Amyotrophic Lateral SclerosisNCT01154283
Study of Standard Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in ALS Patients
A study testing Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine) in people with Amyotrophic Lateral Sclerosis, run by Kirsten Gruis at 1 site in United States.
Trial summary
The purpose of the study is to test whether noninvasive positive pressure ventilation (NIPPV) without expiratory positive airway pressure (EPAP) (inspiratory positive airway pressure (IPAP)-only) will result in an increase in patient usage of NIPPV compared with standard, Bi-level NIPPV. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Oct 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age greater than 18 years
- Definite or probable ALS by El Escorial criteria
- Maximal inspiratory force < 60 cm/H2O or FVC <50% predicted (American College of Chest Physicians' criteria for initiating NIPPV)
- Patients must have used NIPPV (typically administered as BiPAP) for at least 2 months
- Currently using NIPPV ≥5 days a week with an EPAP = 4 cm H2O.
Who cannot join
- Any medical condition that will interfere with participation
- Inability to consent for him/herself
- Known obstructive sleep apnea or obstructive pulmonary disease.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized Crossover Study of Standard, Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in Amyotrophic Lateral Sclerosis (ALS) Patients
The purpose of the study is to test whether noninvasive positive pressure ventilation (NIPPV) without expiratory positive airway pressure (EPAP) (inspiratory positive airway pressure (IPAP)-only) will result in an increase in patient usage of NIPPV compared with standard, Bi-level NIPPV. Secondarily, the investigators will assess measures of dyspnea, quality of life, patient satisfaction, and side effects.
Read the detailed description
Subjects will be randomized on a 1:1 basis to one of two groups differing in the sequence of NIPPV settings ((a) standard, Bi-level NIPPV followed by IPAP-only NIPPV or (b) IPAP-only NIPPV followed by standard, Bi-level, NIPPV). Subjects will then spend 6 weeks using the first NIPPV set-up before crossing over to the other NIPPV treatment for 6 weeks. At the beginning of the study, subjects will undergo approximately two hours of training and education by a respiratory therapist regarding proper use of the Viasys® Healthcare, Pulmonetic Systems, lap-top ventilators (LTV machine). They will be fitted with the appropriate NIPPV set-up. The patients will use the same mask interface with the study NIPPV machine that they were using with their NIPPV (bi-level positive airway pressure (BiPAP)) machine prior to study entry.
The patients will have two additional study visits after the consent/training session: one at the end of 6 weeks, after the first NIPPV treatment period (prior to crossing over to the second NIPPV treatment), and the second at the end of 12 weeks, after the second NIPPV treatment period.
NIPPV usage will be recorded as hours of use as measured objectively by the NIPPV machine hour meter. When the patient returns for his/her first study visit at the end of week six, the NIPPV machine hour meter will be recorded by the study investigator. By subtracting the hours recorded at the beginning of week two from the hours recorded at the end of week six, the NIPPV hours of usage for weeks two, three, four, five and six will be obtained. This same procedure will be repeated to record NIPPV hours of usage for the beginning of week eight and the end of week twelve to measure the hours of usage with the second NIPPV set-up for weeks eight, nine, ten, eleven and twelve. This allows study subjects one week to adjust to the new pressure settings of each treatment arm.
Dyspnea as measured by the transition dyspnea index (TDI), and quality of life as measured by the EuroQol Visual analogue scale(VAS) will be re-administered at both 6 and 12 week study visits by study personnel. Lastly, patient preference of NIPPV treatment will be subjectively assessed at the 12 week study visit on a Likert scale (definitely first treatment, probably first treatment, no preference, probably second treatment, definitely second treatment).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)Device
Noninvasive positive airway pressure ventilation
| Study group | Role | What they receive |
|---|---|---|
| Bi-level, standard, NIPPV | Active comparator | Standard NIPPV with both an inspiratory and expiratory positive airway pressure. Receives: Device: Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine) |
| IPAP-only, NIPPV | Experimental | NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure Receives: Device: Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hours of NIPPV Usage
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.
Measured: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
Secondary outcomes (3)
Patient Preference (Likert Scale) "Definitely IPAP-only" "Probably IPAP-only" "Uncertain" "Probably Bi-level" "Definitely Bi-level"
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.
Measured: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
Transitional Dyspnea Index
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.
Measured: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
EuroQol Visual Analogue Scale(VAS)
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.
Measured: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2010 (estimated)
- Study startJan 2008
- Primary completionOct 2011
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kirsten Gruis
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association
- Responsible party
- Sponsor-investigator, Kirsten Gruis, University of Michigan
- Investigators
- Kirsten Gruis, MD, Principal investigator, University of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01154283
- Sponsor's protocol ID
- ALSA-9611
- Status last verified
- Nov 2016
- First submitted
- 29 Jun 2010
- Last updated
- 2 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Crossover Study of Standard, Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in Amyotrophic Lateral Sclerosis (ALS) Patients
- ClinicalTrials.gov ID
- NCT01154283
- Acronym / sponsor ID
- ALSA-9611
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Kirsten GruisUniversity, hospital or other
- Collaborators
- ALS Association
- Study start
- Jan 2008
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 30 Jun 2010 (estimated)
- Results
- Posted 2 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to test whether noninvasive positive pressure ventilation (NIPPV) without expiratory positive airway pressure (EPAP) (inspiratory positive airway pressure (IPAP)-only) will result in an increase in patient usage of NIPPV compared with standard, Bi-level NIPPV. Secondarily, the investigators will assess measures of dyspnea, quality of life, patient satisfaction, and side effects.
Detailed description
Subjects will be randomized on a 1:1 basis to one of two groups differing in the sequence of NIPPV settings ((a) standard, Bi-level NIPPV followed by IPAP-only NIPPV or (b) IPAP-only NIPPV followed by standard, Bi-level, NIPPV). Subjects will then spend 6 weeks using the first NIPPV set-up before crossing over to the other NIPPV treatment for 6 weeks. At the beginning of the study, subjects will undergo approximately two hours of training and education by a respiratory therapist regarding proper use of the Viasys® Healthcare, Pulmonetic Systems, lap-top ventilators (LTV machine). They will be fitted with the appropriate NIPPV set-up. The patients will use the same mask interface with the study NIPPV machine that they were using with their NIPPV (bi-level positive airway pressure (BiPAP)) machine prior to study entry.
The patients will have two additional study visits after the consent/training session: one at the end of 6 weeks, after the first NIPPV treatment period (prior to crossing over to the second NIPPV treatment), and the second at the end of 12 weeks, after the second NIPPV treatment period.
NIPPV usage will be recorded as hours of use as measured objectively by the NIPPV machine hour meter. When the patient returns for his/her first study visit at the end of week six, the NIPPV machine hour meter will be recorded by the study investigator. By subtracting the hours recorded at the beginning of week two from the hours recorded at the end of week six, the NIPPV hours of usage for weeks two, three, four, five and six will be obtained. This same procedure will be repeated to record NIPPV hours of usage for the beginning of week eight and the end of week twelve to measure the hours of usage with the second NIPPV set-up for weeks eight, nine, ten, eleven and twelve. This allows study subjects one week to adjust to the new pressure settings of each treatment arm.
Dyspnea as measured by the transition dyspnea index (TDI), and quality of life as measured by the EuroQol Visual analogue scale(VAS) will be re-administered at both 6 and 12 week study visits by study personnel. Lastly, patient preference of NIPPV treatment will be subjectively assessed at the 12 week study visit on a Likert scale (definitely first treatment, probably first treatment, no preference, probably second treatment, definitely second treatment).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Bi-level, standard, NIPPV | Active comparator | Device: Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)Standard NIPPV with both an inspiratory and expiratory positive airway pressure. |
| IPAP-only, NIPPV | Experimental | Device: Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)NIPPV with only inspiratory positive airway pressure, no expiratory positive airway pressure |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age greater than 18 years
- Definite or probable ALS by El Escorial criteria
- Maximal inspiratory force < 60 cm/H2O or FVC <50% predicted (American College of Chest Physicians' criteria for initiating NIPPV)
- Patients must have used NIPPV (typically administered as BiPAP) for at least 2 months
- Currently using NIPPV ≥5 days a week with an EPAP = 4 cm H2O.
Key exclusion criteria (3 listed)
- Any medical condition that will interfere with participation
- Inability to consent for him/herself
- Known obstructive sleep apnea or obstructive pulmonary disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)Noninvasive positive airway pressure ventilation | Device | Bi-level, standard, NIPPV, IPAP-only, NIPPV |
Primary outcome
Hours of NIPPV Usage
Time frame: 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.
3 secondary outcomes
Patient Preference (Likert Scale) "Definitely IPAP-only" "Probably IPAP-only" "Uncertain" "Probably Bi-level" "Definitely Bi-level"
Time frame: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
Transitional Dyspnea Index
Time frame: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
EuroQol Visual Analogue Scale(VAS)
Time frame: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kirsten Gruis, MD principal investigator
University of Michigan
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Viasys® Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine) in people with Amyotrophic Lateral Sclerosis, run by Kirsten Gruis at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hours of NIPPV Usage (time frame: Assessed at 6 weeks (first intervention) and 12 weeks (second intervention)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jan 2017.