ALS
Conditions
Keywords
Telemedicine, Pulmonary Function Testing, Telehealth
Brief summary
The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program.
Detailed description
The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program. The central hypothesis is that guided home assessment of respiratory function is a valid method for detecting respiratory insufficiency leading to noninvasive ventilation (NIV) recommendation. This study has the potential to transform the current practice of conducting breathing assessments every three months, resulting in timelier detection of respiratory insufficiency, thereby staining quality of life and lengthening survival. This protocol has the potential to demonstrate telemanagement exceeding the standards of ALS care. This is a self-controlled study which will enroll 40 patients from the ALS clinic. On the day of their clinical visit, study participants will perform both a standard PFT as well as a simulated rPFT, both generating three valid repetitions of forced vital capacity (FVC) and maximal inspiratory pressure (MIP) procedures. The simulated rPFT will mimic the practice of home telemonitoring by having patients be instructed by a respiratory therapist over the telemanagement portal while in a research room within the ALS clinic. The primary hypothesis is that there is no difference in the results of PFT and rPFT for respiratory assessment of FVC and MIP.
Interventions
Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Sponsors
Milton S. Hershey Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Intervention model description
A self-controlled validation study of experimental remote PFT assessment vs standard of care (non-randomized)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Part 1 Patients: 1. Possess a diagnosis of definite, probable, probable laboratory-supported, or possible ALS by revised El Escorial research criteria \[Brooks2000\]. 2. Be 18 years of age or older. 3. Have a caregiver available to participate in the study Caregivers: 1. Be 18 years of age or older, of either gender. 2. Be able and willing to provide informed consent. Respiratory Therapist 1. Be a member of the Hershey Medical Center ALS multidisciplinary care team. 2. Be able and willing to provide verbal informed consent after receiving a summary explanation of research (SER). Part 2 imposes additional inclusion criteria for patients only. Patients: 4\) Symptom onset within the last three years. 5) Have a computer and home internet service sufficient for engaging in telemedicine sessions. 6\) Have a second device capable of downloading the spirometer application from an app store (Android- or iOS-based smartphone or tablet).
Exclusion criteria
*
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Standard PFT - Forced Vital Capacity | One administration - 10 minutes | Respiratory therapist will administer three valid maneuvers of forced vital capacity (FVC) The best FVC value is the outcome. |
| Standard PFT - Maximal Inspiratory Pressure | One administration - 10 minutes | Respiratory therapist will administer three valid maneuvers of maximal inspiratory pressure (MIP). The best MIP value is the outcome. |
| Remote PFT - Forced Vital Capacity | One administration - 10 minutes | Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid FVC maneuvers. The best FVC value is the outcome. |
| Remote PFT - Maximal Inspiratory Pressure | One administration - 10 minutes | Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid MIP maneuvers. The best MIP value is the outcome. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Patient and Caregiver Reported Outcomes | 10 minute survey administered following completion of standard and remote PFT of Part 1 | Survey responses from the patient/caregiver pair. Likert-type scales are used to generate subscores pertaining to: General Acceptability, Forced Vital Capacity Acceptability, and Maximal Inspiratory Pressure Acceptability. Subscales (evaluated separately): General Acceptability \[0-5 (worst-best)\] Forced Vital Capacity Acceptability \[0-5 (worst-best) Maximal Inspiratory Pressure Acceptability \[0-5 (worst-best)\] |
| Therapist Reported Outcomes | 10 minute survey administered following completion of standard and remote PFT of Part 1 | Survey responses from the respiratory therapist. Likert-type scales are used to generate subscores pertaining to: General Acceptability, Forced Vital Capacity Acceptability, and Maximal Inspiratory Pressure Acceptability. Subscales (evaluated separately): General Acceptability \[0-5 (worst-best)\] Forced Vital Capacity Acceptability \[0-5 (worst-best) Maximal Inspiratory Pressure Acceptability \[0-5 (worst-best)\] |
Countries
United States
Participant flow
Recruitment details
49 subjects (including 3 patients not completing procedures, 3 controls, and 3 therapists)
Participants by arm
| Arm | Count |
|---|---|
| Remote PFT (rPFT) Validation Subjects perform both standard and remote PFT assessments in order to validate the procedure. | 40 |
| Total | 40 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Practice controls or therapists | 6 |
| Overall Study | Protocol Violation | 1 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Remote PFT (rPFT) Validation | — |
|---|---|---|
| Age, Categorical <=18 years | 0 Participants | — |
| Age, Categorical >=65 years | 19 Participants | — |
| Age, Categorical Between 18 and 65 years | 21 Participants | — |
| Age, Continuous | 61.5 years STANDARD_DEVIATION 9.7 | — |
| ALS Functional Rating Scale - Revised (ALSFRS-R) | 31.3 units on a scale STANDARD_DEVIATION 7.9 | — |
| Diagnosis ALS | 30 Participants | — |
| Diagnosis PLS | 9 Participants | — |
| Diagnosis PMA | 1 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment United States | 40 participants | — |
| Sex: Female, Male Female | 16 Participants | — |
| Sex: Female, Male Male | 24 Participants | — |
| Time Since Symptom Onset | 59.2 months STANDARD_DEVIATION 56.8 | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 40 |
| other Total, other adverse events | 0 / 40 |
| serious Total, serious adverse events | 0 / 40 |
Outcome results
Primary
Remote PFT - Forced Vital Capacity
Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid FVC maneuvers. The best FVC value is the outcome.
Time frame: One administration - 10 minutes
Population: 40 patients taking part in validation study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Remote PFT (rPFT) Validation | Remote PFT - Forced Vital Capacity | 79.7 Percent predicted of FVC | Standard Deviation 20.4 |
Primary
Remote PFT - Maximal Inspiratory Pressure
Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid MIP maneuvers. The best MIP value is the outcome.
Time frame: One administration - 10 minutes
Population: 40 patients taking part in validation study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Remote PFT (rPFT) Validation | Remote PFT - Maximal Inspiratory Pressure | -66.2 cm water | Standard Deviation 28.6 |
Primary
Standard PFT - Forced Vital Capacity
Respiratory therapist will administer three valid maneuvers of forced vital capacity (FVC) The best FVC value is the outcome.
Time frame: One administration - 10 minutes
Population: 40 patients taking part in validation study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Remote PFT (rPFT) Validation | Standard PFT - Forced Vital Capacity | 72.7 Percent Predicted FVC | Standard Deviation 18 |
Primary
Standard PFT - Maximal Inspiratory Pressure
Respiratory therapist will administer three valid maneuvers of maximal inspiratory pressure (MIP). The best MIP value is the outcome.
Time frame: One administration - 10 minutes
Population: 40 patients taking part in validation study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Remote PFT (rPFT) Validation | Standard PFT - Maximal Inspiratory Pressure | -68.7 cm water | Standard Deviation 26 |
Secondary
Patient and Caregiver Reported Outcomes
Survey responses from the patient/caregiver pair. Likert-type scales are used to generate subscores pertaining to: General Acceptability, Forced Vital Capacity Acceptability, and Maximal Inspiratory Pressure Acceptability. Subscales (evaluated separately): General Acceptability \[0-5 (worst-best)\] Forced Vital Capacity Acceptability \[0-5 (worst-best) Maximal Inspiratory Pressure Acceptability \[0-5 (worst-best)\]
Time frame: 10 minute survey administered following completion of standard and remote PFT of Part 1
Population: One subject did not complete the survey
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Remote PFT (rPFT) Validation | Patient and Caregiver Reported Outcomes | General Acceptability | 4.50 units on a scale | Standard Deviation 0.58 |
| Remote PFT (rPFT) Validation | Patient and Caregiver Reported Outcomes | Forced Vital Capacity Acceptability | 4.45 units on a scale | Standard Deviation 0.63 |
| Remote PFT (rPFT) Validation | Patient and Caregiver Reported Outcomes | Maximal Inspiratory Pressure Acceptability | 4.48 units on a scale | Standard Deviation 0.59 |
Secondary
Therapist Reported Outcomes
Survey responses from the respiratory therapist. Likert-type scales are used to generate subscores pertaining to: General Acceptability, Forced Vital Capacity Acceptability, and Maximal Inspiratory Pressure Acceptability. Subscales (evaluated separately): General Acceptability \[0-5 (worst-best)\] Forced Vital Capacity Acceptability \[0-5 (worst-best) Maximal Inspiratory Pressure Acceptability \[0-5 (worst-best)\]
Time frame: 10 minute survey administered following completion of standard and remote PFT of Part 1
Population: There were three therapists who produced 35 separate survey responses. Responses from 5 patient interactions were not available.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Remote PFT (rPFT) Validation | Therapist Reported Outcomes | General Acceptability | 4.16 units on a scale | Standard Deviation 0.5 |
| Remote PFT (rPFT) Validation | Therapist Reported Outcomes | Forced Vital Capacity Acceptability | 3.75 units on a scale | Standard Deviation 0.63 |
| Remote PFT (rPFT) Validation | Therapist Reported Outcomes | Maximal Inspiratory Pressure Acceptability | 4.04 units on a scale | Standard Deviation 0.44 |