Idiopathic Pulmonary Fibrosis
Conditions
Brief summary
Single-arm, open-label observational study in idiopathic pulmonary fibrosis (IPF) patients receiving usual care at an interstitial lung disease specialist center. The objectives are \[1\] to characterise the longitudinal trends of patient-measured Forced Vital Capacity (FVC) and impact of IPF on daily life Patient Reported Outcome Measures (PROM) in a cohort of patients with IPF \[2\] to determine the correlation (if any) between patient-measured FVC and PROMs with clinic-observed measurements and \[3\] to assess if longitudinal trends in patient-measured FVC are predictive of clinical health outcomes in IPF. An additional purpose is to assess the acceptability and utility of the patientMpower app in helping IPF patients and their healthcare professional caregivers manage their condition. Patients will record FVC, symptoms (e.g. dyspnea) and activity (step count) daily and PROM once a week on the patientMpower app. The planned observation period is sixteen weeks. No additional clinic visits are required (versus usual care). In-clinic assessments of lung function, dyspnea and PROM will be done at baseline and study end. Patients and healthcare professionals will provide their opinion on utility and acceptability of patientMpower app at study end.
Detailed description
This is a single-arm, open-label observational study in IPF patients receiving usual care at an interstitial lung disease specialist centre. The objectives are \[1\] to characterise the longitudinal trends of patient-measured FVC and PROM in a cohort of patients with IPF \[2\] to determine the correlation (if any) between patient-measured FVC and PROMs with clinic-observed measurements and \[3\] to assess if longitudinal trends in patient-measured FVC are predictive of clinical health outcomes in IPF. An additional purpose is to assess the acceptability and utility of the patientMpower app in helping IPF patients and their healthcare professional caregivers manage their condition. This app has been specially developed for patients with lung fibrosis and is owned by patientMpower Ltd., Dublin, Ireland. The app is downloaded to the patient's mobile phone/tablet device. Patients can record symptoms (e.g. dyspnea), activity (steps/day) and lung function (FVC) via a Bluetooth-connected hand-held spirometer. The app can also be used to remind patients to take their medicines. The app includes a PROM to capture impact of IPF on daily life (once/week). This planned observation period is sixteen weeks. No additional clinic visits are required (compared with usual care). Only patients with a confirmed diagnosis of IPF who provide written informed consent will participate. At a usual care visit to the IPF clinic at the study centre, the research team will discuss the study with the patient (face-to-face) and seek written informed consent. The study starts at this visit (baseline). The IPF clinic will record the usual measurements which would be done at a routine visit to assess IPF. These will include FVC, assessment of dyspnea (modified Medical Research Council score) and PROM. After written informed consent, patientMpower Ltd. will send an information pack via e-mail and a Medical International Research Spirobank Smart spirometer to the patient. Technical support on installation of the app and spirometer will be provided by patientMpower Ltd. The patient will use the patientMpower app to record lung function, breathlessness, adherence to medication and symptoms every day at home until their next visit to the IPF clinic. Every week, patients will be reminded to record the impact of IPF on daily life PROM on the app. There will be no changes to the patient's usual care (e.g. prescribed medicines or exercises) during the study. After about sixteen weeks, patients will return to the IPF clinic for routine assessment of their IPF. The clinic will record all of the usual measurements as at baseline. Patients and healthcare professionals will provide their opinion on the utility and acceptability of the patientMpower app. The study is concluded at this visit. However, patients can continue to use the patientMpower app and spirometer if they wish.
Interventions
None listed
Sponsors
Boehringer Ingelheim
patientMpower Ltd.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* confirmed diagnosis of idiopathic pulmonary fibrosis * daily unrestricted access to smartphone/tablet device * has an email address * has home broadband or mobile data package * demonstrates understanding of correct use of spirometer and patientMpower app * able and willing to perform spirometry at home and record information on patientMpower app daily * gives written informed consent
Exclusion criteria
* significant confusion or any concomitant medical condition which would limit teh ability of the patient to record symptoms or use a home spirometer regularly * new prescription of antifibrotic therapy for IPF (e.g. pirfenidone, nintedanib) within four weeks of baseline visit * recent exacerbation of IPF or other clinically significant change in patient's medical condition in the four weeks before the baseline visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Patient-measured Spirometry Trend Over Time | First 7 days at baseline and last 7 days in study up to 116 days | Mean patient-measured Forced Vital Capacity (FVC) in last seven days - FVC in first seven days after baseline |
| Correlation Between Patient-measured and Clinic-measured Spirometry at Baseline | First 7 days after baseline | Bootstrap distribution of correlations analysis of patient-measured versus clinic-measured Forced Vital Capacity (FVC) at baseline calculated as follows. Means of first 7 days of patient-recorded FVC after baseline were calculated. (Patients without a single spirometry value in the 7-day window were excluded). Correlation between in-clinic FVC and patient-recorded FVC was calculated by randomly sampling from the data and calculating the Pearson correlation coefficient. This was repeated 1000 times, giving an empirical distribution for correlation (known as bootstrapping). The mean of this distribution is the inferred correlation. |
| Correlation Between Patient-measured and Clinic-measured Spirometry at End of Study | Last 7 days before end-of-study clinic visit | Bootstrap distribution of correlations analysis of patient-measured versus clinic-measured Forced Vital Capacity (FVC) at end of study calculated as follows: Means of last 7 days of patient-recorded FVC after baseline were calculated. (Patients without a single spirometry value in the 7-day window were excluded). Correlation between in-clinic FVC and patient-recorded FVC was calculated by randomly sampling from the data and calculating the Pearson correlation coefficient. This was repeated 1000 times, giving an empirical distribution for correlation (known as bootstrapping). The mean of this distribution is the inferred correlation. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Patient-reported Patient Reported Outcome Measure at End | End-of-study (116 days) | Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure; psychological & physical impact of breathlessness, psychological well-being, fatigue domains, values range 1-4, higher scores worse outcome. Reported by patients at home. |
| In-clinic Dyspnea Score at Start | Baseline | modified Medical Research Council dyspnea score (range 0-4; worst dyspnea = 4) |
| In-clinic Dyspnea Score at End | 116 days | modified Medical Research Council dyspnea score (range 0-4; worst dyspnea = 4) |
| Patient Opinion: Instructions Understandable? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: Helped me Take Correct Dose of Medicines? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: Helped me Achieve Exercise Goal? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: Helped me Walk Further? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| In-clinic Patient Reported Outcome Measure at Start | Baseline | Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at clinic. |
| Patient Opinion: Useful to Measure Impact of Pulmonary Fibrosis on Daily Life? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: I Liked Using the Application? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: the Application Was Easy to Use? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: it Was Irritating to Use the Application? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| Patient Opinion: Was the Effect of Application on Impact on Daily Life Positive? | 116 days | Number of subjects responding yes. 2 possible responses: yes/no |
| Patient Opinion: I Want to Continue Using the Application? | 116 days | Number of subjects responding yes. 2 possible responses: yes/no |
| Patient Opinion: I Would Recommend the Application to Others? | 116 days | Number of subjects responding yes. 2 possible responses: yes/no |
| Patient Opinion: Using Application Gave me More Confidence? | 116 days | Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree; |
| In-clinic Patient Reported Outcome Measure at End | End-of-study (116 days) | Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at clinic. |
| Patient-reported Patient Reported Outcome Measure at Start | Baseline | Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at home. |
Countries
Ireland
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Treated All patients who gave informed consent, downloaded the patientMpower application and used the patientMpower application with a MIR Spirobank Smart spirometer at least once. | 20 |
| Total | 20 |
Baseline characteristics
| Characteristic | Treated |
|---|---|
| Age, Continuous | 66.6 years STANDARD_DEVIATION 9.02 |
| In-clinic Forced Vital Capacity (absolute) | 3.30 Litres STANDARD_DEVIATION 0.74 |
| In-clinic Forced Vital Capacity (percent predicted) | 92 percent predicted STANDARD_DEVIATION 17 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 20 Participants |
| Region of Enrollment Ireland | 20 participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 20 |
| other Total, other adverse events | 6 / 20 |
| serious Total, serious adverse events | 0 / 20 |
Outcome results
Primary
Correlation Between Patient-measured and Clinic-measured Spirometry at Baseline
Bootstrap distribution of correlations analysis of patient-measured versus clinic-measured Forced Vital Capacity (FVC) at baseline calculated as follows. Means of first 7 days of patient-recorded FVC after baseline were calculated. (Patients without a single spirometry value in the 7-day window were excluded). Correlation between in-clinic FVC and patient-recorded FVC was calculated by randomly sampling from the data and calculating the Pearson correlation coefficient. This was repeated 1000 times, giving an empirical distribution for correlation (known as bootstrapping). The mean of this distribution is the inferred correlation.
Time frame: First 7 days after baseline
Population: Patients in treated dataset for whom data were available in 7 days after baseline
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | Correlation Between Patient-measured and Clinic-measured Spirometry at Baseline | 0.72 Distribution of correlation coefficient | Standard Deviation 0.02 |
Primary
Correlation Between Patient-measured and Clinic-measured Spirometry at End of Study
Bootstrap distribution of correlations analysis of patient-measured versus clinic-measured Forced Vital Capacity (FVC) at end of study calculated as follows: Means of last 7 days of patient-recorded FVC after baseline were calculated. (Patients without a single spirometry value in the 7-day window were excluded). Correlation between in-clinic FVC and patient-recorded FVC was calculated by randomly sampling from the data and calculating the Pearson correlation coefficient. This was repeated 1000 times, giving an empirical distribution for correlation (known as bootstrapping). The mean of this distribution is the inferred correlation.
Time frame: Last 7 days before end-of-study clinic visit
Population: Patients in treated dataset for whom data were available in the 7 days before end of study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | Correlation Between Patient-measured and Clinic-measured Spirometry at End of Study | 0.77 Distribution of correlation coefficient | Standard Deviation 0.01 |
Primary
Patient-measured Spirometry Trend Over Time
Mean patient-measured Forced Vital Capacity (FVC) in last seven days - FVC in first seven days after baseline
Time frame: First 7 days at baseline and last 7 days in study up to 116 days
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | Patient-measured Spirometry Trend Over Time | -0.035 litre | Standard Deviation 0.49 |
Secondary
In-clinic Dyspnea Score at End
modified Medical Research Council dyspnea score (range 0-4; worst dyspnea = 4)
Time frame: 116 days
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Treated | In-clinic Dyspnea Score at End | 2 score on a scale |
Secondary
In-clinic Dyspnea Score at Start
modified Medical Research Council dyspnea score (range 0-4; worst dyspnea = 4)
Time frame: Baseline
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Treated | In-clinic Dyspnea Score at Start | 2 score on a scale |
Secondary
In-clinic Patient Reported Outcome Measure at End
Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at clinic.
Time frame: End-of-study (116 days)
Population: Patients reporting outcomes at end
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | In-clinic Patient Reported Outcome Measure at End | 1.78 score on a scale | Standard Deviation 0.83 |
Secondary
In-clinic Patient Reported Outcome Measure at Start
Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at clinic.
Time frame: Baseline
Population: Patients reporting outcomes at beginning
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | In-clinic Patient Reported Outcome Measure at Start | 1.76 score on a scale | Standard Deviation 0.76 |
Secondary
Patient Opinion: Helped me Achieve Exercise Goal?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Helped me Achieve Exercise Goal? | 9 Participants |
Secondary
Patient Opinion: Helped me Take Correct Dose of Medicines?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Helped me Take Correct Dose of Medicines? | 9 Participants |
Secondary
Patient Opinion: Helped me Walk Further?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Helped me Walk Further? | 6 Participants |
Secondary
Patient Opinion: I Liked Using the Application?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: I Liked Using the Application? | 10 Participants |
Secondary
Patient Opinion: Instructions Understandable?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Instructions Understandable? | 10 Participants |
Secondary
Patient Opinion: it Was Irritating to Use the Application?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: it Was Irritating to Use the Application? | 1 Participants |
Secondary
Patient Opinion: I Want to Continue Using the Application?
Number of subjects responding yes. 2 possible responses: yes/no
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: I Want to Continue Using the Application? | 9 Participants |
Secondary
Patient Opinion: I Would Recommend the Application to Others?
Number of subjects responding yes. 2 possible responses: yes/no
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: I Would Recommend the Application to Others? | 9 Participants |
Secondary
Patient Opinion: the Application Was Easy to Use?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: the Application Was Easy to Use? | 9 Participants |
Secondary
Patient Opinion: Useful to Measure Impact of Pulmonary Fibrosis on Daily Life?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Useful to Measure Impact of Pulmonary Fibrosis on Daily Life? | 9 Participants |
Secondary
Patient Opinion: Using Application Gave me More Confidence?
Number of subjects responding strongly agree or agree shown. 4 possible responses: strongly agree/agree/disagree/strongly disagree;
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Using Application Gave me More Confidence? | 8 Participants |
Secondary
Patient Opinion: Was the Effect of Application on Impact on Daily Life Positive?
Number of subjects responding yes. 2 possible responses: yes/no
Time frame: 116 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treated | Patient Opinion: Was the Effect of Application on Impact on Daily Life Positive? | 9 Participants |
Secondary
Patient-reported Patient Reported Outcome Measure at End
Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure; psychological & physical impact of breathlessness, psychological well-being, fatigue domains, values range 1-4, higher scores worse outcome. Reported by patients at home.
Time frame: End-of-study (116 days)
Population: Patients reporting outcome measure at home (not available for all subjects entered)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | Patient-reported Patient Reported Outcome Measure at End | 1.79 score on a scale | Standard Deviation 0.93 |
Secondary
Patient-reported Patient Reported Outcome Measure at Start
Idiopathic Pulmonary Fibrosis Patient Reported Outcome Measure. Combined score from 4 domains (psychological & physical impact of breathlessness, psychological well-being, fatigue). Values range: 1-4. Higher scores indicate worse outcome. Reported by patient at home.
Time frame: Baseline
Population: Patients reporting outcome measure at home (not available for all subjects entered)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treated | Patient-reported Patient Reported Outcome Measure at Start | 1.60 score on a scale | Standard Deviation 0.6 |