMpower app every day. Goal is for patient to 
record one FVC measurement (seated) per day. 
 
3 Clinic to record changes in IPF medication or other medicines prescribed 
for respiratory conditions. 
 

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4 Impact of IPF on daily life (Patient Reported Outcome Measure; PROM) to 
be assessed by clinic team at baseline and end of study visit and recorded 
on patientMpower app. 
 
5 Impact of IPF on daily life (PROM) to be reported by patient on 
patientMpower app every week. 
 
6 Reported by patient as often as possible, ideally each day. These 
measurements are optional and will only be recorded where practical for the 
patient. 
 
7 Patient and healthcare professional perspective. If patient is withdrawn 
prematurely, try to capture patient perspective of utility and acceptability of 
app at time of withdrawal. 
 
 

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List of Tables 
 
None 
 
 

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List of Figures 
 
Figure 1:  
Patient recruitment process 
 
 
 
 
 
 

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List of Abbreviations 
 
ADR  
Adverse reaction 
AE 
 
Adverse event 
ATS 
 
American Thoracic Society 
BI 
 
Boehringer Ingelheim 
DLCO  
Diffusing Capacity of the Lung for Carbon Monoxide 
ERS  
European Respiratory Society 
FEV1  
Forced Expiratory Volume in 1 second 
FVC 
 
Forced Vital Capacity 
IEC 
 
Independent ethics committee 
ILFA  
Irish Lung Fibrosis Association 
IP 
 
Internet Protocol 
IPF 
 
Idiopathic pulmonary fibrosis 
IRB 
 
Institutional review board 
JRS 
 
Japanese Respiratory Society 
K-BILD 
King’s Brief Interstitial Lung Disease Questionnaire 
MIR 
 
Medical International Research 
mMRC 
modified Medical Research Council 
PEF 
 
Peak Expiratory Flow 
PFW  
PF Warriors of Texas 
PROM patient reported outcome measure 
SSH  
Secure Shell 
SSL 
 
Secure Sockets Layer 
TLS 
 
Transport Layer Security 
UCSD  
University of California San Diego 
 
 

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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 
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 [5]. This PROM can be used on a 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 product evaluated in this study is an electronic health diary developed by 
patientMpower Ltd., The Digital Depot, Thomas Street, Dublin 8, Ireland. The 
patientMpower electronic health diary has been developed for and is already 
used in providing support to patients post-renal transplant or with IPF. The 
patientMpower electronic health diary is downloaded as an application or 
“app” to a mobile phone or tablet device and patients record various 
parameters (e.g. blood pressure, symptoms, medication compliance, impact 
of their medical condition on daily life) on a daily basis. The patient has a 
permanent health diary of their self-reported measurements available to them 
on their mobile device. The app 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 

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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 app.  
 
The patientMpower app for renal transplant recipients is now offered as 
standard care at the national renal transplantation centre in Ireland. In the 
renal transplant setting, patients use the patientMpower app every day to 
monitor health parameters (e.g. medicines adherence, blood pressure, body 
temperature) which are important in maintaining the health of the 
transplanted kidney. The data collected on the patientMpower app is 
identifiable (i.e. the identity of the patients is known to patientMpower Ltd) so 
that it can be shared with the renal transplant clinical centre to aid in the 
post-transplant care of the patients. The patientMpower app has been in use 
at the national renal transplantation centre in Ireland since summer 2015 and 
has also been used at renal transplant centres in other countries. To date, 
over 200 renal transplant patients have used the patientMpower app. 
 
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 observational study will evaluate a version of the patientMpower app 
which 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 [6,7]. The patientMpower 
app can link to home spirometry devices to allow longitudinal collection and 
upload of long-term patient-measured FVC data to the app. The Spirobank 
Smart spirometer [Medical International Research (MIR), 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 app. 
 
The patientMpower app (including daily patient-recorded spirometry using 
the MIR Spirobank Smart spirometer) has been assessed in user experience 
surveys (42-day observation period) in over 50 patients with lung fibrosis. 
These surveys were conducted in collaboration with two patient advocacy 

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groups [Irish Lung Fibrosis Association (ILFA), Dublin, Ireland and PF 
Warriors (PFW), Plano, Texas, USA]. The identity of each participant in these 
surveys is known to patientMpower Ltd. Knowledge of the participant’s 
identity is needed in order to maintain access for the participant to the app, 
provision of individualised technical support and to enable later deletion of 
the participant’s data if they were to request deletion at a later stage. 
Feedback from the surveys’ participants demonstrated that daily home 
recording of spirometry and symptoms is feasible and acceptable to people 
with lung fibrosis. In the ILFA study, the majority of participants (9/11; 82%) 
wished to continue using the patientMpower app after the end of the study 
[8] and at least 8 participants (73%) continued to use the app for at least 200 
days [9]. The PFW study is currently being analysed. 
 
The patientMpower app (including daily patient-recorded spirometry using 
the MIR device) is also being evaluated in an open-label, usual care 
controlled clinical trial in patients with IPF at Galway University Hospital, 
Ireland [10, www.clinicaltrials.gov NCT03104322]. This study has received 
ethics committee approval, patient recruitment is complete and follow-up is 
ongoing at the time of writing. The identity of each patient in this study is 
known to patientMpower Ltd. 
 
Another version of the patientMpower application will shortly be evaluated in 
a pilot-scale study in haemodialysis patients [11, www.clinicaltrials.gov 
NCT03403491]. This study has been approved by the independent research 
ethics committee of the study centre in Ireland.  
 
The study described in this protocol will capture longitudinal data on home 
measurement (by the patient) of FVC and PROMs within the same cohort of 
patients. This will enable assessment of the degree of correlation (if any) 
between patient-reported spirometry and the impact of IPF on daily life (as 
assessed by the PROM). 
 
2. 
Rationale, objectives and benefit-risk 
assessment 
2.1  
Rationale for performing the study 
 
There are few data on longitudinal trends in patient-measured FVC and 
PROMs in IPF. It is possible that these data may be predictive of important 
health outcomes (e.g. exacerbations). The patientMpower app provides a 
tool to collect and share this type of information between patients and their 
healthcare professionals. 

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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 app in a controlled 
observational setting. 
 
2.2  
Study objectives 
 
The objectives of this observational study are: 
● to assess and characterise the longitudinal trends of patient-measured 
FVC and PROMs in a cohort of patients with IPF  
● to determine the correlation (if any) between patient-measured FVC 
and PROMs with clinic-observed measurements 
● to assess if longitudinal trends in patient-measured FVC outcomes are 
predictive of clinical health outcomes in IPF 
 
An additional purpose of this observational study is to assess the 
acceptability and utility of the patientMpower app in helping IPF patients and 
their healthcare professional caregivers manage their condition. These will be 
assessed from both the patient and healthcare professional perspective.  
 
2.3  
Benefit-risk assessment 
 
The patientMpower app has been used in other clinical settings (post renal 
transplant). The renal transplant version of the patientMpower app 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. 
 
Initial feedback from participants in the user experience surveys and clinical 
trial in lung fibrosis indicates that patients are willing to use the app and 
record spirometry at home on a very regular basis (usually daily) and that 
they find it helpful in managing their health. These studies are ongoing at the 
time of writing. 
 
It is not expected that the study or patientMpower app will create any 
additional risks for IPF patients. 
3.  
Description of design and study population 
3.1  
Overall design and plan 
 

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This is an open-label, single group observational study of a population of IPF 
patients treated with usual care. Patients who enter the study will be trained 
in correct use of the patientMpower app 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 study 
 
This is a single centre study in Ireland. The study centre is an expert centre 
for the diagnosis, staging and treatment of IPF. 
 
The patientMpower app 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 Dr. Katherine O’Reilly, Mater Misericordiae 
University Hospital, Dublin (the principal investigator) and patientMpower Ltd. 
 
Financial support for the study will be provided as an unrestricted research 
grant from Boehringer Ingelheim Ltd. The financial sponsor will not play any 
role in the design, conduct or analysis of the study. 
 
3.2  
Discussion of study design 
 
This is an open-label, single group observational study. There is no control 
group.  
 
Patients will follow their usual care programme throughout the study. The 
study observation period (16 ± 8 weeks) is approximately equal to the interval 
between clinic visits in the usual care of IPF.  
 
This design will enable assessment of longitudinal trends in FVC and PROMs 
in a cohort of patients with IPF undergoing usual care. 
 
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 app. Patients will also measure FVC at home once per 
day using the Spirobank Smart spirometer. The patient measured FVC and 
other spirometry data will be captured automatically by the patientMpower 
app. These data will form the patient-reported outcome database. 
 
Clinical assessments (e.g. FVC, dyspnoea) in the same cohort of patients will 
be assessed at the beginning and end of the study. PROMs will also be 
recorded at the clinic 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 app in a patient population managed by centres with 
expertise in the diagnosis and management of IPF. This will be assessed 
from both the patient and healthcare professional perspectives. 
 
The patientMpower app 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 study population 
 
3.3.1  Main diagnosis for study entry 
 
Idiopathic pulmonary fibrosis 
3.3.2  Inclusion criteria 
 
Aged at least 18 years 
 
Confirmed diagnosis of IPF [according to American Thoracic Society (ATS), 
European Respiratory Society (ERS) or Japanese Respiratory Society (JRS) 
criteria] [1]. 
 
Has daily unrestricted access to a suitable smartphone or tablet device at 
home*.  
 
Has an e-mail address. 
 
Has home broadband and/or mobile data as part of their mobile phone 
service 
 
Demonstrates understanding of correct use of the Spirobank Smart 
spirometer and the patientMpower app. 
 
Able and willing to perform spirometry at home and record information on the 
patientMpower app on a daily basis. 
 
Willing to give written informed consent. 
 

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(*patientMpower Ltd will provide a suitable tablet device if the patient does 
not have access to their own device.)  
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. 
 
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  Discontinuation of patients from study or assessments 
 
3.3.4.1  
Discontinuation of individual patients 
 
Patients are free to withdraw from the study at any time without any impact 
on their ongoing medical care. Patients can continue to use the 
patientMpower app and spirometer after withdrawal from the study of they 
wish. 
 
Investigators 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 as a whole. 
 
The study may be terminated 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 observational intervention 
 

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4.1.1  Identity of study observational intervention 
 
The study observational intervention is an electronic health diary, the 
patientMpower app. This has been developed specifically for patients with 
IPF. The patientMpower app is an electronic application downloaded to the 
patient’s mobile phone or tablet device. The app 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 app 
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 once every week.  
 
Additional patient-reported measures which can be reported on the 
patientMpower app include vital signs (e.g. heart rate, blood pressure), 
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 observational intervention 
 
All patients who give informed consent and enter the study will be given 
access to the patientMpower app and Spirobank Smart home spirometer 
and will be encouraged to use them on a daily basis. 
 
There is only one treatment group in this study. The same study-related 
observations will be assessed and recorded for all patients who are eligible 
and who give written informed consent. 
 
4.1.3  Blinding and procedures for unblinding 
 
The study is open-label.  
 
4.2  
Management of exacerbations or adverse events and 
guidance on concomitant treatments 
4.2.1  Management of acute exacerbations or adverse events 
 

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Any exacerbations of the patient’s IPF or any other underlying medical 
condition(s) should be treated according to standard procedures. 
 
Any other adverse events should be treated according to standard 
procedures.  
 
Any adverse events observed with medical treatments should be reported to 
the manufacturers of those treatments. (See Section 8.4 for further 
information on the process.)  
 
4.2.2  Guidance on concomitant treatments 
 
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. 
 
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 app to record daily compliance with 
medications prescribed for treatment of their IPF (if they are receiving 
medications for treatment of IPF). 
5  
Variables and their assessment 
 
5.1  
Efficacy 
  
The objectives of this observational study are: 
● to assess and characterise the longitudinal trends of patient-measured 
FVC and PROMs in a cohort of patients with IPF  
● to determine the correlation (if any) between patient-measured FVC 
and PROMs with clinic-observed measurements 
● to assess if longitudinal trends in patient-measured FVC outcomes are 
predictive of clinical health outcomes in IPF 
● to assess the acceptability and utility of the patientMpower app  
 
5.1.1  Efficacy endpoints 
 

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5.1.1.1  
Primary endpoint 
 
The primary endpoint is the longitudinal trend in patient-reported FVC. The 
correlation between patient-reported FVC and clinic-reported measures and 
outcomes will also be assessed.  
 
The primary endpoint variable is the daily patient-reported FVC. 
 
5.1.1.2  
Secondary endpoints 
 
The secondary endpoints include 
● the longitudinal trend in PROMs 
● the correlation between patient-reported measures (PROMs) and 
clinic-reported measures and outcomes. 
● assessment if patient-measured FVC predicts clinical health outcomes 
● the impact of active engagement and self-monitoring using the 
patientMpower app on the impact of IPF on daily life (PROM) 
● the effect of the patientMpower app on medication compliance 
● the acceptability and utility of the patientMpower app (from patient 
and healthcare professional perspective) 
 
The secondary endpoint variables (reported by patients) are: 
● 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/week; 
responses to questions in PROM) 
 
Additional secondary endpoint variables which can be recorded by the 
patients (if measurement devices are available to the patient and it is 
practical for the patients to record these variables) 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) 
 

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Patients will assess dyspnoea with the mMRC dyspnoea scale [12] which 
may be a useful prognostic indicator in IPF [13]. 
 
The secondary endpoint variables (assessed and recorded by the clinic) are: 
● FVC 
● Dyspnoea [e.g. University of California San Diego (UCSD) Shortness of 
Breath Questionnaire or other measure usually used by the research 
sites] [14] 
● impact of the patient’s medical condition on their daily life (using the 
same PROM questions used by the patient) 
● health outcomes e.g. 
o medication adherence 
o change in IPF medication (dose change or new medicine 
prescribed) 
o oxygen usage 
o exacerbations of IPF 
o hospitalisations due to IPF 
 
The patient’s opinion of the utility and acceptability of the patientMpower 
app as assessed by their response to questions:  
 
● the instructions given in using the patientMpower app were clearly 
understandable (strongly agree/agree/disagree/strongly disagree) 
● using the patientMpower app helped me to take the correct dose of 
my medicines for lung fibrosis every day (strongly 
agree/agree/disagree/strongly disagree) [only asked if patient is taking 
medication specifically prescribed for IPF] 
● using the patientMpower app helped me to reach my personal 
exercise goal every day (strongly agree/agree/disagree/strongly 
disagree) 
● using the patientMpower app helped me to walk further (or exercise 
more) compared with before (strongly agree/agree/disagree/strongly 
disagree) 
● using the patientMpower app 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) 
● I liked using the patientMpower app (strongly 
agree/agree/disagree/strongly disagree) 
● the patientMpower app was easy to use (strongly 
agree/agree/disagree/strongly disagree) 
● the effect of using the patientMpower app on the impact of lung 
fibrosis on my well-being and daily life (positive, negative) 
● I found it tiring or irritating to use the patientMpower app (strongly 
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● I want to continue using the patientMpower app after the end of the 
study (yes, no) 
● I would recommend other people with my condition to use the 
patientMpower app (yes/no) 
● what other measurements, reminders or information would be useful 
to have on the patientMpower app? (Open text for participant to give 
opinion) 
● describe the benefits and/or disadvantages of using the 
patientMpower app (Open text for participant to give opinion) 
 
The secondary endpoint will also be assessed by the healthcare 
professional’s response to the following questions: 
 
● preference for using the patientMpower app versus not using it (yes, 
no preference, no) 
● difficulty rating in using the patientMpower app (very easy, easy, 
difficult, very difficult) 
● did using the patientMpower app help me to help the patient manage 
their IPF better? (yes, no) 
● did using the patientMpower app help the patient to take their IPF 
medicines at the correct dose every day (yes, no) [only asked if patient 
is taking medication specifically prescribed for IPF] 
● do I believe the patient should continue using the patientMpower app 
after the end of the study (yes, no) 
● what other measurements, reminders or information would be useful 
to have on the patientMpower app? (Open text for healthcare 
professional to give opinion) 
● describe the benefits and/or disadvantages of using the 
patientMpower app (Open text for healthcare professional to give 
opinion) 
 
5.1.2  Assessment of efficacy 
 
The primary efficacy endpoint data will be assessed by analysis of changes 
in the patient-reported FVC over time and comparison with the clinic-
measured FVC at beginning and end of the study, clinic-derived parameters 
and health outcomes over time. 
 
The secondary efficacy endpoint data will be assessed by analysis of 
changes in the patient-reported health parameters (including PROMs) over 
time and comparison with clinic-derived parameters and health outcomes 
over time (described above in 5.1.1.2). 
 

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The acceptability and utility of the patientMpower app 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 app or the Spirobank Smart spirometer. 
 
Any adverse events (AEs) observed in patients who have received medical 
treatments should be reported to the manufacturers of those treatments. The 
process for reporting AEs is described in section 8.4 and further information 
on the definitions of and reporting requirements of AEs is given in Appendix 
10.3. 
 
There are no safety endpoints in this study. 
 
5.3.  Other variables 
 
Demographic data (date of birth, gender, date of IPF diagnosis, other 
respiratory conditions, medication prescribed for IPF or respiratory 
conditions). 
 
Type of smartphone or tablet device (i.e. iPad or Android tablet, iPhone or 
Android phone) 
 
Engagement of patients with the patientMpower app 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 app application to their 
smartphone or tablet device 
● patients who use the app at least once after downloading 
● patients who use the app 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 PROM at home 
● frequency of recording other measures at home 
 
5.3.2  Other assessments 
 

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Data on the air quality at the patients’ geographical location can be made 
available to them via the patientMpower app during the study. It is possible 
that these data 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 may be captured. These data 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 parameter FVC is an important measure of disease 
severity in IPF and a decline in FVC is consistent with disease progression [1, 
15]. 
 
The secondary 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. 
6 
Investigational plan 
 
Patients who participate in the study will follow their usual care programme 
and will be encouraged to use the patientMpower app and MIR 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 sixteen (16) ± eight (8) weeks. This time 
interval coincides with the interval between usual clinic visits. 
 
6.2  
Details of study procedures at all visits 
 
6.2.1  Patient recruitment process 
 
The patient recruitment process is summarized in the flow-chart 1 on page 6 
and in Figure 1 on page 10. 

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At a usual care visit to the study centre, the research team will discuss the 
study with the patient (face-to-face), confirm their understanding of the study 
processes and their willingness to participate. During the informed consent 
discussion, it will be made clear to the patient that their identity and email 
address will be made known to patientMpower Ltd and that data collected 
on the app will be stored and analysed by patientMpower Ltd. 
 
Written informed consent will be obtained before any study-specific 
procedures and before installation of the patientMpower app. 
 
The study starts at this visit (baseline visit). 
 
After the patient has given written informed consent to participate, the study 
centre will advise patientMpower Ltd. of the email address of patient and that 
they have consented to participate.  
 
patientMpower Ltd. will send an information pack via e-mail to the patient. 
This will include instructions on installation of the patientMpower app and 
Bluetooth pairing of the spirometer. patientMpower will also send a MIR 
Spirobank Smart spirometer to the patient. These will be sent to the patient 
within two working days of the baseline visit. 
 
If requested by the patient, patientMpower Ltd will contact them to offer help 
with installation of patientMpower app and pairing of spirometer. 
 
6.2.2  Baseline clinic visit 
 
The study procedures and assessments during the observation period at 
each clinic visit are summarized in flow-chart 2 on page 7.  
 
At a usual care visit to the study centre, the research team will discuss the 
study with the patient (face-to-face), confirm their understanding of the study 
processes and their willingness to participate. 
 
Written informed consent will be obtained before any study-specific 
assessments and before installation of the patientMpower app. 
 
The study starts at this visit. 
 
The patient will be instructed in the correct use of the Spirobank Smart home 
spirometer and uploading of data to the patientMpower app. The patient’s 
understanding of the patientMpower app, Spirobank Smart home spirometer 
and the study procedures should be checked before they leave the clinic. 
 

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Demographic data, medical history and concomitant therapy for IPF and 
other respiratory conditions will be recorded. 
 
The following parameters will be assessed and recorded in the clinic records: 
● spirometry (to include FVC) 
● dyspnoea (e.g. UCSD Shortness of Breath Questionnaire or other 
measure usually used by the investigator sites) 
impact of IPF on daily life (using the same PROM as in the 
patientMpower app) 
 
The study centre will advise patientMpower Ltd that the patient has 
consented to participate in the study and provide the patient’s contact 
details. 
 
patientMpower Ltd. will send the patient an information pack (via email) and 
a home spirometer (via regular mail or courier). The information pack will give 
instructions (including video clips) on how to install the patientMpower app 
onto your smartphone or tablet device and how to pair the spirometer with 
the app. During the installation, some information on the patient (e.g. date of 
birth and gender) will be recorded on the patientMpower app. If necessary, 
the patient may be loaned a tablet device (like an iPad) which will have the 
patientMpower app installed. If requested by the patient, patientMpower Ltd. 
will provide technical support to the patient in downloading the app and 
pairing with the spirometer.  
 
6.2.3  Treatment and observation period 
 
Patients will be encouraged to record the parameters described in section 
5.1.1.1 and 5.1.1.2 on a regular basis.  
 
The following should be recorded by the patient each day: 
● FVC (one spirometry manoeuvre each day ideally at approximately the 
same time each day but this is not essential) 
● compliance with medication prescribed for IPF (if prescribed) 
● 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 every 
week. 
 
Patients will receive reminders (via the patientMpower app) at appropriate 
intervals to record study parameters. 
 

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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 (e.g. cylinders/month). 
 
If requested by the patient, patientMpower Ltd will provide technical support 
on correct use of the app or spirometer at any time during the study. 
 
6.2.4  End of treatment clinic visit (16 ± 8 weeks) and follow-up period 
 
The following parameters will be assessed and recorded in the clinic records: 
● spirometry (to include FVC) 
● dyspnoea (e.g. UCSD Shortness of Breath Questionnaire or other 
measure usually used by the investigator sites) 
● impact of IPF on daily life (using the same PROM as in the 
patientMpower app)  
● medication adherence (if prescribed for IPF) 
● changes in or addition of any new medicines for the treatment of IPF 
(if prescribed) 
● exacerbations of IPF since previous visit 
● hospitalisations due to IPF since previous visit 
 
The patient and healthcare professional opinion on the usefulness and 
acceptability of the patientMpower app will be assessed and recorded. (The 
questions are described in section 5.1.1.2 above.) 
7.  
Statistical methods and determination of sample 
size 
 
This a pilot study to assess the feasibility of patients with IPF using the 
patientMpower app including home-based FVC to monitor their health. 
 
7.1  
Statistical design and model 
 
Open-label, single-arm observational study. 
 
7.2  
Null and alternative hypotheses 
 
Not relevant. 
 

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7.3  
Planned analyses 
 
Results will be collected and summarized for descriptive statistical display. 
 
7.3.1  Primary analyses 
 
The primary efficacy endpoint will be assessed by analysis of changes in 
patient-reported FVC, clinic-derived parameters and health outcomes over 
time. 
 
The patient-reported FVC will be compared with the clinic-derived measures 
and health outcomes to determine if there are any correlations between 
them.  
 
7.3.2  Secondary analyses 
 
The secondary efficacy endpoints data will be assessed by analysis of 
changes in the patient-reported health parameters, clinic-derived parameters 
and health outcomes over time. 
 
The patient-reported health parameters and will be compared with the clinic-
derived measures and health outcomes to determine if there are any 
correlations between them.  
 
The acceptability and utility of the patientMpower app will be assessed by 
analysis of the responses to the patient and healthcare professional 
questionnaires (described above in 5.1.1.2).  
 
7.3.3  Safety analyses 
 
None planned. 
 
7.3.4  Interim analyses 
 
None planned. 
 
7.3.5  Health economic analyses 
 
Health outcomes (e.g. exacerbations of IPF) will be recorded. 
 

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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 proposed sample size of 25 subjects has been 
chosen arbitrarily.  
 
 
 

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8.  
Informed consent, data protection and study 
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 
written information (both electronic and paper) describing the study 
conditions and procedures. 
 
All patients will give electronic 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 recorded in the patientMpower application will be 
the source document.  
 
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 sites.  
 
The endpoint data collected by the participants and recorded on the 
patientMpower app will be stored 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. Patients can request 
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8.4 
Reporting of adverse events 
 
An AE is defined as any untoward medical occurrence in a patient or clinical 
investigation subject administered a medicinal product and which does not 
necessarily have a causal relationship with this treatment. An AE can 
therefore be any unfavourable and unintended sign (e.g. an abnormal 
laboratory finding), symptom, or disease temporally associated with the use 
of a medicinal product, whether or not considered related to the medicinal 
product.  
 
All observed AEs must be reported to the manufacturers of the medicinal 
product(s) administered (irrespective of causal relationship). 
 
The patientMpower app contains a journal entry page where a patient can 
enter information from a list of pre-defined symptoms (e.g. cough) or can 
enter free text (e.g. “nausea”) in example screenshot shown below in Figure 
8.4: 1. Therefore, it is possible that the patient can record AEs which may or 
may not be related to drug treatment. 
 
Figure 8.4: 1 Screenshot of journal entry page: 
 

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As there could be a delay of up to sixteen weeks between the patient 
recording an AE on the journal entry page and the investigator becoming 
aware of the event, the following process is proposed to facilitate timely 
reporting of AEs to medicine manufacturers.  
 
● On the first working day of each month, patientMpower Ltd will 
prepare a listing of all events reported on the journal entry page for 
each patient. This listing will contain the following information: date of 
preparation of listing, patient identification number and all reported 
events (with date and time) per patient.  
 
● patientMpower Ltd will send the listing to the principal investigator at 
the study centre.. 
 
● The investigator will review the listing of events and compare with the 
medicinal treatments given to each patient and determine if any of the 
events recorded by patients are AEs (as defined above). 
 
● Any AEs temporally associated with the use of a medicinal product 
should be reported to the manufacturers of the relevant medicinal 
treatments. 

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If an AE is associated with a Boehringer Ingelheim (BI) medicinal product, the 
event should be reported to the BI contact address described in section 
10.3.3 of Appendix 10.3. 
 
8.5  
Statement of confidentiality 
 
Patients will only be identified by a unique identification number on the study 
database and data will be anonymised. All data will be treated as 
confidential.  
Each patient’s data is linked to their unique identification number.  
 
The patientMpower app is designed with stringent security protocols. The 
solution is hosted in Google Compute Engine.  The security protocols used 
include: 
● app uses a PostgreSQL database (https://www.postgresql.org/) which 
is backed up nightly 
● app 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 app’s cloud 
infrastructure uses an Extended Validation Secure Sockets Layer 
(SSL) Certificate issued by Digicert (https://www.digicert.com) 
● data on the server is encrypted, only authenticated users can access 
the server 
8.6  
Completion of study 
 
The study will be complete when 25 patients have completed the 16-week 
observation period. 
 

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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 the investigator). 
 
8.7  
Protocol violations 
 
All data will be analysed on an intention-to-treat basis without regard to 
protocol violations. 
 
 
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. George’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 48 OA4573 doi 
10.1183/13993003congress-2016.OA4573 
6. Russell A-M, Adamali H, Molyneaux PL, Lukey PT, Marshall RP, 
Renzoni EA, Wells AU, Maher TM. Daily home spirometry: an 
effective tool for detecting progression in idiopathic pulmonary 
fibrosis. Am J Respir Crit Care Med 2016. 10.1164/rccm.201511-
2152OC.  
7. Johansson KA, Vittinghoff E, Morisset J, Lee JS, Balmes JR, Collard 
HR. Home monitoring improves endpoint efficiency in idiopathic 
pulmonary fibrosis. Eur. Respir. J. 2017 50 1602406 
https://doi.org/101183/13993003.02406-2016.  
8. Edwards C, Cassidy N, Costello E, Russell A-M. Pilot study to test 
the utility and acceptability of an electronic health record 

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(patientMpower) for patients with lung fibrosis. iproc 2017;3(1): e10) 
doi:10.2196/iproc.8389 
9. Edwards C, Cassidy N, Costello E, Russell A-M. A longitudinal 
approach to supported self-management: sustainability of the 
idiopathic interstitial pneumonia (IIP) electronic health journal 
(patientMpower) with integrated home spirometry. Ir. J. Med. Sci. 
2017 186 (Suppl 10) S440.  
10. O’Regan A, Edwards C, Costello E. A pilot-scale observational study 
of self-monitoring of symptoms and spirometry via the 
patientMpower platform in patients with idiopathic pulmonary 
fibrosis. www.clinicaltrials.gov NCT 03104322. 
11. O’Seaghdha C, Edwards C, Costello E, Sexton D. A pilot-scale, 
randomized study comparing self-monitoring of weight and blood 
pressure via an electronic health journal (patientMpower platform) 
with usual care in haemodialysis patients. www.clinicaltrials.gov 
NCT 03403491. 
12. Stenton C. The MRC breathlessness scale. Occupational Med. 
2008; 58: 226-7. 
13. 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. 
14. Eakin EG, Resnikoff PM, Prewitt LM, Ries AL, Kaplan RM. Validation 
of a new dyspnea measure: the UCSD Shortness of Breath 
Questionnaire. University of California, San Diego. Chest 1998; 113: 
619-24. 
15. Richeldi L, du Bois R, Raghu G, Azuma A, Brown KK, Costabel U et 
al. Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. 
New Engl. J. Med. 2014; 370: 2071-82. 
 
 
 

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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 
 
10.2 Instructions for performing spirometry at home 
 
Patients will perform one spirometry reading at home once per day (ideally at 
the same time each day).  
 
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 app. 
 
10.3 Management and reporting of adverse events/adverse 
reactions 
 
10.3.1 Definitions of adverse events 
 
An adverse event (AE) is defined as any untoward medical occurrence in a 
patient or clinical investigation subject administered a medicinal product and 
which does not necessarily have a causal relationship with this treatment. An 
AE can therefore be any unfavourable and unintended sign (e.g. an abnormal 
laboratory finding), symptom, or disease temporally associated with the use 
of a medicinal product, whether or not considered related to the medicinal 
product.  
 
An adverse reaction (ADR) is defined as a response to a medicinal product 
which is noxious and unintended. Response in this context means that a 

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causal relationship between a medicinal product and an adverse event is at 
least a reasonable possibility. ADRs may arise from use of the product within 
or outside the terms of the marketing authorisation or from occupational 
exposure. Conditions of use outside the marketing authorization include off-
label use, overdose, misuse, abuse and medication errors. 
 
A serious adverse event is defined as any AE which 
● results in death,  
● is life-threatening,  
● requires in-patient hospitalization, or 
● prolongation of existing hospitalisation,  
● results in persistent or significant disability or incapacity  
● is a congenital anomaly/birth defect  
 
Life-threatening in this context refers to a reaction in which the patient was at 
risk of death at the time of the reaction; it does not refer to a reaction that 
hypothetically might have caused death if more severe. 
 
Medical and scientific judgement should be exercised in deciding whether 
other situations should be considered serious reactions, such as important 
medical events that might not be immediately life threatening or result in 
death or hospitalisation but might jeopardise the patient or might require 
intervention to prevent one of the other outcomes listed above. Examples of 
such events are intensive treatment in an emergency room or at home for 
allergic bronchospasm, blood dyscrasias or convulsions that do not result in 
hospitalisation or development of dependency or abuse. Any suspected 
transmission via a medicinal product of an infectious agent is also 
considered a serious adverse reaction. 
 
10.3.2 Adverse event and serious adverse event collection and reporting 
to regulatory authorities and ethics committees. 
 
The investigator shall maintain and keep detailed records of all AEs in their 
patient records.  
 
The investigator will be responsible for reporting AEs which occur during the 
conduct of the study to the competent regulatory authorities, accredited 
Institutional Review Boards and/or Independent Ethics Committee(s) 
[IRB/IEC(s)] in accordance with the applicable laws and regulations.  
 
10.3.3 Adverse event reporting to Boehringer Ingelheim (for adverse 
events associated with BI products) 
 

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All adverse events associated with use of any Boehringer Ingelheim 
medicinal products must be reported to Boehringer Ingelheim.  
 
The investigator will report 
1. all ADRs (serious and non-serious)  
2. all AEs with fatal outcome  
3. pregnancies in female subjects and partners of male subjects 
 
which are associated with nintedanib by fax to the BI Unique Entry Point as 
specified in Safety Data Exchange Agreement using the BI NIS AE report 
form with the following timelines. 
 
Report to: 
Dr Mike Warwick-Sanders 
Boehringer Ingelheim UK and Ireland Pharmacovigilance Department  
Phone: 01 2913960 
Fax: +44 1344 742661 
E-mail: PV_local_UK_Ireland@boehringer-ingelheim.com 
 
 
 
All Serious ADRs and AEs with fatal outcome shall be forwarded immediately 
[within twenty-four (24) hours or next business day whichever is shorter]. All 
non-serious ADRs and Pregnancy Monitoring Forms shall be forwarded 
within seven (7) calendar days 
 
The investigator will carefully assess whether an AE constitutes an ADR using 
the information below.  
 
Causal relationship of adverse event: 
The definition of an ADR implies at least a reasonable possibility of a causal 
relationship between a suspected medicinal product and an adverse event. 
An ADR, in contrast to an AE, is characterized by the fact that a causal 
relationship between a medicinal product and an occurrence is suspected.   
 
Medical judgment should be used to determine the relationship, considering 
all relevant factors, including pattern of reaction, temporal relationship, de-
challenge or re-challenge, confounding factors such as concomitant 
medication, concomitant diseases and relevant history.  
 
Causality should be assessed for each event as either “yes” or “no”. No other 
variation should be reported.  
 
Intensity of adverse event 
The intensity of the AE should be judged based on the following: 
● mild: awareness of sign(s) or symptom(s) which is/are easily tolerated 
● moderate: enough discomfort to cause interference with usual activity 

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● severe: incapacitating or causing inability to work or to perform usual 
activities 
 
Pregnancy: 
In rare cases, pregnancy might occur in a study. Once a subject has been 
enrolled into the study, after having taken BI Drug administered for the 
disease in scope of the study, the investigator must report any drug 
exposure during pregnancy, which occurred in a female subject or in a 
partner to a male subject to BI by means of BI Pregnancy Monitoring Form 
provided. 
 
In the absence of a reportable AE, only the Pregnancy Monitoring Form must 
be completed, otherwise the NIS AE form is to be completed and forwarded 
as well within the respective timelines.  
 
Reporting of related Adverse Events associated with any other BI drug 
The investigator is encouraged to report all adverse events related to any BI 
drug other than nintedanib according to the local regulatory requirements for 
spontaneous AE reporting at the investigator’s discretion by using the locally 
established routes and AE report forms. The term AE includes drug exposure 
during pregnancy, and, regardless of whether an AE occurred or not, any 
abuse, off-label use, misuse, medication error, occupational exposure, lack 
of effect, and unexpected benefit. 
 
11  Summary of clinical study protocol modifications 
 
None to date.