Trial Outcomes & Findings for Remote Pulmonary Function Testing in Amyotrophic Lateral Sclerosis (Pilot) (NCT NCT03214224)

Results Overview

Respiratory therapist will administer three valid maneuvers of forced vital capacity (FVC) The best FVC value is the outcome.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

49 participants

Primary outcome timeframe

One administration - 10 minutes

Results posted on

2020-08-07

Participant Flow

49 subjects (including 3 patients not completing procedures, 3 controls, and 3 therapists)

Participant milestones

Participant milestones
Measure	Remote PFT (rPFT) Validation Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure.
Overall Study STARTED	49
Overall Study COMPLETED	40
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Remote PFT (rPFT) Validation Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure.
Overall Study Protocol Violation	1
Overall Study Withdrawal by Subject	2
Overall Study Practice controls or therapists	6

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Remote PFT (rPFT) Validation n=40 Participants Subjects perform both standard and remote PFT assessments in order to validate the procedure.
Age, Categorical <=18 years	0 Participants n=40 Participants
Age, Categorical Between 18 and 65 years	21 Participants n=40 Participants
Age, Categorical >=65 years	19 Participants n=40 Participants
Age, Continuous	61.5 years STANDARD_DEVIATION 9.7 • n=40 Participants
Sex: Female, Male Female	16 Participants n=40 Participants
Sex: Female, Male Male	24 Participants n=40 Participants
Region of Enrollment United States	40 participants n=40 Participants
ALS Functional Rating Scale - Revised (ALSFRS-R)	31.3 units on a scale STANDARD_DEVIATION 7.9 • n=40 Participants
Time Since Symptom Onset	59.2 months STANDARD_DEVIATION 56.8 • n=40 Participants
Diagnosis ALS	30 Participants n=40 Participants
Diagnosis PLS	9 Participants n=40 Participants
Diagnosis PMA	1 Participants n=40 Participants

PRIMARY outcome

Timeframe: One administration - 10 minutes

Population: 40 patients taking part in validation study

Respiratory therapist will administer three valid maneuvers of forced vital capacity (FVC) The best FVC value is the outcome.

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=40 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Standard PFT - Forced Vital Capacity	72.7 Percent Predicted FVC Standard Deviation 18.0

PRIMARY outcome

Timeframe: One administration - 10 minutes

Population: 40 patients taking part in validation study

Respiratory therapist will administer three valid maneuvers of maximal inspiratory pressure (MIP). The best MIP value is the outcome.

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=40 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Standard PFT - Maximal Inspiratory Pressure	-68.7 cm water Standard Deviation 26.0

PRIMARY outcome

Timeframe: One administration - 10 minutes

Population: 40 patients taking part in validation study

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.

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=40 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Remote PFT - Forced Vital Capacity	79.7 Percent predicted of FVC Standard Deviation 20.4

PRIMARY outcome

Timeframe: One administration - 10 minutes

Population: 40 patients taking part in validation study

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.

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=40 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Remote PFT - Maximal Inspiratory Pressure	-66.2 cm water Standard Deviation 28.6

SECONDARY outcome

Timeframe: 10 minute survey administered following completion of standard and remote PFT of Part 1

Population: One subject did not complete the survey

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 \[0-5 (worst-best)\]

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=39 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Patient and Caregiver Reported Outcomes General Acceptability	4.50 units on a scale Standard Deviation .58
Patient and Caregiver Reported Outcomes Forced Vital Capacity Acceptability	4.45 units on a scale Standard Deviation .63
Patient and Caregiver Reported Outcomes Maximal Inspiratory Pressure Acceptability	4.48 units on a scale Standard Deviation .59

SECONDARY outcome

Timeframe: 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.

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 \[0-5 (worst-best)\]

Outcome measures

Outcome measures
Measure	Remote PFT (rPFT) Validation n=35 Participants Those in the first part of the study will perform both standard and remote PFT assessments in order to validate the procedure. remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Therapist Reported Outcomes General Acceptability	4.16 units on a scale Standard Deviation .50
Therapist Reported Outcomes Forced Vital Capacity Acceptability	3.75 units on a scale Standard Deviation .63
Therapist Reported Outcomes Maximal Inspiratory Pressure Acceptability	4.04 units on a scale Standard Deviation .44

Adverse Events

Remote PFT (rPFT) Validation

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Andrew Geronimo

Penn State College of Medicine

Phone: 717-531-0003

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place