Trial Outcomes & Findings for Study of Standard Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in ALS Patients (NCT NCT01154283)

Results Overview

Patients experienced the first intervention period during weeks 1-6 and the second intervention period during weeks 7-12. Patients were given one week to learn how to use the NIPPV equipment at the beginning of each intervention period. Therefore, only the total NIPPV hours used during the last 5 weeks of each intervention period were collected and divided by the number of weeks to obtain the average, weekly hours of NIPPV usage assessed at 6 weeks (first intervention) and 12 weeks (second intervention) for each patient.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

28 participants

Primary outcome timeframe

Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)

Results posted on

2017-01-02

Participant Flow

Participant milestones

Participant milestones
Measure	Bi-level, Standard, NIPPV Then IPAP-only NIPPV Standard NIPPV with both an inspiratory and expiratory positive airway pressure then NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	IPAP-only, NIPPV Then Bi-level, Standard NIPPV NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure then Standard NIPPV with both an inspiratory and expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)
0-6 Weeks STARTED	14	14
0-6 Weeks COMPLETED	7	11
0-6 Weeks NOT COMPLETED	7	3
7-12 Weeks STARTED	7	11
7-12 Weeks COMPLETED	6	10
7-12 Weeks NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Bi-level, Standard, NIPPV Then IPAP-only NIPPV Standard NIPPV with both an inspiratory and expiratory positive airway pressure then NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	IPAP-only, NIPPV Then Bi-level, Standard NIPPV NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure then Standard NIPPV with both an inspiratory and expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)
0-6 Weeks Withdrawal by Subject	7	2
0-6 Weeks Death	0	1
7-12 Weeks Death	1	0
7-12 Weeks Withdrawal by Subject	0	1

Baseline Characteristics

Study of Standard Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in ALS Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bi-level, Standard, NIPPV Then IPAP-only NIPPV n=14 Participants Standard NIPPV with both an inspiratory and expiratory positive airway pressure then NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	IPAP-only, NIPPV Then Bi-level, Standard NIPPV n=14 Participants NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure then Standard NIPPV with both an inspiratory and expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	Total n=28 Participants Total of all reporting groups
Age, Continuous	62 years n=5 Participants	59 years n=7 Participants	60 years n=5 Participants
Gender Female	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Gender Male	8 Participants n=5 Participants	7 Participants n=7 Participants	15 Participants n=5 Participants

PRIMARY outcome

Timeframe: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)

Patients experienced the first intervention period during weeks 1-6 and the second intervention period during weeks 7-12. Patients were given one week to learn how to use the NIPPV equipment at the beginning of each intervention period. Therefore, only the total NIPPV hours used during the last 5 weeks of each intervention period were collected and divided by the number of weeks to obtain the average, weekly hours of NIPPV usage assessed at 6 weeks (first intervention) and 12 weeks (second intervention) for each patient.

Outcome measures

Outcome measures
Measure	Bi-level, Standard, NIPPV n=17 Participants Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation	IPAP-only, NIPPV n=17 Participants NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation
Hours of NIPPV Usage	49.94 hours Interval 3.6 to 126.8	45.75 hours Interval 0.0 to 159.2

SECONDARY outcome

Timeframe: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)

Population: One patient died before completion of 12 weeks (second intervention) and could not have patient preference assessed.

Patient Preference of NIPPV mode: definitely IPAP-only probably IPAP-only uncertain probably Bi-level definitely Bi-level Number of patients who chose definitely or probably are reported.

Outcome measures

Outcome measures
Measure	Bi-level, Standard, NIPPV n=16 Participants Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation	IPAP-only, NIPPV n=16 Participants NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation
Patient Preference (Likert Scale) "Definitely IPAP-only" "Probably IPAP-only" "Uncertain" "Probably Bi-level" "Definitely Bi-level"	2 participants	12 participants

SECONDARY outcome

Timeframe: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)

Population: One patient died and unable to report dyspnea at end of 12 weeks. Two additional patients completed 12 weeks study but did not complete dyspnea assessments.

Transitional Dyspnea Index scores patient reported changes in difficulty breathing during each intervention period from -3 (major deterioration) to +3 (major improvement) in three categories: functional impairment, magnitude of task, and magnitude of effort. The total TDI score ranges from -9 to +9.

Outcome measures

Outcome measures
Measure	Bi-level, Standard, NIPPV n=14 Participants Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation	IPAP-only, NIPPV n=14 Participants NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation
Transitional Dyspnea Index	-3.14 units on a scale Standard Deviation 2.98	-1.21 units on a scale Standard Deviation 2.72

SECONDARY outcome

Timeframe: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)

Population: One patient died and unable to report quality of life at end of 12 weeks. Two additional patients completed 12 weeks study but did not complete quality of life assessments.

Quality of life assessed with visual analogue scale. Where the patient is asked to place a mark on a piece of paper with a vertical, scale from 100 at the top to 0 at the bottom of the scale. Where the patient is informed that 100 represents his or her best imaginable health state and 0 is his or her worst imaginable health state.

Outcome measures

Outcome measures
Measure	Bi-level, Standard, NIPPV n=14 Participants Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation	IPAP-only, NIPPV n=14 Participants NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation
EuroQol Visual Analogue Scale(VAS)	52.86 units on a scale Standard Deviation 25.32	62.79 units on a scale Standard Deviation 17.28

Adverse Events

Bi-level, Standard, NIPPV

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

IPAP-only, NIPPV

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bi-level, Standard, NIPPV n=28 participants at risk Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	IPAP-only, NIPPV n=28 participants at risk NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)
Gastrointestinal disorders Dysphagia	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.
Respiratory, thoracic and mediastinal disorders Bronchial Disorder	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.
Respiratory, thoracic and mediastinal disorders Pulmonary Embolism	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.
Infections and infestations Lower Respiratory Tract Infection	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.
Infections and infestations Urinary Tract Infection	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.
Gastrointestinal disorders Gastrointestinal motility and defaecation conditions	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.

Other adverse events

Other adverse events
Measure	Bi-level, Standard, NIPPV n=28 participants at risk Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)	IPAP-only, NIPPV n=28 participants at risk NIPPV with only inspiratory positive airway pressure (IPAP-only), no expiratory positive airway pressure. Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine): Noninvasive positive airway pressure ventilation (NIPPV)
Gastrointestinal disorders Dysphagia	10.7% 3/28 • Collected during study intervention time frame of 12 weeks.	0.00% 0/28 • Collected during study intervention time frame of 12 weeks.
Respiratory, thoracic and mediastinal disorders Dyspnea	10.7% 3/28 • Collected during study intervention time frame of 12 weeks.	10.7% 3/28 • Collected during study intervention time frame of 12 weeks.
Respiratory, thoracic and mediastinal disorders Cough Decreased	3.6% 1/28 • Collected during study intervention time frame of 12 weeks.	7.1% 2/28 • Collected during study intervention time frame of 12 weeks.

Additional Information

Kirsten Gruis

UMichigan

Phone: 7346041172

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place